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The Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) Partnership on May 18, 2020

Posted in Coronavirus Gene Expression, COVID-19, Drug Development Process, Drug Discovery Chemistry, drug repurposing, Personal Health Applications: Tech Innovations serves HealhCare, Personalized and Precision Medicine & Genomic Research, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, Pharmacogenomics, Pharmacovigilance, Population Health Management, Population Health Management, Genetics & Pharmaceutical, SARS-CoV-2, Serology tests for coronavirus antibodies, Vaccinology, Virus Infective Acute Respiratory Syndrome: SARS-CoV on May 18, 2020| Leave a Comment »


The Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) Partnership on May 18, 2020: Leadership of AbbVie, Amgen, AstraZeneca, Bristol Myers Squibb, Eisai, Eli Lilly, Evotec, Gilead, GlaxoSmithKline, Johnson & Johnson, KSQ Therapeutics, Merck, Novartis, Pfizer, Roche, Sanofi, Takeda, and Vir. We also thank multiple NIH institutes (especially NIAID), the FDA, BARDA, CDC, the European Medicines Agency, the Department of Defense, the VA, and the Foundation for NIH

Reporter: Aviva Lev-Ari, PhD, RN

May 18, 2020

Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) An Unprecedented Partnership for Unprecedented Times

Francis S. Collins, MD, PhD1; Paul Stoffels, MD2
Article Information
JAMA. Published online May 18, 2020. doi:10.1001/jama.2020.8920
Author Affiliations

  • 1Office of the Director, National Institutes of Health, Bethesda, Maryland
  • 2Johnson & Johnson, New Brunswick, New Jersey

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It has been more than a century since the world has encountered a pandemic like coronavirus disease 2019 (COVID-19), and the rate of spread of COVID-19 around the globe and the associated morbidity and mortality have been staggering.1 To address what may be the greatest public health crisis of this generation, it is imperative that all sectors of society work together in unprecedented ways, with unprecedented speed. In this Viewpoint, we describe such a partnership.

First reported in Wuhan, China, in December 2019, COVID-19 is caused by a highly transmissible novel coronavirus, SARS-CoV-2 (severe acute respiratory syndrome coronavirus 2). By March 2020, as COVID-19 moved rapidly throughout Europe and the US, most researchers and regulators from around the world agreed that it would be necessary to go beyond “business as usual” to contain this formidable infectious agent. The biomedical research enterprise was more than willing to respond to the challenge of COVID-19, but it soon became apparent that much-needed coordination among important constituencies was lacking.

Clinical trials of investigational vaccines began as early as January, but with the earliest possible distribution predicted to be 12 to 18 months away. Clinical trials of experimental therapies had also been initiated, but most, except for a trial testing the antiviral drug remdesivir,2 were small and not randomized. In the US, there was no true overarching national process in either the public or private sector to prioritize candidate therapeutic agents or vaccines, and no efforts were underway to develop a clear inventory of clinical trial capacity that could be brought to bear on this public health emergency. Many key factors had to change if COVID-19 was to be addressed effectively in a relatively short time frame.

On April 3, leaders of the National Institutes of Health (NIH), with coordination by the Foundation for the National Institutes of Health (FNIH), met with multiple leaders of research and development from biopharmaceutical firms, along with leaders of the US Food and Drug Administration (FDA), the Biomedical Advanced Research and Development Authority (BARDA), the European Medicines Agency (EMA), and academic experts. Participants sought urgently to identify research gaps and to discuss opportunities to collaborate in an accelerated fashion to address the complex challenges of COVID-19.

These critical discussions culminated in a decision to form a public-private partnership to focus on speeding the development and deployment of therapeutics and vaccines for COVID-19. The group assembled 4 working groups to focus on preclinical therapeutics, clinical therapeutics, clinical trial capacity, and vaccines (Figure). In addition to the founding members, the working groups’ membership consisted of senior scientists from each company or agency, the Centers for Disease Control and Prevention (CDC), the Department of Veterans Affairs (VA), and the Department of Defense.

Figure.

Accelerating COVID-19 Therapeutic Interventions and Vaccines

ACTIV’s 4 working groups, each with one cochair from NIH and one from industry, have made rapid progress in establishing goals, setting timetables, and forming subgroups focused on specific issues (Figure). The goals of the working group, along with a few examples of their accomplishments to date, include the following.

 

The Preclinical Working Group was charged to standardize and share preclinical evaluation resources and methods and accelerate testing of candidate therapies and vaccines to support entry into clinical trials. The aim is to increase access to validated animal models and to enhance comparison of approaches to identify informative assays. For example, through the ACTIV partnership, this group aims to extend preclinical researchers’ access to high-throughput screening systems, especially those located in the Biosafety Level 3 (BSL3) facilities currently required for many SARS-CoV-2 studies. This group also is defining a prioritization approach for animal use, assay selection and staging of testing, as well as completing an inventory of animal models, assays, and BSL 3/4 facilities.

 

The Therapeutics Clinical Working Group has been charged to prioritize and accelerate clinical evaluation of a long list of therapeutic candidates for COVID-19 with near-term potential. The goals have been to prioritize and test potential therapeutic agents for COVID-19 that have already been in human clinical trials. These may include agents with either direct-acting or host-directed antiviral activity, including immunomodulators, severe symptom modulators, neutralizing antibodies, or vaccines. To help achieve these goals, the group has established a steering committee with relevant expertise and objectivity to set criteria for evaluating and ranking potential candidate therapies submitted by industry partners. Following a rigorous scientific review, the prioritization subgroup has developed a complete inventory of approximately 170 already identified therapeutic candidates that have acceptable safety profiles and different mechanisms of action. On May 6, the group presented its first list of repurposed agents recommended for inclusion in ACTIV’s master protocol for adaptive clinical trials. Of the 39 agents that underwent final prioritization review, the group identified 6 agents—including immunomodulators and supportive therapies—that it proposes to move forward into the master protocol clinical trial(s) expected to begin later in May.

 

The Clinical Trial Capacity Working Group is charged with assembling and coordinating existing networks of clinical trials to increase efficiency and build capacity. This will include developing an inventory of clinical trial networks supported by NIH and other funders in the public and private sectors, including contract research organizations. For each network, the working group seeks to identify their specialization in different populations and disease stages to leverage infrastructure and expertise from across multiple networks, and establish a coordination mechanism across networks to expedite trials, track incidence across sites, and project future capacity. The clinical trials inventory subgroup has already identified 44 networks, with access to adult populations and within domestic reach, for potential inclusion in COVID-19 trials. Meanwhile, the survey subgroup has developed 2 survey instruments to assess the capabilities and capacities of those networks, and its innovation subgroup has developed a matrix to guide deployment of innovative solutions throughout the trial life cycle.

 

The Vaccines Working Group has been charged to accelerate evaluation of vaccine candidates to enable rapid authorization or approval.4 This includes development of a harmonized master protocol for adaptive trials of multiple vaccines, as well as development of a trial network that could enroll as many as 100 000 volunteers in areas where COVID-19 is actively circulating. The group also aims to identify biomarkers to speed authorization or approval and to provide evidence to address cross-cutting safety concerns, such as immune enhancement. Multiple vaccine candidates will be evaluated, and the most promising will move to a phase 2/3 adaptive trial platform utilizing large geographic networks in the US and globally.5 Because time is of the essence, ACTIV will aim to have the next vaccine candidates ready to enter clinical trials by July 1, 2020.

References

1.

Desai  A .  Twentieth-century lessons for a modern coronavirus pandemic.   JAMA. Published online April 27, 2020. doi:10.1001/jama.2020.4165
ArticlePubMedGoogle Scholar

2.

NIH clinical trial shows remdesivir accelerates recovery from advanced COVID-19. National Institutes of Health. Published April 29, 2020. Accessed May 7, 2020. https://www.nih.gov/news-events/news-releases/nih-clinical-trial-shows-remdesivir-accelerates-recovery-advanced-covid-19

3.

NIH to launch public-private partnership to speed COVID-19 vaccine and treatment options. National Institutes of Health. Published April 17, 2020. Accessed May 7, 2020. https://www.nih.gov/news-events/news-releases/nih-launch-public-private-partnership-speed-covid-19-vaccine-treatment-options

4.

Corey  L , Mascola  JR , Fauci  AS , Collins  FS .  A strategic approach to COVID-19 vaccine R&D.   Science. Published online May 11, 2020. doi:10.1126/science.abc5312PubMedGoogle Scholar

5.

Angus  DC .  Optimizing the trade-off between learning and doing in a pandemic.   JAMA. Published online March 30, 2020. doi:10.1001/jama.2020.4984
ArticlePubMedGoogle Scholar

6.

Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) portal. National Institutes of Health. Accessed May 15, 2020. https://www.nih.gov/ACTIV

7.

