Expanded Access to Experimental Medicines – Where is Federal Legislation taking us?

By: Jane Reese-Coulbourne, MS ChE


In the past week, there has been a lot of activity concerning federal “Right to Try” legislation. There are two pieces of federal legislation in play right now, while 37 States currently have somewhat varying “Right to Try” laws on their books. The question is will all of this legislation actually lead to improved access for patients to experimental medicines.

Two important bills have very recently advanced in the Senate that address expanded access to experimental medicines – Senator Ron Johnson’s (R-WI) standalone legislation S.204 (115) and the Prescription Drug User Fee Act (PDUFA) Hatch Amendment.

Last Thursday, August 3, Sen. Ron Johnson (R-WI) agreed to pull back his threat to hold up the FDA user fee package in exchange for the Senate to pass a stand-alone bill S.204 (115).  After the user fee vote, an amended version of his “Right to Try” legislation was passed in the Senate under unanimous consent. Now it has to go to the House for a vote after the summer break and if successful there, will then go to President Trump for his signature.

Championed by the Goldwater Institute, and originally called the “Trickett Wendler, Frank Mongiello, and Jordan McLinn Right to Try Act”, the stand-alone bill S.204 (115) has been significantly modified by bill opponents since its initial introduction.

While the bill contains some provisions for advancing patient access to experimental medicines, there is also some ambiguity in the language. The first section of the document says a drug must have filed for an IND to qualify for expanded access, but in the “Exemptions” section of S.204 (115) it says that “IND law” does not apply to expanded access medicines. This could be interpreted to mean that access to unapproved drugs is NOT overseen by FDA. This will surely need to be clarified.

Basic provisions of S.204 (115) include:

  • The use of unapproved medicines by patients diagnosed with a life-threatening or serious condition is allowed when:
    • Drugs in question have already gone through preliminary testing on humans (i.e. completed phase I)
    • Medicines continue to be evaluated in research overseen by the FDA
    • Eligible patients have exhausted other treatment options and are unable to participate in ongoing clinical trials
    • Patient provides informed consent
  • HHS Secretary will not use clinical outcome data associated with use of investigational drug to delay or adversely affect review/approval
    • HHS Secretary may allow use of outcome data if they see it as critical to determining the safety of an investigational drug or if the sponsor requests the use of the data
    • The sponsor shall submit to the Secretary an annual summary of such use that includes number of doses supplied, number of patients treated, uses for which the drug was made available, and any known serious adverse events
  • There can be NO liability against the sponsor, prescriber, and/or dispenser
    • Unless they exhibit reckless or willful misconduct or gross negligence
    • If they choose to NOT provide access to their investigational drug
  • Legislation does not establish a new entitlement or mandate

Also last Thursday, the Senate Committee on Health, Education, Labor & Pensions (HELP) advanced a bill, called the Prescription Drug User Fee Act (PDUFA) Hatch Amendment, that would reauthorize the pharmaceutical, medical device, generic drug and biosimilar user fee agreements that expire at the end of September. Having already been voted on in the House, it now goes to President Trump for his signature, which seems imminent at this point.

The Prescription Drug User Fee Act (PDUFA), included the Hatch Amendment which requires:

  • FDA Commissioner is to work with the National Institutes of Health (NIH), patients and other stakeholders to convene a public meeting and issue a report on clinical trial inclusion and exclusion criteria to inform new FDA guidance to address methodological approaches that a sponsor of an investigation of a new drug may take to
    • Broaden eligibility criteria for clinical trials, with respect to drugs for the treatment of serious and life-threatening conditions for which there is an unmet medical need
    • Develop eligibility criteria to increase trial recruitment to clinical trials so that enrollment in such trials more accurately reflects the patients most likely to receive the drug
  • FDA will have to issue guidance or regulations to streamline the institutional review board review for individual pediatric and adult patient expanded access” protocols.
  • A section in the federal Food, Drug and Cosmetic Act requiring a licensed physician to determine that the person has no comparable or satisfactory alternative therapy available to treat their disease and that the probable risk to the person from the investigational drug is not greater than the probable risk from the disease or condition.

There are still many outstanding issues about expanded access/compassionate use that have not been resolved or even addressed in these proposed laws. Continued support of “Right to Try” laws by the Goldwater Foundation, some members of Congress, and the Trump Administration ensure that the discussions and political maneuvering related to patient access to experimental medicines will continue for the foreseeable future.

We will be discussing new best practices for the utilization of expanded access programs in the clinical development process at The 2017 Expanded Access Summit, September 15, 2017 in Cambridge, MA.  I will have the honor of speaking alongside a stellar group of experts on this topic at this event. I hope to see you there!

For more information on some current developments about expanded patient access, you can read the following articles: Politico, Vox, NY Times, RAP, Washington Post, Clinical Leaders. Please keep an eye out for my future blogs on news and developments on the expanded drug access front.

 




About the Author:

Jane Reese-Coulbourne, MS ChE

Jane is a senior consultant at MK&A.  Jane brings comprehensive knowledge of evolving regulatory requirements and sensitivity to patients and sponsors’ needed in the conception and design of pre-approval drug access programs. She has helped shape policy and practice in this new paradigm of patient advocacy for early access to experimental treatments.

Most recently, Jane was the founding executive director of the Reagan-Udall Foundation for the FDA. Prior to that, she was an advisor to Dr. Richard Klausner, director of the National Cancer Institute. She served as executive vice president of the National Breast Cancer Coalition and as the board chair of the Lung Cancer Alliance.  Jane holds an MS in chemical engineering from the University of Virginia.

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