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NCCN Shares Latest Expert Recommendations for Prostate Cancer in Spanish and Portuguese
Reporter: Stephen J. Williams, Ph.D.
Currently many biomedical texts and US government agency guidelines are only offered in English or only offered in different languages upon request. However Spanish is spoken in a majority of countries worldwide and medical text in that language would serve as an under-served need. In addition, Portuguese is the main language in the largest country in South America, Brazil.
The LPBI Group and others have noticed this need for medical translation to other languages. Currently LPBI Group is translating their medical e-book offerings into Spanish (for more details see https://pharmaceuticalintelligence.com/vision/)
Below is an article on The National Comprehensive Cancer Network’s decision to offer their cancer treatment guidelines in Spanish and Portuguese.
PLYMOUTH MEETING, PA [8 September, 2021] — The National Comprehensive Cancer Network® (NCCN®)—a nonprofit alliance of leading cancer centers in the United States—announces recently-updated versions of evidence- and expert consensus-based guidelines for treating prostate cancer, translated into Spanish and Portuguese. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) feature frequently updated cancer treatment recommendations from multidisciplinary panels of experts across NCCN Member Institutions. Independent studies have repeatedly found that following these recommendations correlates with better outcomes and longer survival.
“Everyone with prostate cancer should have access to care that is based on current and reliable evidence,” said Robert W. Carlson, MD, Chief Executive Officer, NCCN. “These updated translations—along with all of our other translated and adapted resources—help us to define and advance high-quality, high-value, patient-centered cancer care globally, so patients everywhere can live better lives.”
Prostate cancer is the second most commonly occurring cancer in men, impacting more than a million people worldwide every year.[1] In 2020, the NCCN Guidelines® for Prostate Cancer were downloaded more than 200,000 times by people outside of the United States. Approximately 47 percent of registered users for NCCN.org are located outside the U.S., with Brazil, Spain, and Mexico among the top ten countries represented.
“NCCN Guidelines are incredibly helpful resources in the work we do to ensure cancer care across Latin America meets the highest standards,” said Diogo Bastos, MD, and Andrey Soares, MD, Chair and Scientific Director of the Genitourinary Group of The Latin American Cooperative Oncology Group (LACOG). The organization has worked with NCCN in the past to develop Latin American editions of the NCCN Guidelines for Breast Cancer, Colon Cancer, Non-Small Cell Lung Cancer, Prostate Cancer, Multiple Myeloma, and Rectal Cancer, and co-hosted a webinar on “Management of Prostate Cancer for Latin America” earlier this year. “We appreciate all of NCCN’s efforts to make sure these gold-standard recommendations are accessible to non-English speakers and applicable for varying circumstances.”
NCCN also publishes NCCN Guidelines for Patients®, containing the same treatment information in non-medical terms, intended for patients and caregivers. The NCCN Guidelines for Patients: Prostate Cancer were found to be among the most trustworthy sources of information online according to a recent international study. These patient guidelines have been divided into two books, covering early and advanced prostate cancer; both have been translated into Spanish and Portuguese as well.
NCCN collaborates with organizations across the globe on resources based on the NCCN Guidelines that account for local accessibility, consideration of metabolic differences in populations, and regional regulatory variation. They can be downloaded free-of-charge for non-commercial use at NCCN.org/global or via the Virtual Library of NCCN Guidelines App. Learn more and join the conversation with the hashtag #NCCNGlobal.
[1] Bray F, Ferlay J, Soerjomataram I, Siegel RL, Torre LA, Jemal A. Global Cancer Statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin, in press. The online GLOBOCAN 2018 database is accessible at http://gco.iarc.fr/, as part of IARC’s Global Cancer Observatory.