Accelerating Medicines Partnership (AMP). National Institutes of Health. Published February 4, 2014. Accessed May 7, 2020. https://www.nih.gov/research-training/accelerating-medicines-partnership-amp
SOURCE

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Advancing Drug Development – 12/12/2019, 8:30AM – 8:30PM at The University of Massachusetts Club, One Beacon Street, Boston, MA

Posted in Drug Delivery Platform Technology, Drug Development Process, Drug Discovery Chemistry, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, Pharmacogenomics, Rapid automation of plasma protein pools on November 4, 2019| Leave a Comment »


Advancing Drug Development – 12/12/2019, 8:30AM – 8:30PM at The University of Massachusetts Club, One Beacon Street, Boston, MA

 

Reporter: Aviva Lev-Ari, PhD, RN

4th Advancing Drug Development Forum – Making the Impossible Possible – Harnessing Small Molecule Drug Development scheduled to take place December 12th, 2019 at The University of Massachusetts Club, in Boston, Massachusetts from 8:30 AM – 8:30 PM.

http://advdrug.com/agenda/

 

Scientists are more than just chipping away and kicking down the barricades to develop complex small molecule products better and faster.  Successful companies are spending quality time finding novel and clever approaches and powerful technologies to better support their knowledgeable teams.  Often it takes establishing strong partnerships with 1 or more specialized service providers, cleverly combining resources – always striving to raise the bar in order to make life threatening diseases more of a chronic and tolerable disease or eradicated completely.

Hear from key opinion leaders in pharma, biotech, the investment community and innovative service providers on how they are meeting the challenges. Keep in mind, it takes being open-minded, flexible and willing sometimes to redesigning a new formulation that better enhances bioavailability, optimizes drug-delivery profiles, reduces dosing frequency, or improves the patient experience to have the potential to deliver quicker returns on investments than developing a completely new drug.

PROGRAM AGENDA Thursday, December 12, 2019
8:30 AM Registration and Networking Continental Breakfast
9:00 AM Welcome Address and Opening Remarks
Kevin Bittorf, Ph.D., & Shelly Amster
9:15 AM Opening VC Keynote
9:45 AM Bridging the Gap between Experimentation and Implementation
Panel Discussion
10:15 AM Refreshment Break
10:45 AM Cross-Talk Between Clin-Ops and Tech-Ops
Panel Discussion
11:15 AM The Cost of Speed and Value in Drug Development
Panel Discussion
12:00 PM Networking Luncheon
1:00 PM Advances in the Delivery of Therapeutics to the Brain
Academic Keynote
Mansoor M. Amiji, Ph.D., University Distinguished Professor, Professor of Pharmaceutical Sciences & Professor of Chemical Engineering, Northeastern University
1:30 PM Advancing Drug Delivery and Controlled Release
Panel Discussion
2:00 PM Drowning in DATA
2:30 PM Disruptive AI Technologies Improving Drug Development
3:00 PM Refreshment Break
3:30 PM Small Specialty VS Full Service – What Makes Sense for US?
Panel Discussion
4:00 PM Fireside Chat
Michael Bonney, Executive Chair, Kaleido Biosciences
Heinrich Schlieker, Ph.D., SVP Technical Operations, Sage Therapeutics
5:00 PM – 8:00 PM Networking Social
Direct electronic communication with Shelly Amster

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scPopCorn: A New Computational Method for Subpopulation Detection and their Comparative Analysis Across Single-Cell Experiments

Posted in Advanced Computing Platform, Artificial Intelligence Applications in Health Care, Artificial Intelligence in Health Care - Tools & Innovations, Artificial Intelligence in Medicine - Applications in Therapeutics, Big Data & Analytics, BioIT: BioInformatics, BioIT: BioInformatics, NGS, Clinical & Translational, Pharmaceutical R&D Informatics, Clinical Genomics, Cancer Informatics, Biotechnology, Cancer Genomics, Cardiovascular Pharmacogenomics, Cell Biology, Clinical Genomics, Computational Biology/Systems and Bioinformatics, Data Science, Genome Biology, Genomic Analysis of EKG Electrophysiology Genomics, Genomic Endocrinology, Genomic Expression, Genomic Testing: Methodology for Diagnosis, Genomics Pharmacy, Immuno-Oncology & Genomics, Nutrigenomics, Personalized and Precision Medicine & Genomic Research, Pharmacogenomics, Preimplantation Genetic Diagnosis and Reproductive Genomics, Reproductive Andrology, Embryology, Genomic Endocrinology, Preimplantation Genetic Diagnosis and Reproductive Genomics, RNA Biology, RNA Biology, Cancer and Therapeutics, Single Cell Genomics, Single-cell sequencing, Uncategorized, tagged , , , , , on July 16, 2019| Leave a Comment »


scPopCorn: A New Computational Method for Subpopulation Detection and their Comparative Analysis Across Single-Cell Experiments

Reporter and Curator: Dr. Sudipta Saha, Ph.D.

 

Present day technological advances have facilitated unprecedented opportunities for studying biological systems at single-cell level resolution. For example, single-cell RNA sequencing (scRNA-seq) enables the measurement of transcriptomic information of thousands of individual cells in one experiment. Analyses of such data provide information that was not accessible using bulk sequencing, which can only assess average properties of cell populations. Single-cell measurements, however, can capture the heterogeneity of a population of cells. In particular, single-cell studies allow for the identification of novel cell types, states, and dynamics.

 

One of the most prominent uses of the scRNA-seq technology is the identification of subpopulations of cells present in a sample and comparing such subpopulations across samples. Such information is crucial for understanding the heterogeneity of cells in a sample and for comparative analysis of samples from different conditions, tissues, and species. A frequently used approach is to cluster every dataset separately, inspect marker genes for each cluster, and compare these clusters in an attempt to determine which cell types were shared between samples. This approach, however, relies on the existence of predefined or clearly identifiable marker genes and their consistent measurement across subpopulations.

 

Although the aligned data can then be clustered to reveal subpopulations and their correspondence, solving the subpopulation-mapping problem by performing global alignment first and clustering second overlooks the original information about subpopulations existing in each experiment. In contrast, an approach addressing this problem directly might represent a more suitable solution. So, keeping this in mind the researchers developed a computational method, single-cell subpopulations comparison (scPopCorn), that allows for comparative analysis of two or more single-cell populations.

 

The performance of scPopCorn was tested in three distinct settings. First, its potential was demonstrated in identifying and aligning subpopulations from single-cell data from human and mouse pancreatic single-cell data. Next, scPopCorn was applied to the task of aligning biological replicates of mouse kidney single-cell data. scPopCorn achieved the best performance over the previously published tools. Finally, it was applied to compare populations of cells from cancer and healthy brain tissues, revealing the relation of neoplastic cells to neural cells and astrocytes. Consequently, as a result of this integrative approach, scPopCorn provides a powerful tool for comparative analysis of single-cell populations.

 

This scPopCorn is basically a computational method for the identification of subpopulations of cells present within individual single-cell experiments and mapping of these subpopulations across these experiments. Different from other approaches, scPopCorn performs the tasks of population identification and mapping simultaneously by optimizing a function that combines both objectives. When applied to complex biological data, scPopCorn outperforms previous methods. However, it should be kept in mind that scPopCorn assumes the input single-cell data to consist of separable subpopulations and it is not designed to perform a comparative analysis of single cell trajectories datasets that do not fulfill this constraint.

 

Several innovations developed in this work contributed to the performance of scPopCorn. First, unifying the above-mentioned tasks into a single problem statement allowed for integrating the signal from different experiments while identifying subpopulations within each experiment. Such an incorporation aids the reduction of biological and experimental noise. The researchers believe that the ideas introduced in scPopCorn not only enabled the design of a highly accurate identification of subpopulations and mapping approach, but can also provide a stepping stone for other tools to interrogate the relationships between single cell experiments.

 

References:

 

https://www.sciencedirect.com/science/article/pii/S2405471219301887

 

https://www.tandfonline.com/doi/abs/10.1080/23307706.2017.1397554

 

https://ieeexplore.ieee.org/abstract/document/4031383

 

https://genomebiology.biomedcentral.com/articles/10.1186/s13059-016-0927-y

 

https://www.sciencedirect.com/science/article/pii/S2405471216302666

 

 

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Verily kicked off Project Baseline in April 2017, with a health study geared to gather health data from 10,000 people over four years – Partnership with Big Pharma on Clinical Trials announced on 5/21/2019

Posted in Health Care System by Country, HealthCare IT, Pharmaceutical R&D Informatics, Pharmacogenomics, United States on May 21, 2019| Leave a Comment »


Verily kicked off Project Baseline in April 2017, with a health study geared to gather health data from 10,000 people over four years – Partnership with Big Pharma on Clinical Trials announced on 5/21/2019

 

Reporter: Aviva Lev-Ari, PhD, RN

 

UPDATED on 5/22/2019

On Tuesday morning, Verily, Alphabet’s unit focused on life sciences, announced that it had formed alliances with Novartis, Sanofi, Otsuka, and Pfizer to work on clinical trials. What are those drug giants getting out of the deal? STAT sat down with Scarlet Shore, who leads Verily’s project Baseline, to learn about the company’s vision for the clinical trial of the future. The conversation took place at CNBC’s “Healthy Returns” conference, where the partnerships were unveiled.

