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. It establishes eligibility criteria, a standard of review, requirements for labeling and patient consent, and rules for renewal, withdrawal, and transition to full approval.

Sec. 2, Conditional approval of new human drugs for individuals with 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 need, or a rare disease that shortens lifespan or reduces quality of life. The Secretary must give priority review and may use real-world evidence. Conditional approval requires evidence of safety from a phase 1 trial or other demonstration, and evidence of effectiveness from an ongoing or completed phase 2 trial, or for pediatric rare diseases, preliminary evidence from animal studies. The sponsor must affirm intent to pursue full approval.

Sec. 2, FDA authority to withdraw conditional approval: The Secretary may withdraw conditional approval if safety data no longer supports it, the application contained a false statement, or the drug is no longer eligible. Upon withdrawal, the sponsor cannot make the drug available to new patients but may continue for existing patients. The Secretary must establish an appeals process. Conditional approval automatically withdraws if the sponsor does not apply for renewal on time or if the drug is not marketed within one year.

Sec. 2, Labeling and review of materials: All labeling and advertising must state 'conditionally approved for a limited population' prominently. Promotional materials must be submitted to the Secretary for review at least 45 days before distribution. The Secretary must maintain a public list of conditionally approved drugs.

Sec. 2, Renewal and duration: Conditional approval is effective for 2 years and can be renewed for up to three additional 2-year periods, for a total of 8 years. Renewal applications require additional preliminary evidence of effectiveness. The Secretary may exempt small population diseases from this requirement.

Sec. 2, Observational registries: The sponsor must establish an observational registry for patients treated with the conditionally approved drug. Patients must enroll and provide informed consent. The sponsor must submit patient data and risk/benefit data to the registry. The Secretary must establish data submission standards and an online portal.

Sec. 2, Informed consent and liability: Patients must provide informed consent before being prescribed a conditionally approved drug. The bill limits liability for sponsors, manufacturers, and health care providers to cases of intentional wrongdoing.

Sec. 2, Reports and transition: The Secretary must report to Congress every two years on conditionally approved drugs. A drug with conditional approval may be granted full approval, and the conditional approval is then automatically withdrawn.