Participant Level Data from Clinical Trials used for Licensure Should be made Publicly Available to Anyone

Group 2Abhirup Datta, Michelle Gleason, Emily Goren, Reilly Hourigan,Eleena Iisakka, Ahmad Raza, Youssef Roman, Robert Stewart

Professional PerspectiveFulfills 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 PerspectiveSecondary 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 PerspectiveTransparency 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 & JohnsonHans-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 trustClinician confidenceSecondary data usesImprove trial designsAccountabilityPublic health interest

Patient privacyPharma 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 extensively proven to be clinically ineffective.

• Innovation starts with proprietary information/ intellectual property

• In many cases proprietary information comes from a trial that company has managed.

But in practice, open access would disincentivize innovation

Operating Costs

Profit “Retained Earnings”

Proprietary Info.

Proprietary drugs.

Competitors Would take Advantage of Free Information

Competitor Investor

“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.

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 causation?

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