SOURCE

https://www.statnews.com/2019/05/21/four-of-the-worlds-largest-drug-companies-are-teaming-with-verily-here-is-what-they-get/?utm_source=STAT+Newsletters&utm_campaign=1630aad75d-Readout_COPY_03&utm_medium=email&utm_term=0_8cab1d7961-1630aad75d-150237109

Novartis, Otsuka, Pfizer, Sanofi join Verily’s Project Baseline

“Evidence generation through research is the backbone of improving health outcomes. We need to be inclusive and encourage diversity in research to truly understand health and disease, and to provide meaningful insights about new medicines, medical devices and digital health solutions,” said Jessica Mega, M.D., Verily’s chief medical and scientific officer, in the statement. “Novartis, Otsuka, Pfizer and Sanofi have been early adopters of advanced technology and digital tools to improve clinical research operations, and together we’re taking another step towards making research accessible and generating evidence to inform better treatments and care.”
Jessica Mega, M.D., Verily’s chief medical and scientific officer, in the statement. “Novartis, Otsuka, Pfizer and Sanofi have been early adopters of advanced technology and digital tools to improve clinical research operations, and together we’re taking another step towards making research accessible and generating evidence to inform better treatments and care.”
SOURCE

 

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The Journey of Antibiotic Discovery

Posted in Allergy and Infectious Diseases, Antibiotic resistance, Artificial Intelligence Applications in Health Care, Artificial Intelligence in Health Care - Tools & Innovations, Biological Networks, Electronic Health Record, Genomics Pharmacy, Health Care System by Country, Health Economics and Outcomes Research, Health Law & Patient Safety, Healthcare Reform, Human Immune System in Health and in Disease, Infectious Disease & New Antibiotic Targets, Infectious Disease Immunodiagnostics, Metabolomics, Microbiologial genetics, Microbiology, microbiome, Nutrigenomics, Personal Health Applications: Tech Innovations serves HealhCare, Pharmacogenomics, Population Health Management, Population Health Management, Genetics & Pharmaceutical, Population Health Management, Nutrition and Phytochemistry, Proteomics, tagged , , , , , on May 19, 2019| Leave a Comment »


The Journey of Antibiotic Discovery

Reporter and Curator: Dr. Sudipta Saha, Ph.D.

 

The term ‘antibiotic’ was introduced by Selman Waksman as any small molecule, produced by a microbe, with antagonistic properties on the growth of other microbes. An antibiotic interferes with bacterial survival via a specific mode of action but more importantly, at therapeutic concentrations, it is sufficiently potent to be effective against infection and simultaneously presents minimal toxicity. Infectious diseases have been a challenge throughout the ages. From 1347 to 1350, approximately one-third of Europe’s population perished to Bubonic plague. Advances in sanitary and hygienic conditions sufficed to control further plague outbreaks. However, these persisted as a recurrent public health issue. Likewise, infectious diseases in general remained the leading cause of death up to the early 1900s. The mortality rate shrunk after the commercialization of antibiotics, which given their impact on the fate of mankind, were regarded as a ‘medical miracle’. Moreover, the non-therapeutic application of antibiotics has also greatly affected humanity, for instance those used as livestock growth promoters to increase food production after World War II.

 

Currently, more than 2 million North Americans acquire infections associated with antibiotic resistance every year, resulting in 23,000 deaths. In Europe, nearly 700 thousand cases of antibiotic-resistant infections directly develop into over 33,000 deaths yearly, with an estimated cost over €1.5 billion. Despite a 36% increase in human use of antibiotics from 2000 to 2010, approximately 20% of deaths worldwide are related to infectious diseases today. Future perspectives are no brighter, for instance, a government commissioned study in the United Kingdom estimated 10 million deaths per year from antibiotic resistant infections by 2050.

 

The increase in antibiotic-resistant bacteria, alongside the alarmingly low rate of newly approved antibiotics for clinical usage, we are on the verge of not having effective treatments for many common infectious diseases. Historically, antibiotic discovery has been crucial in outpacing resistance and success is closely related to systematic procedures – platforms – that have catalyzed the antibiotic golden age, namely the Waksman platform, followed by the platforms of semi-synthesis and fully synthetic antibiotics. Said platforms resulted in the major antibiotic classes: aminoglycosides, amphenicols, ansamycins, beta-lactams, lipopeptides, diaminopyrimidines, fosfomycins, imidazoles, macrolides, oxazolidinones, streptogramins, polymyxins, sulphonamides, glycopeptides, quinolones and tetracyclines.

 

The increase in drug-resistant pathogens is a consequence of multiple factors, including but not limited to high rates of antimicrobial prescriptions, antibiotic mismanagement in the form of self-medication or interruption of therapy, and large-scale antibiotic use as growth promotors in livestock farming. For example, 60% of the antibiotics sold to the USA food industry are also used as therapeutics in humans. To further complicate matters, it is estimated that $200 million is required for a molecule to reach commercialization, with the risk of antimicrobial resistance rapidly developing, crippling its clinical application, or on the opposing end, a new antibiotic might be so effective it is only used as a last resort therapeutic, thus not widely commercialized.

 

Besides a more efficient management of antibiotic use, there is a pressing need for new platforms capable of consistently and efficiently delivering new lead substances, which should attend their precursors impressively low rates of success, in today’s increasing drug resistance scenario. Antibiotic Discovery Platforms are aiming to screen large libraries, for instance the reservoir of untapped natural products, which is likely the next antibiotic ‘gold mine’. There is a void between phenotanypic screening (high-throughput) and omics-centered assays (high-information), where some mechanistic and molecular information complements antimicrobial activity, without the laborious and extensive application of various omics assays. The increasing need for antibiotics drives the relentless and continuous research on the foreground of antibiotic discovery. This is likely to expand our knowledge on the biological events underlying infectious diseases and, hopefully, result in better therapeutics that can swing the war on infectious diseases back in our favor.

 

During the genomics era came the target-based platform, mostly considered a failure due to limitations in translating drugs to the clinic. Therefore, cell-based platforms were re-instituted, and are still of the utmost importance in the fight against infectious diseases. Although the antibiotic pipeline is still lackluster, especially of new classes and novel mechanisms of action, in the post-genomic era, there is an increasingly large set of information available on microbial metabolism. The translation of such knowledge into novel platforms will hopefully result in the discovery of new and better therapeutics, which can sway the war on infectious diseases back in our favor.

 

References:

 

https://www.mdpi.com/2079-6382/8/2/45/htm

 

https://www.ncbi.nlm.nih.gov/pubmed/19515346

 

https://www.ajicjournal.org/article/S0196-6553(11)00184-2/fulltext

 

https://www.ncbi.nlm.nih.gov/pubmed/21700626

 

http://www.med.or.jp/english/journal/pdf/2009_02/103_108.pdf

 

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LIVE 13th Annual BioPharma and Healthcare Summit, Thursday, May 9, 2019, Marriott Hotel, Cambridge, MA

Posted in BioTechnology - Venture Creation, BioTechnology - Venture Creation, Venture Capital, Conference Coverage with Social Media, Drug Development Process, drug repurposing, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, Pharmacodynamics and Pharmacokinetics, Pharmacogenomics, Pharmacovigilance, Value-based Drug Pricing on April 30, 2019| 2 Comments »


LIVE 13th Annual BioPharma and Healthcare Summit, Thursday, May 9, 2019, Marriott Hotel, Cambridge, MA

 

http://www.usaindiachamber.org

8:40 AM – 9:10 AM Registration and Networking
9:10 AM – 9:20 AM Welcome address: Karun Rishi, President, USAIC

Opening comments: Dr Andrew Plump, President R&D and Director, Takeda Pharmaceuticals
9:20 AM – 9:40 AM Fireside Chat

  • Mark Abdoo, Acting Deputy Commissioner, U.S. Food and Drug Administration
  • Dr Eswara Reddy, Drug Controller General of India, Central Drug Control Organization

