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Archive for the ‘Pharmaceutical R&D Informatics’ Category


Real Time Coverage @BIOConvention #BIO2019: Precision Medicine Beyond Oncology June 5 Philadelphia PA

Reporter: Stephen J Williams PhD @StephenJWillia2

Precision Medicine has helped transform cancer care from one-size-fits-all chemotherapy to a new era, where patients’ tumors can be analyzed and therapy selected based on their genetic makeup. Until now, however, precision medicine’s impact has been far less in other therapeutic areas, many of which are ripe for transformation. Efforts are underway to bring the successes of precision medicine to neurology, immunology, ophthalmology, and other areas. This move raises key questions of how the lessons learned in oncology can be used to advance precision medicine in other fields, what types of data and tools will be important to personalizing treatment in these areas, and what sorts of partnerships and payer initiatives will be needed to support these approaches and their ultimate commercialization and use. The panel will also provide an in depth look at precision medicine approaches aimed at better understanding and improving patient care in highly complex disease areas like neurology.
Speaker panel:  The big issue now with precision medicine is there is so much data and hard to put experimental design and controls around randomly collected data.
  • The frontier is how to CURATE randomly collected data to make some sense of it
  • One speaker was at a cancer meeting and the oncologist had no idea what to make of genomic reports they were given.  Then there is a lack of action or worse a misdiagnosis.
  • So for e.g. with Artificial Intelligence algorithms to analyze image data you can see things you can’t see with naked eye but if data quality not good the algorithms are useless – if data not curated properly data is wasted
Data needs to be organized and curated. 
If relying of AI for big data analysis the big question still is: what are the rates of false negative and false positives?  Have to make sure so no misdiagnosis.

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@pharma_BI

@AVIVA1950

@BIOConvention

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#BIO2019 (official meeting hashtag)

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Real Time Coverage @BIOConvention #BIO2019:  Issues of Risk and Reproduceability in Translational and Academic Collaboration; 2:30-4:00 June 3 Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

Derisking Academic Science: The Unmet Need  

Translating academic research into products and new therapies is a very risky venture as only 1% of academic research has been successfully translated into successful products.

Speakers
Collaboration from Chicago area universities like U of Chicago, Northwestern, etc.  First phase was enhance collaboration between universities by funding faculty recruitment and basic research.  Access to core facilities across universities.  Have expanded to give alternatives to company formation.
Half of the partnerships from Harvard and companies have been able to spin out viable startups.
Most academic PI are not as savvy to start a biotech so they bring in biotechs and build project teams as well as developing a team of ex pharma and biotech experts.  Derisk as running as one asset project.  Partner as early as possible.  A third of their pipeline have been successfully partnered.  Work with investors and patent attorneys.
Focused on getting PIs to get to startup.  Focused on oncology and vaccines and I/O.  The result can be liscensing or partnership. Running around 50 to 60 projects. Creating a new company from these US PI partnerships.
Most projects from Harvard have been therapeutics-based.  At Harvard they have a network of investors ($50 million).   They screen PI proposals based on translateability and what investors are interested in.
In Chicago they solicit multiple projects but are agnostic on area but as they are limited they are focused on projects that will assist in developing a stronger proposal to investor/funding mechanism.
NYU goes around university doing due diligence reaching out to investigators. They shop around their projects to wet their investors, pharma appetite future funding.  At Takeda they have five centers around US.  They want to have more input so go into the university with their scientists and discuss ideas.
Challenges:

Takeda: Data Validation very important. Second there may be disconnect with the amount of equity the PI wants in the new company as well as management.  Third PIs not aware of all steps in drug development.

Harvard:  Pharma and biotech have robust research and academic does not have the size or scope of pharma.  PIs must be more diligent on e.g. the compounds they get from a screen… they only focus narrowly

NYU:  bring in consultants as PIs don’t understand all the management issues.  Need to understand development so they bring in the experts to help them.  Pharma he feels have to much risk aversion and none of their PIs want 100% equity.