About the National Comprehensive Cancer Network
The National Comprehensive Cancer Network® (NCCN®) is a not-for-profit alliance of leading cancer centers devoted to patient care, research, and education. NCCN is dedicated to improving and facilitating quality, effective, efficient, and accessible cancer care so patients can live better lives. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) provide transparent, evidence-based, expert consensus recommendations for cancer treatment, prevention, and supportive services; they are the recognized standard for clinical direction and policy in cancer management and the most thorough and frequently-updated clinical practice guidelines available in any area of medicine. The NCCN Guidelines for Patients® provide expert cancer treatment information to inform and empower patients and caregivers, through support from the NCCN Foundation®. NCCN also advances continuing education, global initiatives, policy, and research collaboration and publication in oncology. Visit NCCN.org for more information and follow NCCN on Facebook @NCCNorg, Instagram @NCCNorg, and Twitter @NCCN.
Please see LPBI Group’s efforts in medical text translation and Natural Language Processing of Medical Text at
As part of the all-of-America approach to fighting the COVID-19 pandemic, the U.S. Food and Drug Administration has been working with partners across the U.S. government, academia and industry to expedite the development and availability of critical medical products to treat this novel virus. Today, we are providing an update on one potential treatment called convalescent plasma and encouraging those who have recovered from COVID-19 to donate plasma to help others fight this disease.
Convalescent plasma is an antibody-rich product made from blood donated by people who have recovered from the disease caused by the virus. Prior experience with respiratory viruses and limited data that have emerged from China suggest that convalescent plasma has the potential to lessen the severity or shorten the length of illness caused by COVID-19. It is important that we evaluate this potential therapy in the context of clinical trials, through expanded access, as well as facilitate emergency access for individual patients, as appropriate.
The response to the agency’s recently announced national efforts to facilitate the development of and access to convalescent plasma has been tremendous. More than 1,040 sites and 950 physician investigators nationwide have signed on to participate in the Mayo Clinic-ledExternal Link Disclaimer expanded access protocol. A number of clinical trials are also taking place to evaluate the safety and efficacy of convalescent plasma and the FDA has granted numerous single patient emergency investigational new drug (eIND) applications as well.
FDA issues guidelines on clinical trials and obtaining emergency enrollment concerning convalescent plasma
FDA has issued guidance to provide recommendations to health care providers and investigators on the administration and study of investigational convalescent plasma collected from individuals who have recovered from COVID-19 (COVID-19 convalescent plasma) during the public health emergency.
The guidance provides recommendations on the following:
Because COVID-19 convalescent plasma has not yet been approved for use by FDA, it is regulated as an investigational product. A health care provider must participate in one of the pathways described below. FDA does not collect COVID-19 convalescent plasma or provide COVID-19 convalescent plasma. Health care providers or acute care facilities should instead obtain COVID-19 convalescent plasma from an FDA-registered blood establishment.
Excerpts from the guidance document are provided below.
Background
The Food and Drug Administration (FDA or Agency) plays a critical role in protecting the United States (U.S.) from threats including emerging infectious diseases, such as the Coronavirus Disease 2019 (COVID-19) pandemic. FDA is committed to providing timely guidance to support response efforts to this pandemic.
One investigational treatment being explored for COVID-19 is the use of convalescent plasma collected from individuals who have recovered from COVID-19. Convalescent plasma that contains antibodies to severe acute respiratory syndrome coronavirus 2 or SARS-CoV-2 (the virus that causes COVID-19) is being studied for administration to patients with COVID-19. Use of convalescent plasma has been studied in outbreaks of other respiratory infections, including the 2003 SARS-CoV-1 epidemic, the 2009-2010 H1N1 influenza virus pandemic, and the 2012 MERS-CoV epidemic.
Although promising, convalescent plasma has not yet been shown to be safe and effective as a treatment for COVID-19. Therefore, it is important to study the safety and efficacy of COVID-19 convalescent plasma in clinical trials.
Pathways for Use of Investigational COVID-19 Convalescent Plasma
The following pathways are available for administering or studying the use of COVID-19 convalescent plasma:
Clinical Trials
Investigators wishing to study the use of convalescent plasma in a clinical trial should submit requests to FDA for investigational use under the traditional IND regulatory pathway (21 CFR Part 312). CBER’s Office of Blood Research and Review is committed to engaging with sponsors and reviewing such requests expeditiously. During the COVID-19 pandemic, INDs may be submitted via email to CBERDCC_eMailSub@fda.hhs.gov.
