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:
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:
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.
Veronica Lopez, MPH, is an independent consultant working with VOZ. Veronica helps VOZ develop new ways of building equitable and responsible partnerships between industry and patients, advocates, health care professionals, and policy makers.