BioPharma, Policy

Gottlieb at BIO: FDA developing processes around right-to-try law

FDA commissioner gives clues of agency’s right-to-try strategy in Q&A session during chat with BIO President Jim Greenwood.

Perhaps it was inevitable that with Food and Drug Administration Commissioner Scott Gottlieb speaking before an auditorium full of biotechnology executives, a particularly contentious topic would come up: the new right-to-try law.

Gottlieb was at the BIO annual meeting in Boston Thursday for a fireside chat with the organization’s president, Jim Greenwood. During the question-and-answer session, the CEO of a private company developing a drug for pancreatic cancer told Gottlieb that he had recently received a “threatening” phone call from the daughter of a patient who wanted to obtain the drug via right-to-try.

President Trump signed the federal right-to-try bill into law on May 30, following its passage in the House. The law allows terminally ill patients to obtain directly from drug companies medicines that have passed Phase I testing, but unlike the FDA’s expanded access program, they can bypass the regulatory agency. Proponents say the law is necessary to provide desperate patients with quick access to otherwise unobtainable experimental drugs, while critics note the FDA swiftly approves nearly all expanded access requests, and drug companies have no obligation to comply with right-to-try requests anyway. In a letter to Gottlieb, the bill’s author, Sen. Ron Johnson, R-Wisconsin, wrote that the law was intended to weaken the FDA’s oversight.

Asked for clarity in an interview after the fireside chat, the CEO – who asked not to be named in order to avoid further such contacts from patients – said that the call, which occurred on Sunday night while he was attending the recently concluded ASCO meeting in Chicago, started on a positive note, with the woman explaining that her mother had Stage IV metastatic pancreatic cancer. However, it soon turned negative, and he hung up the phone when the woman became “obnoxious” after finding that she would have to have her mother’s physician fill out a compassionate use form on the website, which she asserted was not required under right-to-try. The CEO said that his personal mobile phone number, which had been on the website, was removed Thursday morning on advice of the company’s lawyers due to the exchange. However, he added that he empathized with the woman, given her situation.

The CEO further said that the company has already received 10 right-to-try requests. However, while saying that he is not entirely against the law because it gives patients a chance to receive drugs, it could also hurt recruitment in clinical trials because it would obviate the need for patients to join them.

During the chat, Gottlieb said that the FDA had convened several agency leaders Wednesday to formulate a process for handling the program, which Rachel Sherman – its principal deputy commissioner – will be in charge of and to come up with a list of potential recommendations to drug companies, which could be as simple as a Q&A on the agency website. The agency remains committed to the expanded access program, he said, adding that it will exist alongside right-to-try.

Photo: Alaric DeArment, MedCity News