Expanded Access
An IND application for expanded access is an alternative for use of COVID-19 convalescent plasma for patients with serious or immediately life-threatening COVID-19 disease who are not eligible or who are unable to participate in randomized clinical trials (21 CFR 312.305). FDA has worked with multiple federal partners and academia to open an expanded access protocol to facilitate access to COVID-19 convalescent plasma across the nation. Access to this investigational product may be available through participation of acute care facilities in an investigational expanded access protocol under an IND that is already in place.
Although participation in clinical trials or an expanded access program are ways for patients to obtain access to convalescent plasma, for various reasons these may not be readily available to all patients in potential need. Therefore, given the public health emergency that the COVID-19 pandemic presents, and while clinical trials are being conducted and a national expanded access protocol is available, FDA also is facilitating access to COVID-19 convalescent plasma for use in patients with serious or immediately life-threatening COVID-19 infections through the process of the patient’s physician requesting a single patient emergency IND (eIND) for the individual patient under 21 CFR 312.310. This process allows the use of an investigational drug for the treatment of an individual patient by a licensed physician upon FDA authorization, if the applicable regulatory criteria are met. Note, in such case, a licensed physician seeking to administer COVID-19 convalescent plasma to an individual patient must request the eIND (see 21 CFR 312.310(b)).
Today, the U.S. Food and Drug Administration issued an emergency use authorization (EUA) for investigational convalescent plasma for the treatment of COVID-19 in hospitalized patients as part of the agency’s ongoing efforts to fight COVID-19. Based on scientific evidence available, the FDA concluded, as outlined in its decision memorandum, this product may be effective in treating COVID-19 and that the known and potential benefits of the product outweigh the known and potential risks of the product.
Today’s action follows the FDA’s extensive review of the science and data generated over the past several months stemming from efforts to facilitate emergency access to convalescent plasma for patients as clinical trials to definitively demonstrate safety and efficacy remain ongoing.
The EUA authorizes the distribution of COVID-19 convalescent plasma in the U.S. and its administration by health care providers, as appropriate, to treat suspected or laboratory-confirmed COVID-19 in hospitalized patients with COVID-19.
Alex Azar, Health and Human Services Secretary:
“The FDA’s emergency authorization for convalescent plasma is a milestone achievement in President Trump’s efforts to save lives from COVID-19,” said Secretary Azar. “The Trump Administration recognized the potential of convalescent plasma early on. Months ago, the FDA, BARDA, and private partners began work on making this product available across the country while continuing to evaluate data through clinical trials. Our work on convalescent plasma has delivered broader access to the product than is available in any other country and reached more than 70,000 American patients so far. We are deeply grateful to Americans who have already donated and encourage individuals who have recovered from COVID-19 to consider donating convalescent plasma.”
Stephen M. Hahn, M.D., FDA Commissioner:
“I am committed to releasing safe and potentially helpful treatments for COVID-19 as quickly as possible in order to save lives. We’re encouraged by the early promising data that we’ve seen about convalescent plasma. The data from studies conducted this year shows that plasma from patients who’ve recovered from COVID-19 has the potential to help treat those who are suffering from the effects of getting this terrible virus,” said Dr. Hahn. “At the same time, we will continue to work with researchers to continue randomized clinical trials to study the safety and effectiveness of convalescent plasma in treating patients infected with the novel coronavirus.”
Scientific Evidence on Convalescent Plasma
Based on an evaluation of the EUA criteria and the totality of the available scientific evidence, the FDA’s Center for Biologics Evaluation and Research determined that the statutory criteria for issuing an EUA criteria were met.