Moderator: Sanat Chattopadhyay, President, Merck Manufacturing Division; Merck & Co.
9:40 AM – 10:00 AM Presentation on CAR (chimeric antigen receptor) T-cell Therapies
Dr. Carl June, Director of Translational Research, Abramson Cancer Center University of Pennsylvania Moderator: Dr. Raju Kucherlapati, Professor of Genetics, Harvard Medical School
10:00 AM – 10:50 AM Panel Discussion: Oncology – The Emperor of BioPharma Development

Panelists:

Moderator: Dr. Christiana Bardon, Managing Director, MPM Capital
10:50 AM – 11:20 AM Networking Break
11:20 AM – 12:10 PM Panel Discussion: Future of Clinical Trials and Drug Development

Panelists:

Moderator: Dr. William Chin, Professor of Medicine, Emeritus, Harvard Medical School
12:10 PM – 1:00 PM Panel Discussion: Manufacturing in the Future

Panelists:

  • Hari Bhartia, Founder and Co-Chairman, Jubilant Bhartia Group
  • Mark Abdoo, Acting Deputy Commissioner, U.S. Food and Drug Administration
  • Dr. Paul McKenzie, Executive Vice President, Pharma Operations & Technology, Biogen
  • Sanat Chattopadhyay, President, Merck Manufacturing Division; Merck & Co.
  • Vinay Ranade, Chief Executive Officer, Reliance Life Sciences

Moderator: Professor N. Venkat Venkatraman, Boston University Questrom School of Business
1:00 PM – 1:50 PM Lunch
1:50 PM – 1:55 PM Video message from Suresh Prabhu, Hon’ble Minister of Commerce & Industry, Gov. of India
1:55 PM – 2:45 PM Panel Discussion: One in a million – Emerging trends in Rare Diseases

Panelists:

Moderator: Dr. Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics
2:45 PM – 3:20 PM Networking & Tea Break
3:20 PM – 3:50 PM Fireside Chat: Value and Access – The ongoing debate

Moderator: Dr Andrew Plump, President R&D, Takeda Pharmaceuticals
3:50 PM – 4:10 PM India update on Clinical Trial Regulations

  • Arun Singhal, Additional Secretary, Ministry of Health & Family Welfare, India
  • Dr Eswara Reddy, Drug Controller General of India, Central Drug Control Organization
4:10 PM – 5:00 PM Panel Discussion: Research and Development Strategies and Trends

Panelists:

Moderator: Dr. Martin Mackay, Co-Founde, Rallybio
5:00 PM – 5:05 PM Closing Remarks
5:05 PM – 6:15 PM Cocktails & Networking Reception

Aviva Lev-Ari, PhD, RN & Leaders in Pharmaceutical Business Intelligence (LPBI) Group

will cover the event in Real Time

REAL TIME COVERAGE USING SOCIAL MEDIA

 

LIVE Images taken by @AVIVA1950

 

 

 

9:10 AM – 9:20 AM

Welcome address: Karun Rishi, President, USAIC

Opening comments: Dr Andrew Plump, President R&D and Director, Takeda Pharmaceuticals

  • tomorrow announcement @Shire
  • India 1.3Billion in India, each person is a potential patient in the largest democracy in the World
  • China – transformation takes place every day
  • The Patient and the Pricing of Drugs the biggest issue missing the ball dialoguing on Panel today

9:20 AM – 9:40 AM

Fireside Chat

  • Mark Abdoo, Acting Deputy Commissioner, U.S. Food and Drug Administration (FDA)
  • Dr Eswara Reddy, Drug Controller General of India (DCGI), Central Drug Control Organization

Moderator: Sanat Chattopadhyay, President, Merck Manufacturing Division; Merck & Co.

9:40 AM – 10:00 AM Presentation on CAR (chimeric antigen receptor) T-cell Therapies
Dr. Carl June, Director of Translational Research, Abramson Cancer Center University of Pennsylvania Moderator: Dr. Raju Kucherlapati, Professor of Genetics, Harvard Medical School

  • Video on child with recurrent twice of leukhimia
  • T-cell HIV Virus infect

 

10:00 AM – 10:50 AM

Panel Discussion: Oncology – The Emperor of BioPharma Development

Panelists:

  1. solid vs blood tumors
  2. T-Cells amplification microenvironment and biology
  3. PD-1 in combination therapies thousand Trials
  4. Biomarker allows to check response in conjunction with genomics data brings insights
  5. Tumors World, Biomarkers in Immuno oncology respond to PD-1 no response to other drug
  6. stratify patients
  1. Protein experimental data compound design from simulations of VIRTUAL compounds,
  2. how to incentivise to take on new innovations
  1. more that one single administration by injection
  2. response rates different even in one patient let alone among patients
  3. detection gene
  4. CAR-T glioblastoma
  5. pancreatic cancer good responses in combination therapies
  6. immunr repertoire biology so complex that biomarkers are limited

Moderator: Dr. Christiana Bardon, Managing Director, MPM Capital

  • 30% patinets with complete cure

10:50 AM – 11:20 AM Networking Break11:20 AM – 12:10 PM

Panel Discussion: Future of Clinical Trials and Drug Development

Panelists:

  1. endpoints need to be redefined it effect price of drug development
  2. in Oncology – Basket and Umbrellas Trial – two stufies approval for melanoma, biomarker
  3. Is response rate is 30% va 50% and Phase 3 is negative Kertuda when worked at Merck dose ranging last phase when response dropped from 60% to 30% in the case of Study C3
  4. 30% of the cost of the study – 30% was translational
  5. CRO model appropriate oversite vs douplication of tasks
  • Dr. Bruce Chabner, Director of Clinical Research, Mass General Hospital Cancer Center
  1. Old paradigm Phase 1,2,3 – off the board now, New drugs do not need the old paradigm
  2. Phase i1 changed if genomics is involved multiple cohorts at same time
  3. FDA play amazing role
  4. patient selection is key
  5. mutations in rare disease vs mutations in cancer
  6. immunotherapy and endogenic drugs with chemo in RENAL cancer
  7. check-points – lung cancer understood money spent to find responders
  8. HOW to select which cheno therapy — no improvement today vs past
  9. 40 drugs approved by accelerated approval one came back on the market
  10. Financial burden of being in a clinical trial
  11. Foundation gives money to Institutions to reimburse patients for flights, meals, acommodation, Pharma are reluctant to participants due to potential accusation of bias id Pharma pays Patients that participate in Clinical Trials
  1. FDA recognizes approval process – systems involved AFTER approval for reimbursement and monitoring after market
  2. regulatory by countires are different
  3. which factors are sacrifiable in the long tern in clinical trial design
  1. Safety – benefit risk is what physicians work with every day
  2. Drugs paradign of small molecules does not hold is you have a drug that deliver entire organelle – how you dose for half life how you prive the rate of replication in the body
  3. Surrogate markers
  4. Taking a drug off the market ->>  conditional approvals [approval can be taken back or require additional studies] not a favorable view of Pharma in the present to support Conditional approval vs accelerated approval

 

  1. speed
  2. differentiation from competition
  3. drug development in crisis is CVD not cancer, US and the rest of the world – lowest investment in drugs is CVD
  4. Studies designed by Physicians using SAME design
  5. need to create experts to use ML in the course of clinical trial design
  6. regulators as Partners not as Barriers
  7. Proof of efficacy is a burden on the developers of the drug not on the Regulatory
  8. Increase use of advertising to recruit
  9. 70% OF PATIENTS WILLING TO PARTICIPATE  lives to far from site of trials
  10. Telecommunication between administrators of study ans clinical Trials participants
  11. Back when I was at Pfizer, designing study – patients burden relieved more willingness to participate
  12. Preferrs to run studies in house vs use CRO they are not effective in monitoring like study run in house

Moderator: Dr. William Chin, Professor of Medicine, Emeritus, Harvard Medical School

  • Probability of success to clinic has not changed
  • challenge is design and execution in clinical trials
  • changes in drug modalities: RNA, DNA,
  • which combination to use
  • how to find the many patients needed
  • Basket and Umbrellas Trial

12:10 PM – 1:00 PM

Panel Discussion: Manufacturing in the Future

Panelists:

  • Hari Bhartia, Founder and Co-Chairman, Jubilant Bhartia Group
  1. supply change
  2. blockchain
  3. quality by design
  4. CPK
  5. productivity will go up variability will decrease
  6. manufacturng must happen in India
  7. Genetics price selection
  8. Secure system, data quality the data logic and the analytics
  9. infrastructure in manufacturing is not completed yet
  10. Training by augmented reality Turnover high in India
  11. cyber security – digitization and central control
  12. demonstration data offense
  • Mark Abdoo, Acting Deputy Commissioner, U.S. Food and Drug Administration
  1. next 10 years India and China will improve regulatory activities and match better the US requirements
  2. review foreign hosts
  3. skills and location of hosts:
  4. India: Standards and unannounced inspections and
  5. China: same
  6. Blockchain is experienced as experimentation at FDA across each all parts of the Agency
  • Dr. Paul McKenzie, Executive Vice President, Pharma Operations & Technology, Biogen
  1. raw material to patients: Pharma very slow than other industries Reliable needs be very high, relationships
  2. Hurrican in PortoRIco affected supply chain
  3. Reality, every one HAVE to be in China
  4. Platforming for each modality for Scaling out vs Scaling up
  5. diversify vs modality x
  6. build capacity and capabilities customization of ultra filtration different in two plants lowers standardizations
  7. Training on Demand, Virtually, documnetation needs to change to electronic
  8. Continueous manufacturing Academic contribution
  • Vinay Ranade, Chief Executive Officer, Reliance Life Sciences
  1. Pharma was slow in India the manufacturing
  2. infantile diarreha vaccine 70,000 in 4 years needs that drug,
  3. massive intellectual capital in India
  4. How to implement and make best use of data to improve processes
  5. cyber security was not experiences
  1. Phase 1 scaling out vs up – it is different in vaccine field
  2. ML, Block chain, supply chain and manufacturing will be adapted in supply chain
  3. Apply analytics and relationships in manufacturing
  4. obsolescence and upgrades
  5. capture data electronically
  6. cyber security can be a hazard hard to mitigate when all systems are down
  7. significant challenges in manufacturing and data security

Moderator: Professor N. Venkat Venkatraman, Boston University Questrom School of Business

  • How can Pharma become leaner
  • heterogenuious environment for production
  • cyber security

1:00 PM – 1:50 PMLunch1:50 PM – 1:55 PM Video message from Suresh Prabhu, Hon’ble Minister of Commerce & Industry, Gov. of India1:55 PM – 2:45 PM

Panel Discussion: One in a million – Emerging trends in Rare Diseases

Panelists:

  1. worked with Academic community on how to treat rare disease in the future
  1. Show clinical benefit and impact multiplemyeloma
  2. patients becoming activists
  3. access
  4. foundation by patients
  5. Patient to get cloud
  • Dr. Dhaval Patel, Executive Vice President  and Chief Scientific Officer, UCB
  1. if a modality will cure a disease justify innovation Model for payment: Mortgage Model
  2. Access INDEX pricing – US will benchmark the price in other parts of the world
  3. Gene therapy is not only got monognenic diseases but for
  4. decrease work involved in development of drugs
  • Dr. James Wilson, Director – Gene Therapy Program, University of Pennsylvania
  1. tension between physicians and development of the perfect drug.
  2. AV
  3. Protein replacement therapy repeated infusion gene therapy infrastructure develop in China for China, Develop in India for India vs develop in US for India or China
  4. Cost of manufacturing to decrease
  • Dr. Timothy Yu, Assistant Professor in Pediatrics, Harvard Medical School
  1. Scalability beyond the one case: the mechanism for the drug has generability for other aptients iwth same mutation the method has no limit
  2. Molecular type of mutation Spice Switching strategy, just-in-time manufacturing

Moderator: Dr. Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics

  1. Rare diseases, potential for cure
  2. Academia, Hospitals, biotech
  3. commercial model of the disease

2:45 PM – 3:20 PMNetworking & Tea Break3:20 PM – 3:50 PM

Fireside Chat: Value and Access – The ongoing debate

  1. since 2003 testify in the House, against Canadian  David Brenner was asked about importation from Canada of breast cancer tamoxiphen at a lower price than in the US.
  2. From importation crisis to Obama Care – stable system Medicare Part D – drug coverage for Olderly
  3. After Obama – Price is part of doing business REBATES $100Billion the valur of REBATES
  4. Co-Insurance
  1. right for innovation will be preserved
  2. price increase
  3. give and take
  4. Co-pay – We need lower co-pay
  5. with current administration, sink finding the Well instead of Well funding the sick
  6. CHange is coming, co-pay will change
  1. Genzyme days vs 2019
  2. changes how drugs are priced?
  3. Flaws of the system:Gevernment induce prices that will change
  4. $800,000 drug is now $80 [ala Regeneron] – R&D was $2Billion
  5. CO-pay for hospital stay is lower than co-pay on drugs – 10% twice a year

Moderator: Dr Andrew Plump, President R&D, Takeda Pharmaceuticals

3:50 PM – 4:10 PM

India update on Clinical Trial Regulations

  • Arun Singhal, Additional Secretary, Ministry of Health & Family Welfare, India
  1. Each patient deserve access to healthcare in India
  2. experimenting
  • Dr Eswara Reddy, Drug Controller General of India, Central Drug Control Organization
  1. Time line for Application approval for drugs, if approved in another country 60 days
  2. Gov’t hospitals can import New drugs which have not been permitted in India

4:10 PM – 5:00 PM

Panel Discussion: Research and Development Strategies and Trends

Panelists:

  1. Neuroscience – Pharma understand biomarkers and now genetics
  2. Vaccines – across species in the animal WORLD
  1. Attempt not to tweak the PIPELINE: CVD, NEUROSCIENCE AND CANCER
  2. 485 Teams doing R&D convluence of interests to develop cure
  3. Modularity – BioMolecule — multimodality biophysical biochemical protein degradation – rewire disease cells with biomolecules combing propertitie of permiability of small molecules
  4. PHARMACOLOGICAL PREVENTION – biotech is inspiring only Pharma can solve
  1. immunooncology – mutation signature – marker protein signature — that group of diseases respond to
  2. colon cancer and multiple myeloma — understanding of the biology was deep

Moderator: Dr. Martin Mackay, Co-Founder, Rallybio

5:00 PM – 5:05 PM Closing Remarks

5:05 PM – 6:15 PM Cocktails & Networking Reception

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Cholesterol Lowering Novel PCSK9 drugs: Praluent [Sanofi and Regeneron] vs Repatha [Amgen] – which drug cuts CV risks enough to make it cost-effective?

Posted in Atherogenic Processes & Pathology, Epigenetics and Cardiovascular Risks, Fatty acids, Frontiers in Cardiology and Cardiovascular Disorders, inflammation independent of lipid levels, Lipid metabolism, Lipidomics, Lipids, Origins of Cardiovascular Disease, PCSK9 Inhibitor Therapy, Pharmaceutical R&D Informatics, Pharmacogenomics, Pharmacotherapy of Cardiovascular Disease, Systemic Inflammatory Diseases as CVD Risk, Value-based Drug Pricing on March 12, 2018| Leave a Comment »


 Cholesterol Lowering Novel PCSK9 drugs: Praluent [Sanofi and Regeneron] vs Repatha [Amgen] – which drug cuts CV risks enough to make it cost-effective?

Reporter: Aviva Lev-Ari, PhD, RN

 

UPDATED on 1/15/2019

In the patent fight over PCSK9 inhibitors, the Supreme Court refused to hear Amgen’s appeal of a 2017 court decision allowing Sanofi and Regeneron to continue selling alirocumab (Praluent). Amgen still has a new patent trial starting in Delaware federal court next month, FiercePharma reports.

Amgen’s Repatha hits wall at SCOTUS but presses ahead—new price breaks included

by Arlene Weintraub |

Amgen has been trying since 2015 to protect its PCSK9 cholesterol drug Repatha by keeping Sanofi and Regeneron’s rival Praluent off the market, even going as far as to ask the U.S. Supreme Court to review an ongoing patent fight.

But that attempt fell short this week as SCOTUS refused to hear the company’s appeal of a 2017 court decision allowing Sanofi and Regeneron to continue selling its head-to-head rival.

Amgen isn’t giving up the fight, though. The company is prepping for a new patent trial starting in Delaware federal court next month. And it’s responding to long-standing criticism of the high cost of PCSK9 drugs, which hit the market in 2015 at list prices of about $14,000 a year.

Amgen had already brought the price of the biweekly version of Repatha down to $5,850 per year before discounts and rebates, and late Monday it said it would lower cost of the monthly injectable dose to that same level.

SOURCE

UPDATED on 11/13/2018

ODYSSEY OUTCOMES: Alirocumab Cost-effective at $6000 a Year

Marlene Busko

November 11, 2018

CHICAGO — Treatment with the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor alirocumab (Praluent, Sanofi/Regeneron) is cost-effective at $6319 a year when the willingness-to-pay threshold is the generally accepted $100,000 per quality-adjusted life-year (QALY), new research reports.

Deepak L. Bhatt, MD, MPH, Brigham and Women’s Hospital Heart and Vascular Center, Harvard Medical School, Boston, Massachusetts, presented these cost-effectiveness findings for alirocumab, based on data from the ODYSSEY OUTCOMES trial, here at the American Heart Association (AHA) 2018 Scientific Sessions

As previously reported, results from ODYSSEY OUTCOMES were presented at American College of Cardiology (ACC) 2018 Annual Scientific Session in March and the study was published November 7 in the New England Journal of Medicine.

