Evolving Landscape of US Food and Drug Administration Drug Approval in the Era of Precision Oncology: Finding the Right Balance Between Access and Safety
- Balance is needed between enhancing drug access and ensuring patient safety.
- Accurate surrogate endpoints and sophisticated trial designs are crucial for predicting clinical benefits.
- Safety data before final approvals and postapproval assessments for vulnerable populations are necessary.
- Value-based pricing is important to reduce financial burdens on patients.
- The FDA must protect patients from adverse effects while ensuring timely access to therapies.
- Ongoing evaluations of expedited approval models and stakeholder collaboration are essential.
Since 1992, the US Food and Drug Administration (FDA) has implemented several programs for streamlined review and approval of agents that treat serious or life-threatening conditions, including the Accelerated Approval Pathway as well as designations for Priority-Review, Fast-Track, and Breakthrough Therapies.
Accelerated approval is based on surrogate end points considered to have a reasonable likelihood of leading to overall clinical benefit, and it is conditional on confirmatory postapproval trials. The trial end points used to determine long-term clinical benefit depend on the specific disease setting and on the availability of effective alternative treatments. Despite limited evidence of clinical benefit and safety, agents that receive accelerated approval enter the market as FDA approved products available for clinical use, contingent on provider, patient, and payer concurrence.
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