Chicago:  they like to publish at early stage so publication freedom is a challenge

Dr. Freedman: Most scientists responding to Nature survey said yes a reproduceability crisis.  The reasons: experimental bias, lack of validation techniques, reagents, and protocols etc.
And as he says there is a great ECONOMIC IMPACT of preclinical reproducability issues: to the tune of $56 billion of irreproducable results (paper published in PLOS Biology).  If can find the core drivers of this issue they can solve the problem.  STANDARDS are constantly used in various industries however academic research are lagging in developing such standards.  Just the problem of cell line authentication is costing $4 billion.
Dr. Cousins:  There are multiple high throughput screening (HTS) academic centers around the world (150 in US).  So where does the industry go for best practices in assays?  Eli Lilly had developed a manual for HTS best practices and in 1984 made publicly available (Assay Guidance Manual).  To date there have been constant updates to this manual to incorporate new assays.  Workshops have been developed to train scientists in these best practices.
NIH has been developing new programs to address these reproducability issues.  Developed a method called
Ring Testing Initiative” where multiple centers involved in sharing reagents as well as assays and allowing scientists to test at multiple facilities.
Dr.Tong: Reproduceability of Microarrays:  As microarrays were the only methodology to do high through put genomics in the early 2000s, and although much research had been performed to standardize and achieve best reproduceability of the microarray technology (determining best practices in spotting RNA on glass slides, hybridization protocols, image analysis) little had been done on evaluating the reproducibility of results obtained from microarray experiments involving biological samples.  The advent of Artificial Intelligence and Machine Learning though can be used to help validate microarray results.  This was done in a Nature Biotechnology paper (Nature Biotechnology volume28pages827–838 (2010)) by an international consortium, the International MAQC (Microarray Quality Control) Society and can be found here
However Dr. Tong feels there is much confusion in how we define reproduceability.  Dr. Tong identified a few key points of data reproduceability:
  1. Traceability: what are the practices and procedures from going from point A to point B (steps in a protocol or experimental design)
  2. Repeatability:  ability to repeat results within the same laboratory
  3. Replicatablilty:  ability to repeat results cross laboratory
  4. Transferability:  are the results validated across multiple platforms?

The panel then discussed the role of journals and funders to drive reproduceability in research.  They felt that editors have been doing as much as they can do as they receive an end product (the paper) but all agreed funders need to do more to promote data validity, especially in requiring that systematic evaluation and validation of each step in protocols are performed..  There could be more training of PIs with respect to protocol and data validation.

Other Articles on Industry/Academic Research Partnerships and Translational Research on this Open Access Online Journal Include

Envisage-Wistar Partnership and Immunacel LLC Presents at PCCI

BIO Partnering: Intersection of Academic and Industry: BIO INTERNATIONAL CONVENTION June 23-26, 2014 | San Diego, CA

R&D Alliances between Big Pharma and Academic Research Centers: Pharma’s Realization that Internal R&D Groups alone aren’t enough

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Biotechnology & Pharma: 2018 Investment Budget in R&D – Top Ten Companies, Top R&D% AZ, Merck, BMS, Eli Lilly

Reporter: Aviva Lev-Ari, PhD, RN

 

AstraZeneca Budget 5,932,000,000 R&D 5,932,000,000 27.0%

Merck            Budget 9,750,000,000 R&D 9,750,000,000 23.0%

BMS               Budget 6,345,000,000 R&D 6,345,000,000 23.0%

Eli Lilly           Budget 5,307,100,000 R&D 5,307,100,000 22.5%

The top 10 pharma R&D budgets in 2018

Annual pharma R&D budgets
Company Measure Names SUM(Budget (copy)) SUM(Budget) SUM(R&D as percentage of revenue)
GlaxoSmithKline Budget 5,196,000,000 5,196,000,000 12.6
Eli Lilly Budget 5,307,100,000 5,307,100,000 22.5
AstraZeneca Budget 5,932,000,000 5,932,000,000 27.0
Bristol-Myers Squibb Budget 6,345,000,000 6,345,000,000 23.0
Sanofi Budget 6,961,000,000 6,961,000,000 17.1
Pfizer Budget 8,006,000,000 8,006,000,000 14.9
Novartis Budget 9,074,000,000 9,074,000,000 17.5
Merck Budget 9,750,000,000 9,750,000,000 23.0
Johnson & Johnson Budget 10,800,000,000 10,800,000,000 13.2
Roche Budget 11,060,000,000 11,060,000,000 19.3
Showing first 20 rows.
SOURCE

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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

 

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.”