Strengths of the current cost analysis include that it used actual trial data as opposed to modeling estimates, Bhatt pointed out to theheart.org | Medscape Cardiology.

SOURCE

https://www.medscape.com/viewarticle/904744?nlid=126063_3866&src=WNL_mdplsfeat_181113_mscpedit_card&uac=93761AJ&spon=2&impID=1799507&faf=1

 

Did Amgen’s Repatha cut CV risks enough to make it cost-effective? Analysts say no

Sanofi, Regeneron’s Praluent pulls off PCSK9 coup with 29% cut to death risks in most vulnerable patients
SEE our curations on PCSK9 drugs:

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CHI’s Discovery on Target, Sheraton Boston, Sept. 25-28, 2018

Posted in Conference Coverage with Social Media, Drug Delivery Platform Technology, Drug Development Process, Drug Discovery Chemistry, Personalized and Precision Medicine & Genomic Research, Pharmaceutical Analytics, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, Pharmacodynamics and Pharmacokinetics, Pharmacogenomics, Pharmacologic toxicities on February 21, 2018| Leave a Comment »


CHI’s Discovery on Target, Sheraton Boston, Sept. 25-28, 2018

Reporter: Aviva Lev-Ari, PhD, RN

 

ANNOUNCEMENT

Leaders in Pharmaceutical Business Intelligence (LPBI) Group is a selected CHI Business Partner for Media Communication for this event as well a provider of REAL TIME PRESS COVERAGE for this cardinal event in the domain of  Drug Discovery and Drug Delivery.

Dr. Aviva Lev-Ari, PhD, RN, Editor-in-Chief, PharmaceuticalIntelligence.com  will be in attendance covering this event for the Press using Social Media via 12 Channels

LOGO of LPBI Group

Follow us on ALL our Media Communication Channels:

Channels for e-Marketing of Biotech Conferences

  • Our Journal has 1,373,977  eReaders on 1/29/2018, for All Time and 7,283 Scientific Comments

http://pharmaceuticalintelligence.com

  • Aviva’s – +6,430 BIOTECH Followers on LinkedIn

http://www.linkedin.com/in/avivalevari

  • Aviva is a Member of +60 LinkedIn Groups in Biotech related fields

https://www.linkedin.com/groups/my-groups

  • LPBI Group’s FaceBook Page

http://www.facebook.com/LeadersInPharmaceuticalBusinessIntelligence

  • LPBI Group’s Twitter Account

http://twitter.com/pharma_BI

  • LPBI Group’s Company’s Page on LinkedIn

https://www.linkedin.com/company/9325543?trk=tyah&trkInfo=clickedVertical%3Acompany%2CclickedEntityId%3A9325543%2Cidx%3A1-1-1%2CtarId%3A1439226813927%2Ctas%3ALeaders%20in%20Pharmaceutica

 

 

For UPDATES on this Cardinal Conference and for REGISTRATION, go to 

http://www.discoveryontarget.com/?utm_source=partner

 

For PROGRAMS, go to 

http://www.discoveryontarget.com/programs

September 26-27

September 27-28

September 26-27

September 27-28

September 26-27

September 27-28

September 26-27

September 27-28

September 25

What is the Role of the Editor-in-Chief at PharmaceuticalIntelligence.com 

Editor-in-Chief’s Roles and Accomplishments

1        Curation Methodology Development

Leadership we provide on curation of scientific findings in the eScientific publishing for Medical Education contents.

In Section 1, the Leadership we provide on curation of scientific findings in the eScientific publishing for Medical Education contents is demonstrated by a subset of several outstanding curations with high electronic Viewer volume. Each article included presents unique content contribution to Medical Clinical Education.

·       These articles are extracted from the list of all Journal articles with >1,000 eReaders, 4/28/2012 to 1/29/2018.

Article Title,         # of electronic Viewers,         Author(s) Name

Is the Warburg Effect the Cause or the Effect of Cancer: A 21st Century View?                      16,114 Larry H. Bernstein, MD, FCAP

Do Novel Anticoagulants Affect the PT/INR? The Cases of XARELTO (rivaroxaban) and PRADAXA (dabigatran) 11,606 Vivek Lal, MBBS, MD, FCIR,

Justin D. Pearlman, MD, PhD, FACC and

Aviva Lev-Ari, PhD, RN
Clinical Indications for Use of Inhaled Nitric Oxide (iNO) in the Adult Patient Market: Clinical Outcomes after Use, Therapy Demand and Cost of Care

 

  5,865 Aviva Lev-Ari, PhD, RN
Peroxisome proliferator-activated receptor (PPAR-gamma) Receptors Activation: PPARγ transrepression for Angiogenesis in Cardiovascular Disease and PPARγ transactivation for Treatment of Diabetes                  1,919 Aviva Lev-Ari, PhD, RN  

 

Bystolic’s generic Nebivolol – Positive Effect on circulating Endothelial Progenitor Cells Endogenous Augmentation  1,059 Aviva Lev-Ari, PhD, RN

 

Triple Antihypertensive Combination Therapy Significantly Lowers Blood Pressure in Hard-to-Treat Patients with Hypertension and Diabetes  1,339 Aviva Lev-Ari, PhD, RN

 

Clinical Trials Results for Endothelin System: Pathophysiological role in Chronic Heart Failure, Acute Coronary Syndromes and MI – Marker of Disease Severity or Genetic Determination?  1,472 Aviva Lev-Ari, PhD, RN
Treatment of Refractory Hypertension via Percutaneous Renal Denervation  1,085 Aviva Lev-Ari, PhD, RN

2        Content Creation and Key Opinion Leader (KOL) Recognition

2.1     Volume of Articles in the Journal and in the 16 Volume-BioMed e-Series

Select

Aviva Lev-Ari, PhD, RN 2012pharmaceutical

3,064 Articles

·       All  (5,288)

 avivalev-ari@alum.berkeley.edu Administrator 3064

2.1     Volume of Articles in the Journal and in the 16 Volume-BioMed e-Series

1.   Volume of Articles in the Journal since Journal inception on 4/28/2012:

·       Total articles by ALL authors in Journal Archive on 1/29/2018 = 5,288

·       ALL articles/posts Authored, Curated, Reported by Aviva Lev-Ari, PhD, RN = 3,064

2.   Volume of Articles in the 16 Volume-BioMed e-Series

·    Editorial & Publication of Articles in e-Books by Leaders in Pharmaceutical Business Intelligence: Contributions of Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2014/10/16/editorial-publication-of-articles-in-e-books-by-leaders-in-pharmaceutical-business-intelligence-contributions-of-aviva-lev-ari-phd-rn/

·       LPBI Group’s Founder: Biography and Bibliographies – Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/founder/

 

2.2     Digital Presence measured by eViews: Clicks on article by Author Name

Top Authors for all days ending 2018-01-29 (Summarized)

All Time

Author Name electronic Views
Aviva Lev-Ari, PhD, RN [2012pharmaceutical]

352,153

 

Our TEAM 5,934  

 
Founder 3,257
BioMed e-Series 3,140

 

Journal PharmaceuticalIntelligence.com 2,214
About 2,054
  VISION   2,803  

 


LPBI Group		             1,201

2.3     Digital KOL Parameters

Key Opinion Leader (KOL) – Aviva Lev-Ari, PhD, RN, as Evidenced by

https://pharmaceuticalintelligence.com/2016/07/21/key-opinion-leader-kol-aviva-lev-ari-phd-rn-as-evidenced-by/

 

3        Team building: Editors and Expert, Authors, Writers

Our Team

Selection of Journal’s Chief Scientific Officer (CSO) and BioMed e-Series Content Consultant (CC): Series B, C, D, E

L.H. Bernstein, MD, FCAP

Editorial & Publication of Articles in e-Books by  Leaders in Pharmaceutical Business Intelligence:  Contributions of Larry H Bernstein, MD, FCAP

https://pharmaceuticalintelligence.com/2014/10/16/editorial-publication-of-articles-in-e-books-by-leaders-in-pharmaceutical-business-intelligence-contributions-of-larry-h-bernstein-md-fcap/

4        Book Title Generation and Cover Page Design

As BioMed e-Series Editor–in-Chief, I was responsible for the following functions of product design and product launch

·       16 Title creations for e-Books

·       Designed 16 Cover Pages for a 16-Volume e-Books e-Series in BioMed

·       Designed Series A eTOCs and approved of all 16 electronic Table of Contents (eTOCs), working in tandem with all the Editors of each volume and all the Author contributors of article contents in the Journal.