The FDA determined that it is reasonable to believe that COVID-19 convalescent plasma may be effective in lessening the severity or shortening the length of COVID-19 illness in some hospitalized patients. The agency also determined that the known and potential benefits of the product, when used to treat COVID-19, outweigh the known and potential risks of the product and that that there are no adequate, approved, and available alternative treatments.
CLINICAL MEMORANDUM From: , OBRR/DBCD/CRS To: , OBRR Through: , OBRR/DBCD , OBRR/DBCD , OBRR/DBCD/CRS Re: EUA 26382: Emergency Use Authorization (EUA) Request (original request 8/12/20; amended request 8/23/20) Product: COVID-19 Convalescent Plasma Items reviewed: EUA request Fact Sheet for Health Care Providers Fact Sheet for Recipients Sponsor: Robert Kadlec, M.D. Assistant Secretary for Preparedness and Response (ASPR) Office of Assistant Secretary for Preparedness and Response (ASPR) U.S. Department of Health and Human Services (HHS) EXECUTIVE SUMMARY COVID-19 Convalescent Plasma (CCP), an unapproved biological product, is proposed for use under an Emergency Use Authorization (EUA) under section 564 of the Federal Food, Drug, and Cosmetic Act (the Act),(21 USC 360bbb-3) as a passive immune therapy for the treatment of hospitalized patients with COVID-19, a serious or life-threatening disease. There currently is no adequate, approved, and available alternative to CCP for treating COVID-19. The sponsor has pointed to four lines of evidence to support that CCP may be effective in the treatment of hospitalized patients with COVID-19: 1) History of convalescent plasma for respiratory coronaviruses; 2) Evidence of preclinical safety and efficacy in animal models; 3) Published studies of the safety and efficacy of CCP; and 4) Data on safety and efficacy from the National Expanded Access Treatment Protocol (EAP) sponsored by the Mayo Clinic. Considering the totality of the scientific evidence presented in the EUA, I conclude that current data for the use of CCP in adult hospitalized patients with COVID-19 supports the conclusion that CCP meets the “may be effective” criterion for issuance of an EUA from section 564(c)(2)(A) of the Act. It is reasonable to conclude that the known and potential benefits of CCP outweigh the known and potential risks of CCP for the proposed EUA. Current data suggest the largest clinical benefit is associated with high-titer units of CCP administered early course of the disease.
A letter, from Senator Warren, to Commissioner Hahn from Senate Committee asking for documentation for any communication between FDA and White House
August 25, 2020 Dr. Stephen M. Hahn, M.D. Commissioner of Food and Drugs U.S. Food and Drug Administration 10903 New Hampshire Avenue Silver Spring, MD 20993 Dear Commissioner Hahn: We write regarding the U.S. Food and Drug Administration’s (FDA) troubling decision earlier this week to issue an Emergency Use Authorization (EUA) for convalescent plasma as a treatment for coronavirus disease 2019 (COVID-19).1 Reports suggests that the FDA granted the EUA amid intense political pressure from President Trump and other Administration officials, despite limited evidence of convalescent plasma’s effectiveness as a COVID-19 treatment.2 To help us better understand whether the issuance of the blood plasma EUA was motivated by politics, we request copies of any and all communications between FDA and White House officials regarding the blood plasma EUA.
The authorization will allow health-care providers in the U.S. to use the plasma to treat hospitalized patients with Covid-19.
The FDA’s emergency use authorization came a day after President Trump accused the agency of delaying enrollment in clinical trials for vaccines or therapeutics.
The criticism from Trump and action from the FDA led some scientists to believe the authorization, which came on the eve of the GOP national convention, was politically motivated.
FDA Commissioner Dr. Stephen Hahn is walking back comments on the benefits of convalescent plasma, saying he could have done a better job of explaining the data on its effectiveness against the coronavirus after authorizing it for emergency use over the weekend.