 

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Tweets and Retweets by @AVIVA1950 and by @pharma_BI for @USAIC and #USAIC2019 at the 13th Annual BioPharma & Healthcare Summit, Thursday, May 9, 2019, Marriott Hotel, Cambridge, MA

 

  1.  liked Tweets you were included in

    18 hours ago

    4 other likes

  2.  liked your Tweets

    18 hours ago

    10 other likes

 

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    Alise Reicin discusses endpoints needed in and effect of cost of in Panel Discussion morning Networking break

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    Alise Reicin Panel Discussion: best done as Basket&Umbrella trial; response rate 30-50% but Phase3 negative

    Translate Tweet

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    Dr. William Chin design challenges with newer in recruiting patients; use Basket&Umbrella Trial design Live

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On risk | benefit: Long term impacts of treatment may be present for the life of the patient

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    Discussion of cyber security at panel moderated by – realization that everything is interconnected: risks to business-critical functions. Much to learn from FinTech & others’ prior experience.

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    Unexpected best thing about – at least three Zimbabweans in the crowd / on the stage.

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    segueing into a discussion of safety, and risk tolerance. Prevention has a higher safety bar than treatment later in the disease process.

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    Most humbling & touching moment: meeting an exec who nodded & teared up as I told him what we do His 9 yo child needs frequent contrast enhanced MRI scans for a rare disease. We felt like the smallest co at yet so important for hope & health

  5.  
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    Great initiatives to bring CAR-T to India an automation to drug manufacturion

    Amazing moderator asking provoking question of best panel in responses by experts made known were different for competing rivals the aim is same best faster largest at lowest keeping maximized

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    Dr. Bruce Chabner Talk: Old Phase 1,2,3 design not needed for & era based . very accommodating in Phase 1 trial design.

  8.   Retweeted

    Dr. Bruce Chabner panel discussion: selection based on new design paradigm; 40 drugs approved by accelerated approval for

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    Dr. Mace Rothenberg talks for approvals vary greatly over multiple countries makes issues of ong-term design and post approval reimbursement

  10.   Retweeted

    Talk : design now depends on systems e.g. organelle delivery. only wants accelerated approval not conditional approval. Surrogate markers critical for new trial design

  11.   Retweeted

    Dr. Rob Scott Panel Talk crisis in not in . Lowest investment in development. Physicians using SAME design for . use in trails increasing

  12.   Retweeted

    Dr. Rob Scott on design @usaic2019: Regulators as partners not barriers but burden of efficacy on . Can use advertising to increase recruitment as 70% willing participants live too far away so use &

  13.   Retweeted

    Dr. Rob Scott discusses recruitment and burdens : prefers to do in house & not use CRO as CRO less effective in monitoring trial

  14.   Retweeted

    “Some drug platforms are mature enough to fall under the practice of medicine” – Tim Yu of

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    Closing R&D strategies panel at is with NIBR’s Janssen’s and Takeda’s Andy Plump. Moderator Martin Mackay (now of newco Rallybio) asks: What are you most excited about?

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    Talk of indexed pricing model in the US may be a challenge for access to other parts of the world, says a speaker on rare disease panel

  17.   Retweeted

    The biggest problem that we have in the industry is the lack of empathy, says Chris Viehbacher

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    Tim Yu’s example left a room full of seasoned biopharma R&D execs wowed. More background here:

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    (May 9, Boston) will feature plenary panel on Emerging R&D Strategies moderated by Dr. Martin Mackay, Co-Founder, Rallybio, with of , Andy Plump of , of Janssen, Michael Ehler of -> register today

  20.   Retweeted

    Check out my latest article: Where is Oncology Drug Development Heading Next? Hear From Top KOLs at 13th BioPharma Summit May 9, Boston via

  21.   Retweeted

    Check out my latest article: One in a Million: Emerging Trends in Rare Diseases at 13th annual BioPharma Summit- May 9, Boston via

  22.   Retweeted

    Check out my latest article: Chris Viehbacher, Gurnet Point Capital joins the USAIC Advisory Board. Please join Chris & other leaders at our annual BioPharma Summit, May 9, Boston via