·       Commissioned Articles by Authors/Curators per Author’s expertise on a daily basis

 

Below, see Volume Titles and Cover Pages:

13 LIVE results for Kindle Store: “Aviva Lev-Ari”

 

 

The VOICES of Patients, Hospitals CEOs, Health Care Providers, Caregivers and Families: Personal Experience with Critical Care and Invasive Medical Procedures … E: Patient-Centered Medicine Book 1)

Oct 16, 2017 | Kindle eBook

by Larry H. Bernstein and Aviva Lev-Ari

$0.00

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Sold by: Amazon Digital Services LLC

Cancer Therapies: Metabolic, Genomics, Interventional, Immunotherapy and Nanotechnology in Therapy Delivery (Series C Book 2)

May 13, 2017 | Kindle eBook

by Larry H. Bernstein and Demet Sag

$0.00

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Sold by: Amazon Digital Services LLC

The Immune System, Stress Signaling, Infectious Diseases and Therapeutic Implications: VOLUME 2: Infectious Diseases and Therapeutics and VOLUME 3: The … (Series D: BioMedicine & Immunology)

Sep 4, 2017 | Kindle eBook

by Larry H. Bernstein and Aviva Lev-Ari

$0.00

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$115.00$ 115 00 to buyKindle Edition

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Sold by: Amazon Digital Services LLC

Perspectives on Nitric Oxide in Disease Mechanisms (Biomed e-Books Book 1)

Jun 20, 2013 | Kindle eBook

by Margaret Baker PhD and Tilda Barliya PhD

$0.00

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5 out of 5 stars 6

Sold by: Amazon Digital Services LLC

Medical Scientific Discoveries for the 21st Century & Interviews with Scientific Leaders (Series E)

Dec 9, 2017 | Kindle eBook

by Larry H. Bernstein and Aviva Lev-Ari

$0.00

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Sold by: Amazon Digital Services LLC

Etiologies of Cardiovascular Diseases: Epigenetics, Genetics and Genomics

Nov 28, 2015 | Kindle eBook

by Justin D. Pearlman MD ME PhD MA FACC and Stephen J. Williams PhD

$0.00

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Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation: The Art of Scientific & Medical Curation

Nov 29, 2015 | Kindle eBook

by Larry H. Bernstein MD FCAP and Aviva Lev-Ari PhD RN

$0.00

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Read for Free

$75.00$ 75 00 to buyKindle Edition

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Sold by: Amazon Digital Services LLC

Medical 3D BioPrinting – The Revolution in Medicine Technologies for Patient-centered Medicine: From R&D in Biologics to New Medical Devices (Series E: Patient-Centered Medicine Book 4)

Dec 30, 2017 | Kindle eBook

by Larry H. Bernstein and Irina Robu

$0.00

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Sold by: Amazon Digital Services LLC

Metabolic Genomics & Pharmaceutics (BioMedicine – Metabolomics, Immunology, Infectious Diseases Book 1)

Jul 21, 2015 | Kindle eBook

by Larry H. Bernstein MD FCAP and Prabodah Kandala PhD

$0.00

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5 out of 5 stars 1

Sold by: Amazon Digital Services LLC

Cancer Biology and Genomics for Disease Diagnosis (Series C: e-Books on Cancer & Oncology Book 1)

Aug 10, 2015 | Kindle eBook

by Larry H Bernstein MD FCAP and Prabodh Kumar Kandala PhD

$0.00

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Sold by: Amazon Digital Services LLC

Genomics Orientations for Personalized Medicine (Frontiers in Genomics Research Book 1)

Nov 22, 2015 | Kindle eBook

by Sudipta Saha PhD and Ritu Saxena PhD

$0.00

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Sold by: Amazon Digital Services LLC

Milestones in Physiology: Discoveries in Medicine, Genomics and Therapeutics (Series E: Patient-Centered Medicine Book 3)

Dec 26, 2015 | Kindle eBook

by Larry H. Bernstein MD FACP and Aviva Lev-Ari PhD RN

$0.00

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Read for Free

$75.00$ 75 00 to buyKindle Edition

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Sold by: Amazon Digital Services LLC

Regenerative and Translational Medicine: The Therapeutic Promise for Cardiovascular Diseases

Dec 26, 2015 | Kindle eBook

by Justin D. Pearlman MD ME PhD MA FACC and Ritu Saxena PhD

$0.00

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Read for Free

$75.00$ 75 00 to buyKindle Edition

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Sold by: Amazon Digital Services LLC

5        Style Setting: Instruction manuals for Journal, Articles, Books

As BioMed e-Series Editor–in-Chief, Aviva Lev-Ari, PhD, RN was responsible for

·       All the documentation (Instruction manuals) on Style setting, and for

·       Training all team members

·       Journal Articles Format

·       Journal Comment Exchange Format

·       e-Books Production Process:

1.               Volume creation from Journal’s Article Archive,

2.               Format Translation from HTML to .mobi for Kindle devices,

3.               Proof reading process,

4.               Title release,

5.               Book electronic Upload to Amazon.com Cloud.

6.               Connection of all articles and e-Books to Social Media, Ping back generation by mentioning other related articles published in the Journal

 

Lastly, 6, below

6        Annual Workflow Management of Multiple eTOCs – Multi-year Book Publishing Scheduling Plan, 2013 – Present

 

Title Date of Publication Number of Pages
Perspectives on Nitric Oxide in Disease Mechanisms 6/21/2013 895
Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation 11/30/2015 11039 KB
Etiologies of Cardiovascular Diseases: Epigenetics, Genetics and Genomics 11/29/2015 12333 KB
Regenerative and Translational Medicine: The Therapeutics Promise for Cardiovascular Diseases 12/26/2015 11668 KB
Genomics Orientations for Personalized Medicine 11/23/2015 11724 KB
Cancer Biology & Genomics for Disease Diagnosis 8/11/2015 13744 KB
Cancer Therapies: Metabolic, Genomics, Interventional, Immunotherapy and Nanotechnology in Therapy Delivery 5/18/2017 5408 pages
Metabolic Genomics and Pharmaceutics 7/21/2015 13927 KB
The Immune System, Stress    Signaling, Infectious Diseases and Therapeutic Implications 9/4/2017 3747 pages
The VOICES of Patients, Hospitals CEOs, Health Care Providers, Caregivers and Families: Personal Experience with Critical Care and Invasive Medical Procedures 10/16/2017 826 pages
Medical Scientific Discoveries for the 21st Century & Interviews with Scientific Leaders 12/9/2017 2862 pages
Milestones in Physiology: Discoveries in Medicine, Genomics and Therapeutics 12/27/2015 11125 KB
Medical 3D BioPrinting – The Revolution in Medicine, Technologies for Patient-centered Medicine: From R&D in Biologics to New Medical Devices 12/30/2017 1005 pages
Pharmacological Agents in Treatment of Cardiovascular Disease

 

 Work-in-Progress, Expected Publishing date in 2018 ???
Interventional Cardiology and Cardiac Surgery for Disease Diagnosis and Guidance of Treatment Work-in-Progress, Expected Publishing date in 2018

 

 ???

 

Read Full Post »

International Award for Human Genome Project

Posted in Affordable Care Act, Award Nominations, BioIT: BioInformatics, BioIT: BioInformatics, NGS, Clinical & Translational, Pharmaceutical R&D Informatics, Clinical Genomics, Cancer Informatics, Biological Networks, Gene Regulation and Evolution, Biomarkers & Medical Diagnostics, Genetics & Innovations in Treatment, Genetics & Pharmaceutical, Genome Biology, Medical and Population Genetics, Personalized and Precision Medicine & Genomic Research, Pharmacogenomics, Population Health Management, Genetics & Pharmaceutical, tagged , , , , , , , on February 9, 2018| Leave a Comment »


International Award for Human Genome Project

Reporter and Curator: Dr. Sudipta Saha, Ph.D.

 

The Thai royal family awarded its annual prizes in Bangkok, Thailand, in late January 2018 in recognition of advances in public health and medicine – through the Prince Mahidol Award Foundation under the Royal Patronage. This foundation was established in 1992 to honor the late Prince Mahidol of Songkla, the Royal Father of His Majesty King Bhumibol Adulyadej of Thailand and the Royal Grandfather of the present King. Prince Mahidol is celebrated worldwide as the father of modern medicine and public health in Thailand.

 

The Human Genome Project has been awarded the 2017 Prince Mahidol Award for revolutionary advances in the field of medicine. The Human Genome Project was completed in 2003. It was an international, collaborative research program aimed at the complete mapping and sequencing of the human genome. Its final goal was to provide researchers with fundamental information about the human genome and powerful tools for understanding the genetic factors in human disease, paving the way for new strategies for disease diagnosis, treatment and prevention.

 

The resulting human genome sequence has provided a foundation on which researchers and clinicians now tackle increasingly complex problems, transforming the study of human biology and disease. Particularly it is satisfying that it has given the researchers the ability to begin using genomics to improve approaches for diagnosing and treating human disease thereby beginning the era of genomic medicine.