In an interview with Bloomberg’s Drew Armstrong, FDA Commissioner Hahn reiterates that his decision was based on hard evidence and scientific fact, not political pressure. The whole interview is at the link below:
Dr. Hahn corrected his initial statement about 35% of people would be cured by convalescent plasma. In the interview he stated:
I was trying to do what I do with patients, because patients often understand things in absolute terms versus relative terms. And I should’ve been more careful, there’s no question about it. What I was trying to get to is that if you look at a hundred patients who receive high titre, and a hundred patients who received low titre, the difference between those two particular subset of patients who had these specific criteria was a 35% reduction in mortality. So I frankly did not do a good job of explaining that.
FDA colleagues had frank discussion after the statement was made. He is not asking for other people in HHS to retract their statements, only is concerned that FDA has correct information for physicians and patients
Hahn is worried that people will not enroll due to chance they may be given placebo
He gave no opinion when asked if FDA should be an independent agency
For more articles on COVID19 please go to our Coronavirus Portal at
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
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.
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
Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) portal. National Institutes of Health. Accessed May 15, 2020. https://www.nih.gov/ACTIV
Anybody can do something once; the problem is: can you do it twice, or for that matter, over and over again?
This is the essential issue faced by those personnel in the throes of the commercialization process.
Any successful commercial process has to meet a number of criteria:
The process must be reproducible — it must yield the same product/results given the same inputs.
The process must be economically viable: given the constraints of raw material, energy, and labor costs, depreciation schedules for equipment, expected process failures, R/D, Marketing, and Sales support costs, the process needs to yield both a profit and positive cash flow
The process should be implemented using readily available commercial components and control instrumentation. On occasion, successful implementation of a project will require specialized components; however these components themselves must meet the criteria for successful commercialization
The process must be “simple” enough so that suitably trained operators can manage the process. A unit that requires Ph.D.’s to maintain operations is doomed to failure
History is replete with novel processes that worked on the lab scale, but were failures when a commercial operation was attempted. The issues that are most responsible for lab-to-production failure are listed under the general classification of “scale-up”. Scale-up principles are covered in my monograph, “The Art of Scale-up” (www.artofscaleup.com), but in general follow these rules:
Identification of those process parameters that will have major impact on commercial viability: reaction kinetics, mass transfer vs. temperature/kinetic control; if multi-phase systems are involved, the type and energy of required stirring; heat transfer considerations; side reactions; etc.
Materials of construction; raw material and product hazards; etc.
Regulatory considerations: FDA, OSHA, EPA.
Failure to address any of these issues prior to commercialization will lead to surprises during commercialization.
In addition to the engineering/scale-up aspects of commercialization, there are several other criteria that may need attention:
When to launch a product – where will the new product fit into the overall corporate product portfolio?
Where is the proper location to launch? A product aimed at flu symptom suppression in cold-weather conditions may not do well in Florida; ….. super-sweet tea does well in the South, and not so well in New England, so that a product to replace sugar might do well in the South.
Who is going to use the product? Are you targeting doctor’s offices, hospitals, or direct to consumer routes?
How to launch – social media and “influencers” have given rise to new avenues of product introductions.