  23.   Retweeted

    Check out my latest article: R&D Panel: BioPharma KOLs Debate R&D Strategies & Trends at 13th annual BioPharma Summit, May 9, Boston via

  24.   Retweeted

    Check out my latest article: What Does The Future Hold For Drug Development & Clinical Trials? Hear Predictions From Top Drug Developers at the 13th BioPharma Summit May Boston 9 via

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    Value and Access – The Ongoing Debate.The BioPharma Summit will feature this special session. Join the discussion with BioPharma KOLs via

  27.   Retweeted

    BioPharma Manufacturing in the Future: Hear KOLs Debate the Challenges and Opportunities at the 13th annual BioPharma Summit, May 9, Boston via

  28.   Retweeted

    Our 13th Biopharma & Healthcare Summit has kicked off with introductory remarks from USAIC President Karun Rishi and emcee Dr. Andrew Plump, President of R&D for Takeda.

  29.   Retweeted

    The session is starting as I attend the focused on US-India bio-pharma healthcare summit. The focus is on and to deliver compelling affordable care with key role for technologies.

  30.   Retweeted

    Carl June believes we’re only a few years away from outpatient CAR therapies, with no need for intensive infrastructure with ICU.

  31.   Retweeted

    Dr. Maus ⁩ monitoring data from clinical trial is very important development of new targets multiple drugs multiple mechanism multiple specificities more modification to one cell contamination results

  32.   Retweeted

    “Cancer is a collection of rare diseases” – , Director of Clinical Research, Cancer

  33.   Retweeted

    Sanat Chattopadhyay of US Merck says costs of manufacturing in US/Europe is significantly higher because technology deployed is ancient, both in small molecules and biologics.

  34.   Retweeted

    Rare disease taking center stage as technologies mature, panel moderated by CRISPR Therapeutics CEO Samarth Kulkarni

  35.   Retweeted
    Replying to  

    Enjoyed moderating the panel on manufacturing in the future as bio pharma companies explore ways to deliver drugs at affordable price and address access challenges. Digital innovation in manufacturing and supply chains will be key.

  36.   Retweeted

    Price is part of doing business…if you are not able to define value, you are sunk, says Chris Viehbacher Gurnet Point Capital

  37.   Retweeted

    All diseases are unique Your perspective changes when you or someone you love is diagnosed with a life-altering disease – rare diseases panel

  38.   Retweeted

    Arun Singhal, addl secretary, health ministry speaks about Ayushman Bharat, trade margin rationalisation, clinical trial rules at meeting

  39.   Retweeted

    Clinical trials in India picking up steadily, says Eswara Reddy, DCGI

  40.   Retweeted

    Regulators will “provide complete support” for clinical trials in India provided drug developers meet the new requirements – Drug Controller General of India Eswara Reddy

  41.   Retweeted

    discusses prevention and treatment of early disease e.g. precancer The challenge is to work out the commercial model says organize around prevention Early diagnosis / discovery of disease processes saves lives (’s raison d’etre)

  42. AMAZING EVENT @AVIVA11950 13th Annual & Healthcare Summit, Thursday, May 9, 2019, Marriott Hotel, Cambridge, MA via

  43. Dr. James Bradner, President, Novartis Institutes for BioMedical Research biophysical biochemical protein degradation – rewire disease cells with biomolecules combing propertitie of permiability of small molecules

  44. World PGD Growth of 4% in India 31%

  45.   Retweeted

    Takeda R&D Head Andy Plump asks question from audience to peers on stage: “I’ve been in the industry for 18 years and I can’t understand why a clinical trial costs so much. Why does it?”

  46.   Retweeted

    Manufacturing in the age of individualized medicine? We may need a completely different thinking for that says Paul Mckenzie, Biogen

  47.   Retweeted

    The so-called low-cost manufacturing edge of India will go away in a few years, says Hari Bhartia, Jubilant…being closer to the customer will be important

  48.   Retweeted

    Tricky question: getting patients to cancer centers to participate in clinical trials. Should patients be reimbursed for long travels and other expenses or will it be seen as an inducement?

