Pink Sheet: global policy and regulatory coverage
By John Davis 31 Mar 2020
This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the...
The 'Right to Try' movement is gaining momentum at the federal level with support from both the Trump Administration and Republicans in Congress to enact legislation that would direct FDA not to interfere with existing state laws and allow terminally ill patients to access unapproved treatments.
The “Right to Try” movement now has the explicit backing of the White House and enactment of legislation to prevent FDA from interfering with state laws in that area may now be inevitable.
Vice President Mike Pence met with right-to-try advocates Feb. 7. Pence is a backer of opening up broader access for compassionate use; as governor of Indiana, he signed state “Right to Try” legislation in law in 2015 and made statements during the Presidential campaign in support of such efforts. (Indiana is one of 33 states to enact legislation.)
The official “readout” of Pence’s meeting makes clear that his support for the effort is now Administration policy.
Pence’s meeting builds on statements made by President Trump before a Jan. 31 meeting with pharmaceutical CEOs, where he talked about the need to “change a lot of rules” in drug regulation. (Also see "Trump Promises Changes To 'A Lot Of Rules' At US FDA" - Pink Sheet, 31 Jan, 2017.)
“One thing that’s always disturbed me is we come up with a new drug for a patient who is terminal, and the FDA says we can’t have this drug used on the patient. But the patient within four weeks will be dead,” President Trump said. “They say ‘well we still can’t approve the drug and we don’t know if the drug works or if it doesn’t work but we can’t approve the drug because we don’t want to hurt the patient.’ But the patient is not going to live more than four weeks.”
Advocates of “Right to Try” failed to include a policy in the “21st Century Cures” Act but have a second chance in the pending FDA user fee legislation (which must be enacted before Oct. 1). Given the White House endorsement and existing strong support from a Republican-controlled Congress, some form of “Right to Try” amendment is probably inevitable in 2017.
The Risk To Drug Manufacturers
The risk to drug manufacturers is that the “Right to Try” debate, which has thus far positioned FDA as the primary barrier to patient access to unapproved therapies, will pivot to an anti-industry theme once legislation is enacted.
Thus far, industry has suggested that broad access to investigational therapies is hindered by regulatory barriers and by potential liability (both in the regulatory and legal sense) from adverse events. “Right to Try” legislation introduced in the House and Senate would seem to address those concerns. As drafted, the language would prevent FDA from using data reported during compassionate use “to delay or otherwise adversely impact review or approval.” The bills also have safe harbors in place to protect manufacturers from product liability suits.
Sen. Ron Johnson’s (R-Wisc.) has been a leading voice on the issue; his “Trickett-Wendler Right to Try Act”, was reintroduced this session on January 24. Identical legislation was introduced February 6 in the House (H.R. 878) by Reps. Andy Biggs (R-Arizona) and Brian Fitzpatrick (R-Penn.). Both bills have broad support among Republicans. In addition, two Democrats – Sen. Joe Donnelly (D-IN) and Joe Manchin (D-WV) – signed onto Sen. Johnson’s bill, and Rep. Andre Carson (D-IN) is a co-sponsor of HR 878.
Sen. Johnson held a hearing on Trickett-Wendler on Sept. 22 in the Senate Homeland Security & Government Affairs Committee, which he chairs. The hearing had a distinct anti-FDA tone, with FDA Associate Commissioner Public Health Strategy and Analysis Peter Lurie repeatedly asked to defend the agency’s policies on compassionate use. (Also see "'Right To Try' Or 'Right To Ask'? Hearing Spotlights Adverse Events As Key Barrier To Expanded Access" - Pink Sheet, 26 Sep, 2016.)
Lurie testified that FDA approves 99% of all requests for compassionate use, and has recently streamlined the process for requests and adopted additional steps to make the system user-friendly for patients. Sen. Johnson dismissed those steps as ineffective, and argued that the cost of providing drugs under compassionate use, as well as the regulatory and legal risk, were “standing in the way” of patient access.
FDA recognizes the continued pressure from the Right-to-Try advocates and is said to be preparing to designate a new position in each of the medical product centers to help facilitate responses to requests for compassionate use.
President Trump’s final pick for FDA commissioner will also dictate how the issue develops. A leading contender for the commissioner job, Jim O’Neill, has advocated for approving drugs after sponsors have demonstrated safety and then demonstrating efficacy in the post-marketing setting. Those statements put him on the spectrum of pushing for Right-to-Try efforts.
The push on compassionate use during the debate on “21st Century Cures” wasn't a complete failure. What survived in the legislation is an expanded access provision that requires drug companies to establish a formal policy for patient requests to access unapproved therapies. Under that provision, manufacturers are required to make public and “readily available” a policy by which the company can evaluate or respond to requests from patients for expanded access to investigational products. Companies have 60 days from enactment to comply with the provision, setting a Feb. 11 deadline.
From the editors of the RPM Report
Pink Sheet: global policy and regulatory coverage
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