The old aphorism of “measure twice, cut once” has a special resonance when doing commercialization of a new process or product. The more the process is thought out ahead of time, the less issues there will be down the road. In the commercial world, there is constant pressure to rush things to meet management deadlines, which always leads to problems and extra expense. A crusty of R/D chemist once remarked, “There is never time to do it right, but always time to do it twice.” Everyone needs to keep this in the back of their mind
Presentation on CAR (chimeric antigen receptor) T-cell Therapies Dr. Carl June, Director of Translational Research, Abramson Cancer Center University of PennsylvaniaModerator:Dr. Raju Kucherlapati, Professor of Genetics, Harvard Medical School
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 PennsylvaniaModerator:Dr. Raju Kucherlapati, Professor of Genetics, Harvard Medical School
Dr. Alise Reicin, President, Global Clinical Development, Celgene
endpoints need to be redefined it effect price of drug development
in Oncology – Basket and Umbrellas Trial – two stufies approval for melanoma, biomarker
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
30% of the cost of the study – 30% was translational
CRO model appropriate oversite vs douplication of tasks
Dr. Bruce Chabner, Director of Clinical Research, Mass General Hospital Cancer Center
Old paradigm Phase 1,2,3 – off the board now, New drugs do not need the old paradigm
Phase i1 changed if genomics is involved multiple cohorts at same time
FDA play amazing role
patient selection is key
mutations in rare disease vs mutations in cancer
immunotherapy and endogenic drugs with chemo in RENAL cancer
check-points – lung cancer understood money spent to find responders
HOW to select which cheno therapy — no improvement today vs past
40 drugs approved by accelerated approval one came back on the market
Financial burden of being in a clinical trial
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
Safety – benefit risk is what physicians work with every day
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
Surrogate markers
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
Dr. Daniel Curran, Head of the Rare Diseases Therapeutic Area Unit, Takeda
worked with Academic community on how to treat rare disease in the future
David Lucchino, Co-Founder and CEO, Frequency Therapeutics
Show clinical benefit and impact multiplemyeloma
patients becoming activists
access
foundation by patients
Patient to get cloud
Dr. Dhaval Patel, Executive Vice President and Chief Scientific Officer, UCB
if a modality will cure a disease justify innovation Model for payment: Mortgage Model
Access INDEX pricing – US will benchmark the price in other parts of the world
Gene therapy is not only got monognenic diseases but for
decrease work involved in development of drugs
Dr. James Wilson, Director – Gene Therapy Program, University of Pennsylvania
tension between physicians and development of the perfect drug.
AV
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
Cost of manufacturing to decrease
Dr. Timothy Yu, Assistant Professor in Pediatrics, Harvard Medical School
Scalability beyond the one case: the mechanism for the drug has generability for other aptients iwth same mutation the method has no limit
Molecular type of mutation Spice Switching strategy, just-in-time manufacturing
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.
From importation crisis to Obama Care – stable system Medicare Part D – drug coverage for Olderly
After Obama – Price is part of doing business REBATES $100Billion the valur of REBATES
Neuroscience – Pharma understand biomarkers and now genetics
Vaccines – across species in the animal WORLD
Dr. James Bradner, President, Novartis Institutes for BioMedical Research
Attempt not to tweak the PIPELINE: CVD, NEUROSCIENCE AND CANCER
485 Teams doing R&D convluence of interests to develop cure
Modularity – BioMolecule — multimodality biophysical biochemical protein degradation – rewire disease cells with biomolecules combing propertitie of permiability of small molecules
PHARMACOLOGICAL PREVENTION – biotech is inspiring only Pharma can solve
Dr. Mathai Mammen, Global Head of R&D, Janssen Pharmaceutical Companies of J&J
immunooncology – mutation signature – marker protein signature — that group of diseases respond to
colon cancer and multiple myeloma — understanding of the biology was deep
Published Date: December 13, 2016 Author: Global Market Insights, Inc.
Pharmacovigilance Marketsize is expected to exceed USD 8 billion by 2024; according to a new research report by Global Market Insights, Inc.
Growing number of adverse drug reactions (ADRs) coupled with increasing prevalence of chronic diseases will drive global pharmacovigilance market size. Furthermore, growing geriatric population base is associated with increased drug consumption for treatment of chronic diseases such as diabetes, oncology cardiovascular and respiratory disorders.
Rising demand for drugs has driven the need for new drug development through clinical trials. Pharmaceutical companies are collaborating with CROs to streamline R&D, medical writing, manufacturing operations, clinical data management and other pharmacovigilance activities to achieve greater efficiency at reduced cost. Outsourcing should enable better regulatory compliance, higher productivity and improved strategic outcomes spurring pharmacovigilance market growth.
Increasing number of National pharmacovigilance centers across the globe along with rising patient awareness regarding adverse drug events will stimulate global pharmacovigilance market growth. However, lack of skilled professionals and risk associated with data security and web-based drug sales will hamper business expansion.
