Drug regulation, promotion, and

advocacy in 2017 JERRY AVORN, M.D.

Professor of Medicine, Harvard Medical School Chief, Division of Pharmacoepidemiology &

Pharmacoeconomics Brigham and Women’s Hospital

Overview: A challenging time for evidence-based prescribing

and drug regulation

• 21st Century Cures Act • “Commercial free speech” attacks • New FDA Commissioner, climate • Whose risk is it, anyway? • What can help

The 21st Century Cures Act

passed December, 2016

A bad solution to a problem that barely exists

• A bogus meme: “FDA is so slow and makes such impossible demands that it keeps drugs from the American people and increases drug development costs higher prices.”

• The truth: not so. – NEJM papers from Krumholz-Ross group – 6 mo. for priority review – Very high approval rate – High prevalence of surrogate measures already

What the 21st Century Cures Act does • Some good things:

– Slightly more funding for NIH, maybe – But still not enough

• Kesselheim, Tan, Avorn: Health Affairs 2015

– Mental health $, opioid $

• New law encourages more approval based on biomarkers (“drug development tools”) – Good and bad aspects of biomarker-based approval – The eteplirsen cautionary example

• Ref: Kesselheim & Avorn, JAMA 2016 and JAMA 1/5/17

“Commercial free speech” vs. science

The 1st Amendment attack • Important background:

– Sorrel, ‘Citizens United’ cases

• Caronia, 2011 – Xyrem: narcolepsy vs. chronic pain, fibromyalgia,

chronic fatigue, use in children

• Amarin: Vascepta and triglycerides • FDA guidances and the future of drug

promotion • Ref: Avorn, Sarpatwari, Kesselheim, NEJM 2015

• Prognosis: grim

New sheriff(s) in town

A whole new world • “Government isn’t the solution to our problem,

government is the problem.” • Pres. Ronald Reagan, 1st Inaugural Address, January 1981

• Two of the finalists for FDA Commissioner job: – Jim O’Neill

• Venture capitalist, no science training • FDA should just make sure drugs are not toxic, then “let

people start using them at their own risk.”

– Scott Gottlieb, MD • American Enterprise Institute, de-regulator • Wrote WSJ article, “How the FDA could cost you your life.”

Patient advocacy groups and “Right to Try” laws

Grass roots, or Astroturf? Does it matter?

• The role of patient groups in drug approval – varied origins and funding – scientific legitimacy – roles: current and ideal

• Interesting overlaps across the law, clinical care, medical ethics, science policy, economics, and raw politics

Whose risk is it, anyway?

• Question is no longer as moot as it was last year. • The ethics of allowing use of (probably)

ineffective and (possibly) unsafe chemicals – semantics of access – who’s to pay?

• Practical impact on generating the data that current and all future patients (and society) need.

One solution: marketing real evidence in a post-fact world

• “Academic detailing” – rationale – ample RCT evidence of effectiveness – evolution

• Alosa Health

– progress – future

Can regulation support this kind of evidence-based education?

• Funding academic detailing outreach programs – state tax on manufacturers – license fees for pharma sales reps – settlement $ from litigation by AGs – tiny % of insurance premiums or care revenues

non-commercial educational programs

• Other regulatory strategies – Competency requirements for prescriber licensing

Acknowledgements

My talented colleagues in the PORTAL program of the Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham & Women’s Hospital and Harvard Medical School:

Aaron Kesselheim, MD, JD MPH Ameet Sarpatwari, JD, PhD Jonathan Darrow, JD, SJD, MBA

For more information:

www.DrugEpi.org www. AlosaHealth.org

Avorn, “Powerful Medicines: The Benefits, Risks, and Costs of Prescription Drugs” www.PowerfulMedicines.org