Promising Pathway Act 2.0

This bill amends the Federal Food, Drug, and Cosmetic Act to create a time-limited conditional approval pathway for certain drugs and biological products intended to treat rare, progressive, and serious diseases, subject to specific obligations.

Sec. 2, Conditional approval for rare, progressive, and serious diseases: This section adds a new section 524C to the FD&C Act. It allows a drug sponsor to apply for conditional approval if the drug treats a rapidly progressive, terminal disease with substantial unmet medical need, or a rare disease that shortens lifespan or reduces quality of life. Drugs for material threats identified by Homeland Security are excluded.

Standard of review for conditional approval: The Secretary must approve if safety is shown by a phase 1 trial or other demonstration, and effectiveness is shown by an ongoing or completed phase 2 trial. For terminal pediatric rare diseases, animal model evidence may suffice. The sponsor must affirm intent to pursue full approval. Rolling, real-time review and real-world evidence are allowed.

FDA authority to withdraw conditional approval: The Secretary may withdraw approval if safety data no longer supports it, the application contained false material facts, or the drug is no longer eligible. Withdrawal stops new patients but allows current patients to continue. Automatic withdrawal occurs if renewal is not sought or the drug is not marketed within one year. Appeals are allowed except for automatic withdrawal.

Labeling and review of materials: All labeling and advertising must state "conditionally approved for a limited population." Promotional materials must be submitted to the FDA 45 days before distribution. The FDA must maintain a public list of conditionally approved drugs.

Renewal of conditional approval: Conditional approval lasts 2 years, renewable up to three times for a total of 8 years. Renewal applications require additional preliminary evidence of effectiveness. Small population diseases may be exempt from this requirement. Renewal may be denied for data fraud.

Observational registries: Sponsors must establish an observational registry for patients treated with the drug. Patients must enroll and provide informed consent. Sponsors submit risk and benefit data. Registries must provide patients access to their data and give researchers access to de-identified data.

Pursuit of a different indication: If conditional approval is withdrawn, expires, or renewal is denied, the sponsor may petition for conditional approval for a different indication within 2 years.

Transition to other forms of approval: A conditionally approved drug may receive full approval under other provisions. The FDA may consider registry evidence for full approval.

Informed consent: Patients must provide informed consent before being prescribed a conditionally approved drug, following federal regulations.

Limitation on liability: No state law liability lies against the sponsor, manufacturer, or health care provider for a conditionally approved drug absent intentional wrongdoing.

Report to Congress: The Secretary must report to Congress every 2 years on all conditionally approved drugs, including patient numbers and data types.