The phase IV clinical trial market size was valued more than USD 2 billion in 2015 and is expected to grow at over 10% by 2024. Phase IV studies ensure that restrictions could be imposed on a drug being sold depending on its safety performance.
Contract outsourcing market size was valued over USD 1 billion in 2015, poised to grow at 12.2% from 2016 to 2024 and surpass USD 4 billion by 2024. Contract outsourcing reduces overall economic losses linked with drug approval delays and trial failures. It is widely opted by companies to avoid huge investments and delegate such activities to specialized firms in this area.
U.S. pharmacovigilance market size was valued at more than USD 1 billion in 2015, with expectations to grow at 10.7% over the forecast period, owing to favorable governmental regulations, huge clinical trial volume and presence of large scale research companies. Additionally, growing patient concerns related to the drug safety and rising adverse drug events related mortality rates will positively impact pharmacovigilance market share.
Spain pharmacovigilance market size was valued over USD 230 million in 2015 and should witness 10.2% CAGR from 2016 to 2024, to surpass USD 550 million by 2024. Rising demand for new drug development, growing geriatric population and increasing outsourcing by pharmaceutical companies should fuel regional industry growth. India pharmacovigilance market growth was more than 14% from 2016 to 2024, and expected to reach USD 668 million by 2024. The strong and robust growth is attributed to increasing number of clinical trials conducted across Asian countries, owing to low cost trial advantage over developed countries.
Key industry players such as Quintiles offer literature monitoring, safety aggregate reporting, benefit risk management, analytics and signal detection services. Synowledge offer signal detection services, which help clinical experts determine medical significance with the use of hi-tech visualization techniques.
Many industry participants are focusing on outsourcing pharmacovigilance services as a feasible cost reduction avenue. Outsourcing helps achieve better pharmacovigilance through regulatory compliance, better quality, enhanced productivity and improved strategic outcomes. Browse key industry insights spread across 111 pages with 66 market data tables & 6 figures& charts from the report, “Pharmacovigilance Market Size By Clinical Trial Phase (Preclinical, Phase I, Phase II, Phase III, Phase IV), By Service Provider (In-house, Contract outsourcing) Industry Analysis Report, Regional Outlook (U.S., Canada, UK, Germany, Spain, Italy, France, China, Japan, India, Australia, Argentina, Brazil, Mexico, South Africa, Saudi Arabia, UAE, Qatar), Application Potential, Price Trends, Competitive Market Share & Forecast, 2016 – 2024” in detail along with the table of contents:
Europe pharmacovigilance market size was over USD 790 million in 2015 growing at anticipated close to 10% CAGR. Germany, UK, Spain together contributed for over 70% of regional pharmacovigilance market share in 2015.
Brazil pharmacovigilance market share was more than 60% of regional revenue for 2015, with target slated to exceed USD 300 million by 2024. South Africa pharmacovigilance market size was more than USD 37 million and anticipated for over 5% growth.
Phase III clinical trial market size was more than USD 450 million in 2015, with expectations to grow over 10% CAGR, due to increasing requirement for drug safety monitoring and evaluation.
Contract outsourcing held more than 50% of pharmacovigilance market share with target market size of over USD 1.7 billion in 2015. Increasing outsourcing trend adopted by pharmaceutical companies will serve as a high impact driver for the business growth.
Global pharmacovigilance market will be driven by collaboration between pharmaceutical companies and contract research organizations (CROs). The key industry participants include Accenture, Quintiles, Cognizant Technology Solutions, Boehringer Ingelheim, Covance, PAREXEL International Corporation, Bristol-Myers Squibb, Janssen Research & Development, Synowlwedge, United BioSource Corporation and ICON.
Pharmacovigilance market research report includes in-depth industry coverage with estimates & forecast in terms of revenue in USD million from 2012 to 2024, for the following segments:
Pharmacovigilance Market By Clinical Trial
Preclinical
Phase I
Phase II
Phase III
Phase IV
Pharmacovigilance Market By End User
In-house
Contract outsourcing
The above information is provided for the following regions and countries:
2012pharmaceutical
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