A revised version of controversial legislation seeking to create a conditional FDA approval pathway for rare disease drugs and require the drugs be covered without cost-sharing has been introduced in the House, signaling potential movement toward including the bill in an expected congressional lame-duck package.
The House version of the revised Promising Pathways Act 2.0 was introduced Oct. 4 and is sponsored by Rep. Bruce Westerman (R-AR), with one cosponsor so far, Rep. Mike Garcia (R-CA). It mirrors a Senate version introduced in May by Sen. Mike Braun (R-IN) with several cosponsors from both parties, which aimed to address issues that kept the original Promising Pathways Act from advancing. The revised bill narrows eligible indications to include only drugs intended to treat rare or terminal diseases, among other changes.
“When individuals struggling with life-threatening diseases face the uncertainty of when they will be able to receive treatments or vital prescription orders, it’s clear that we have a serious problem,” Westerman told Inside Health Policy. “The Promising Pathway Act 2.0 (H.R. 9938) provides a clear-cut solution for patients with life-threatening diseases to receive the care they deserve.”
An upcoming meeting on Capitol Hill is also set to delve into pathways for FDA rare disease approvals. Janet Woodcock, former principal deputy commissioner of FDA, will moderate a panel at the Capitol Visitor Center Oct. 25 with the title “When Exceptions Need Their Own Rule: A Rare Disease Approval Pathway.” The event is hosted by rare disease advocacy group the Haystack Project and will also feature former FDA senior advisor Jeanne Ireland and former FDA associate chief counsel David Fox.
Woodcock joined the Haystack Project as a “mission-focused advisor” earlier this year after leaving FDA and said she does not plan to interact directly with the agency as part of that role or to represent any specific companies.
Experts weighing in on FDA’s decisions for rare disease drugs have also said they’d like to see the agency sometimes relax its safety and efficacy standards for rare disease drugs. At a recent advisory committee meeting on a potential treatment for the ultra-rare Barth syndrome, committee members expressed that they wanted the medication to be available to patients even though they had doubts about its efficacy.
“I’m not sure this met the bar that you guys usually ask for, but I think there needs to be a different structure for rare diseases,” committee member Jonathan Soslow, a professor of pediatrics at Vanderbilt University Medical Center, said.
While FDA has taken numerous steps to meet the needs of rare disease drug developers and encourage innovation, some patient advocates have called on the agency to do more, including increasing use of the accelerated approval pathway. FDA use of accelerated approval has met with criticism from other stakeholders, who say it can give patients false hope and lead to marketing of ineffective drugs.
PPA 2.0, like the previous version of the bill, would let FDA grant time-limited conditional approval for some drugs, though it narrows eligible indications to include only drugs intended to treat rare or terminal diseases. Drugs would receive an initial conditional approval for two years and be able to renew it for up to eight years, after which the sponsor would need to seek approval through another pathway or withdraw it from the market.
The new version is limited to drugs for diseases that are eligible for the Orphan Drug Act and substantially impact quality of life, or progressive and terminal. Drugs must show evidence of safety and promising initial evidence of effectiveness.
Braun’s revised version of the bill also would require federal and private payers to cover conditionally approved drugs without cost-sharing.
To retain conditional approval beyond an initial two-year period, drug sponsors would need to show additional evidence of effectiveness. Sponsors would be required to bring conditionally approved drugs to the market within one year of receiving conditional approval, and patients would be required to participate in a registry during the treatment.
The legislation is endorsed by over 100 rare disease and other health advocacy groups. But one of the largest rare disease advocacy groups, the National Organization for Rare Disorders (NORD), said PPA 2.0 didn’t address its concerns with the original bill, including that conditional approval pathway could mean more patients take medications that initially seem promising but are actually unsafe or ineffective and could act as an obstacle for testing of other, more promising treatments.
