New Momentum for a Time-Limited Conditional Approval Pathway for Rare Disease Drugs

On October 4, 2024, a US House version of the revised Promising Pathway Act (PPA) 2.0 was introduced, sponsored by Rep. Bruce Westerman (R-AR). The bill (H.R.9938) mirrors a US Senate version that was introduced in May 2024 (S.4426) that would authorize the US Food  and Drug Administration (FDA) to grant time-limited conditional approval to drugs for rapidly progressive, terminal diseases with substantial unmet need for treatments that are eligible for the Orphan Drug Act and result in a substantially shortened lifespan, substantial reduction in quality of life, or other substantial adverse health effects.

If approved by both the Senate and the House without further amendment and signed into law, drugs eligible under PPA 2.0 could receive an initial conditional approval for two years that is renewable for up to eight years, after which the sponsor would need to seek approval through another pathway or withdraw the drug from the market. Importantly, the bills exempt small population diseases, which the bills do not define, from having to pursue renewal of conditional approval or present additional preliminary evidence of effectiveness, which would be significant for ultra rare disease drugs that could obtain conditional approval on this new pathway. With the FDA’s newly formed Rare Disease Innovation Hub looking to create efficiencies for the development of rare disease drugs, the time may be right for the advancement of the PPA 2.0 bills in the Senate and House. However, the legislation has been criticized as enabling approval of medications that initially seem promising but do not prove to be safe and effective. As a result, additional amendments to the current proposals may be needed to ensure that conditional approval renewal, even in small population conditions, would not continue in the face of evidence that the drugs are not safe or effective.

Contact our Goodwin Life Sciences Regulatory & Compliance team to discuss the downstream implications of the potential advancement of the PPA 2.0 for your business and to learn how to be added to our regulatory alerts as we continue to monitor developments in this area.

This informational piece, which may be considered advertising under the ethical rules of certain jurisdictions, is provided on the understanding that it does not constitute the rendering of legal advice or other professional advice by Goodwin or its lawyers. Prior results do not guarantee similar outcomes.