 

National Human Genome Research Institute (NHGRI) is devoted to advancing health through genome research. The institute led National Institutes of Health’s (NIH’s) contribution to the Human Genome Project, which was successfully completed in 2003 ahead of schedule and under budget. NIH, is USA’s national medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.

 

Building on the foundation laid by the sequencing of the human genome, NHGRI’s work now encompasses a broad range of research aimed at expanding understanding of human biology and improving human health. In addition, a critical part of NHGRI’s mission continues to be the study of the ethical, legal and social implications of genome research.

 

References:

 

https://www.nih.gov/news-events/news-releases/human-genome-project-awarded-thai-2017-prince-mahidol-award-field-medicine

 

http://www.mfa.go.th/main/en/news3/6886/83875-Announcement-of-the-Prince-Mahidol-Laureates-2017.html

 

http://www.thaiembassy.org/london/en/news/7519/83884-Announcement-of-the-Prince-Mahidol-Laureates-2017.html

 

http://englishnews.thaipbs.or.th/us-human-genome-project-influenza-researchers-win-prince-mahidol-award-2017/

 

http://genomesequencing.com/the-human-genome-project-is-awarded-the-thai-2017-prince-mahidol-award-for-the-field-of-medicine-national-institutes-of-health-press-release/

 

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Novartis’ Kymriah (tisagenlecleucel), FDA approved genetically engineered immune cells, would charge $475,000 per patient, will use Programs that Payers will pay only for Responding Patients 

Posted in Drug Delivery Platform Technology, Drug Development Process, Drug Discovery Chemistry, Patient-centered Medicine, Personalized and Precision Medicine & Genomic Research, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, Pharmacogenomics, Value-based Drug Pricing on August 31, 2017| Leave a Comment »


Novartis’ Kymriah (tisagenlecleucel), FDA approved genetically engineered immune cells, would charge $475,000 per patient, will use Programs that Payers will pay only for Responding Patients

Curator: Aviva Lev-Ari, PhD, RN

 

UPDATED on 9/1/2017:

This Pioneering $475,000 Cancer Drug Comes With A Money-Back Guarantee

Novartis defends the eye-popping price of its pioneering gene therapy with arguments about its $1 billion expenditure—and novel “value-based” pricing.

https://www.fastcompany.com/40461214/how-novartis-is-defending-the-record-475000-price-of-its-pioneering-gene-therapy-cancer-drug-car-t-kymriah

 

On 8/30/2017 we wrote:

FDA has approved the world’s first CAR-T therapy, Novartis for Kymriah (tisagenlecleucel) and Gilead’s $12 billion buy of KitePharma, no approved drug and Canakinumab for Lung Cancer (may be?)

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2017/08/30/fda-has-approved-the-worlds-first-car-t-therapy-novartis-for-kymriah-tisagenlecleucel-and-gileads-12-billion-buy-of-kite-pharma-no-approved-drug-and-canakinumab-for-lung-cancer-may-be/

 

The Price for the Treatment was published on 8/31/2017, a Value-based Pricing Payment Model of a $475,000 per patient charge for the responding patients after ONE month of treatment. Novartis says it takes an average of 22 days to create the therapy, from the time a patient’s cells are removed to when they are infused back into the patient. Kymriah will initially be available at 20 U.S. hospitals within a month, Novartis says. Eventually, 32 total sites will offer the therapy. 

CAR-T gained national attention three years ago when Carl June, a researcher at the University of Pennsylvania, used to put a young girl’s acute lymphoblastic leukemia. Genetically altering the girl’s immune cells had made her deathly ill, but June had used a Roche drug, Actemra, to treat the side effects. She lived, and the results were published in The New England Journal of Medicine. Novartis bought the rights to the Penn treatment for just $20 million up front.

Pharma Buying the right to use from an Academic Institution is a known route to leap frog the R&D lengthy process of Drug discovery.

“I’ve told the team that resources are not an issue. Speed is the issue,” says Novartis’ Chief Executive Joseph Jimenez, told Forbes in a cover story about the work then.

The FDA calls this CAR-T therapy treatment, made by Novartis, the “first gene therapy” in the U.S. The therapy is designed to treat an often-lethal type of blood and bone marrow cancer that affects children and young adults. The FDA defines gene therapy as a medicine that “introduces genetic material into a person’s DNA to replace faulty or missing genetic material” to treat a disease or medical condition. This is the first such therapy to be available in the U.S., according to the FDA.

Two gene therapies for rare, inherited diseases have already been approved in Europe.

To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah.

The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency approach. The clinical review was coordinated by the FDA’s Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The FDA granted the expanded approval of Actemra to Genentech Inc.

FDA commissioner Scott Gottlieb in a statement.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” 

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm

The Protocol

A patient’s T cells are extracted and cryogenically frozen so that they can be transported to Novartis’s manufacturing center in New Jersey. There, the cells are genetically altered to have a new gene that codes for a protein—called a chimeric antigen receptor, or CAR. This protein directs the T cells to target and kill leukemia cells with a specific antigen on their surface. The genetically modified cells are then infused back into the patient.

In a clinical trial of 63 children and young adults with a type of acute lymphoblastic leukemia, 83 percent of patients that received the CAR-T therapy had their cancers go into remission within three months. At six months, 89 percent of patients who received the therapy were still living, and at 12 months, 79 percent had survived.

https://www.technologyreview.com/s/608771/the-fda-has-approved-the-first-gene-therapy-for-cancer/?utm_campaign=add_this&utm_source=email&utm_medium=post

CAR-T Therapies: Product/Molecules/MOA under Development:

  • Similar CAR-T treatments were being developed at other institutions including
  • Memorial Sloan-Kettering Cancer Center,
  • Seattle Children’s Hospital, and
  • The National Cancer Institute.
  • The Memorial and Seattle work was spun off into a startup called Juno Therapeutics, which has fallen behind. Juno Therapeutics ended a CAR-T study earlier this year after patients died from cerebral edema, or swelling in the brain.
  • The NCI work became the basis for the product being developed by Kite Pharma. Kite Pharma, which is awaiting FDA approval for its CAR-T therapy to treat a form of blood cancer in adults, was this week bought out by Gilead in a deal worth $11.9 billion.

On Cambridge Healthtech Institute’s 4th Annual Adoptive T Cell Therapy, Delivering CAR, TCR, and TIL from Research to Reality, August 29 – 30, 2017 | Sheraton Boston | Boston, MA

TUESDAY, AUGUST 29 – I covered in Real Time the talk on Juno Therapeutics: Building Better T Cell Therapies: The Power of Molecular Profiling by Mark Bonyhadi, Ph.D., Head, Research and Academic Affairs, Juno Therapeutics

https://pharmaceuticalintelligence.com/2017/08/29/live-829-chis-oncolytic-virus-immunotherapy-and-adoptive-cell-therapy-august-28-29-2017-sheraton-boston-hotel-boston-ma/

 

Precision Medicine is Costly and not a Rapid manufacturing process

All of the CAR-T products are expensive to make, and must be manufactured on an individual basis for each new patient from the patient’s own T-cells, a type of white blood cells, a process that takes weeks.

  • How quickly companies can speed up manufacturing.
  • Kymriah will be manufactured at a facility in Morris Plains, N.J.
  • CAR-T technology, which has so far been used only in patients with blood cancers that have not been cured by other treatments, can be used earlier in the disease or for solid tumors: Breast, Prostate, Melanomas.

https://www.forbes.com/sites/matthewherper/2017/08/30/fda-approves-novartis-treatment-that-alters-patients-cells-to-fight-cancer/#2aecb25b4400

Prediction How Patients will Far Well – Researchers use a big-data approach to find links between different genes and patient survival.

https://www.technologyreview.com/s/608666/a-cancer-atlas-to-predict-how-patients-will-fare/?set=

A pathology atlas of the human cancer transcriptome

+ See all authors and affiliations

Science  18 Aug 2017:
Vol. 357, Issue 6352, eaan2507
DOI: 10.1126/science.aan2507

Modeling the cancer transcriptome

Recent initiatives such as The Cancer Genome Atlas have mapped the genome-wide effect of individual genes on tumor growth. By unraveling genomic alterations in tumors, molecular subtypes of cancers have been identified, which is improving patient diagnostics and treatment. Uhlen et al. developed a computer-based modeling approach to examine different cancer types in nearly 8000 patients. They provide an open-access resource for exploring how the expression of specific genes influences patient survival in 17 different types of cancer. More than 900,000 patient survival profiles are available, including for tumors of colon, prostate, lung, and breast origin. This interactive data set can also be used to generate personalized patient models to predict how metabolic changes can influence tumor growth.

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