  49.   Retweeted

    A chance to collaborate with my old colleague & friend ! -> another point from same panel: AbbVie CMO Rob Scott predicts tele-health solutions for clinical trial patients will be scalable soon

  50.   Retweeted

    Carl June: There are no CAR-T clinical trials in India. But says countries like India could eventually leapfrog to next gen (outpatient) cell therapy which will require less infrastructure + lower COG

  51.   Retweeted

    The 13th Annual BioPharma & Healthcare Summit is being kicked off by Andrew Plump. In his opening remarks, he commented that we should feel privileged as attendees because not even or is invited to this meeting.

  52.   Retweeted

    A well-represented panel of scientists, CEOs and entrepreneurs discuss a range of discovery research from CAR-Ts to small molecules…on the same panel is Arjun Surya of Curadev that licensed a preclinical oncology lead to Takeda.

  53. The promise of India is the largest democracy, the educated workforce the size of the market for therapeutics, access and price, reimbursement and regulation. DCGI the analogue of FDA is very active and innovated the challenge of 1.3 Billion a population of patients

  54. Great Leader in immunotherapy, Carl June early inventor and endless commitment to patient a Pro for BioPharma

  55. GREAT Conference of who is who in BioPharma, Boston at the top 500 startats of Biotech in Cambridge, MA ten years afo only a handful, boost by Novartis HQS in Cambridge, MA

  56. Liked the analogy of

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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 addressKarun 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 addressKarun 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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Reporter: Gail S. Thornton

 

From The Wall Street Journal (www.wsj.com)

Published January 9, 2019

Health-Care CEOs Outline Strategies at J.P. Morgan Conference

Chiefs at Johnson & Johnson, CVS discuss what’s next on a range of industry issues

One of the biggest health conferences of the year for investors, the J.P. Morgan Health-Care Conference, is taking place this week in San Francisco. Here are some of the hot topics covered at the four-day event, which wraps up Thursday.

BioMarin Mulls Payment Plans

BioMarin Pharmaceutical Inc. CEO Jean-Jacques Bienaimé said he would consider pursuing installment payment arrangements for the biotech’s experimental gene therapy for hemophilia. At the conference, Mr. Bienaimé told the Wall Street Journal that the one-time infusion, Valrox, is likely to cost in the millions because studies have shown it can eliminate bleeding episodes in patients, and current hemophilia treatments taken chronically can cost millions over several years. “We’re not trying to charge more than existing therapies,” he said. “We want to offer a better treatment at the same or lower cost.”

Johnson & Johnson Warns on Pricing

As politicians hammer drug prices, Johnson & Johnson CEO Alex Gorsky suggested companies need to police themselves. At the conference, Mr. Gorsky told investors that drug companies should price drugs reasonably and be transparent. “If we don’t do this as an industry, I think there will be other alternatives that will be more onerous for us,” Mr. Gorsky says. Some drugmakers pulled back from price increases in mid-2018 amid heightened political scrutiny, but prices went up for many drugs at the start of 2019.

Marijuana-Derived Drugs Show Promise

 

CVS Discusses New Stores

CVS Health Corp. Chief Executive Larry Merlo began showing initial concepts the company will be testing as it begins piloting new models of its drugstores that incorporate its Aetna combination. The first new test store will open next month in Houston, he told investors, and it will include expanded health-care services including a new concierge who will help patients with questions. 

Aetna Savings On the Way

Mr. Merlo also spelled out when the company will achieve the initial $750 million in synergies it has promised from the CVS-Aetna deal. In the first quarter, he said the company will see benefits from consolidating corporate functions. Savings from procurement and aligning lists of covered drugs should be seen in the first half, he says. Medical-cost savings will start affecting results toward the end of the year, he noted. 

Lilly Cuts Price

Drugmaker Eli Lilly & Co. expects average net US pricing for its drugs–after rebates and discounts–to decline in the low- to mid-single digits on a percentage basis this year, Chief Financial Officer Josh Smiley told the Journal. Lilly’s net prices had risen during the first half of 2018, but dropped in the third quarter as the company took a “restrained approach,” Mr. Smiley said. Lilly, which hasn’t yet reported fourth-quarter results, took some list price increases for cancer drugs in late December but hasn’t raised prices in the new year, he said.

Peter Loftus at peter.loftus@wsj.com and Anna Wilde Mathews at anna.mathews@wsj.com

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