Participant Level Data from Clinical Trials used for Licensure

Should

be made Publicly Available to Anyone

Group 2

Abhirup Datta, Michelle Gleason, Emily Goren, Reilly Hourigan, Eleena Iisakka, Ahmad Raza, Youssef Roman, Robert Stewart

Professional Perspective Fulfills obligations to research participants EFPIA and PhRMA--biopharmaceutical companies in Europe and America • endorsed joint “ Principles for Responsible Clinical Trial Data Sharing: Our Commitment to Patients and Researchers .” July, 2013.

• “The data sharing commitments reflect...companies’ strong support for sharing clinical trial data to benefit patients and foster scientific discovery in a way that maintains patient privacy, the integrity of regulatory systems, and incentives to invest in biomedical research.” (Richard Bergström, Director General of EFPIA) IOM (Institute of Medicine). 2013. Sharing clinical research data: Workshop summary. Washington, DC: The National Academies Press.

EFPIA and PhRMA Release Joint Principles for Responsible Clinical Trial Data Sharing to Benefit Patients. 2013.

Patient Perspective De-identified to maintain privacy • Main concern of data sharing Patient can make educated decisions • Ability to weigh pros and cons Develop trust with clinician and the process • Comfort Enhance patient care • Develops confidence

Clinician Perspective Patient trust is vital to the patient –physician relationship • Patient trust in clinician provides basis for sharing personal information necessary for the clinician to provide the best care • Patients' trust in their physician are strongly associated with healthcare adherence and patients' satisfaction with their physician (Safran, 1998) • “Extensive research has shown that no matter how knowledgeable a clinician might be, if he or she is not able to open good communication with the patient, he or she may be of no help.” (Asnani, 2009) Safran DG, Taira D, Rogers WH, Kosinski M, Ware JE, Tarlov AR. Linking primary care performance to outcomes of care. Journal of Family Practice. 1998; 47(3):213-220.

Asnani MR. Patient-physician communication. West Indian Med J. 2009; 58(4):357-61.

Clinician Perspective Strengthens evidence-based medicine • Clinician can trust results of clinical trials used to license the drugs they are prescribing • Evidence-based medicine must take into account all evidence on risk and benefits of available treatments, but current published data and reports on clincaltrials.gov are only a fraction of the evidence • “In a profession that seeks to rely on evidence, it is ironic that we tolerate a system that enables evidence to be outside of public view.” (Krumholz, 2012) Cabana MD, Rand CS, Powe NR, et al. Why Don't Physicians Follow Clinical Practice Guidelines?: A Framework for Improvement. JAMA. 1999; 282(15):1458-1465. Krumholz HM. Circulation: Cardiovascular Quality and Outcomes. 2012; 5:141-142.

Research Perspective • Secondary data analysis Test additional hypotheses • Education, Training, and Future Research Provides opportunities for young researchers to use real-world data • • • Keeping them honest Aligns with the general principles of peer-review Opportunity to apply an objective perspective and find something the research might have missed Can also have dramatic ramifications, see for example Reinhart and Rogoff (2010)

Public Health Perspective • • Transparency and Credibility Peer review Publication bias • • Other benefits Better trial designs Increased reputation risk Yale University Open Data Access (YODA) Project • “Provides a means for rigorous and objective evaluation of clinical trial data” • Currently enrolled organizations: Medtronic, Johnson & Johnson Hans Georg Eichler, M.D., Frank Pétavy, M.Sc., Francesco Pignatti, M.D., and Guido Rasi, M.D. N Engl J Med 2013; 369:1577-1579

Pros vs. Cons Patient trust Clinician confidence Secondary data uses Improve trial designs Accountability Public health interest Patient privacy Pharma proprietary interest

Open access of corporate clinical trial data to the general public is not a good idea.

GROUP3: Patrick Kurkiewicz

, Brent Leininger, Lifeng Lin, Xiang Li, Bryan Trottier Jr, Yuanyuan Wang

Federally funded research should have open access.

Not corporate research

In theory, open access is a great idea.

• Makes innovation more efficient • • • Less overlapping trials Increased power from meta-analyses Better understanding of prognostic factors for design of future trials • Companies work together and learn from one another and everyone wins • Public Good • Less participants exposed to risks in trials for products/procedures that have already been

But in practice, open access would disincentivize innovation

Proprietary Info.

• Innovation starts with proprietary information/ intellectual property • In many cases proprietary information comes from a trial Proprietary drugs.

Profit “Retained Earnings ”

Competitors Would take Advantage of Free Information

“Requests for clinical study to the EMA have been filed mostly by competitors of the company who performed the initial study”.

47. Rabesandratana T. Europe: drug watchdog ponders how to open clinical trial data vault. Science 2013;339:1369-70.

Competitor Investor

Media Sensationalizes

-Will play up potential issues with current procedures and devices to gain viewers, listeners, and readers.

The USA is the Most Litigious Society in the World.

-We spend about 2.2 percent of gross domestic product, roughly $310 billion a year, or about $1,000 for each person in the country on TORT litigation, much higher than any other country. (A large part of the cost of healthcare) -How do less scientifically juries and judges determine difference between correlation and

Who pays for open access?

-

The company the acquired the data?

-Current system already has an issue with cost, not quality (relatively speaking).

-

The government?

-Why not just expand FDA to take on more monitoring responsibility?

In terms of spotting issues for the public good…Is Secondary Review More Legitimate?

EVERYONE has some potential for conflict of interest -Academia -Competitors -The Media -The FDA -Manufacturers

Our Current System

But Manufacturers’ interest in profitability goes two ways -Interest in limiting liability and public opinion causes them to share much of their data.

-Rule of law/TORTs -Research necessary for the public good is paid for by government.

-We have an approval process via the FDA -Doctors and Patients