Patient Perspectives on Approval Speed vs Evidentiary Certainty in US Cancer Drug Approvals

Area of Science:

• Oncology
• Regulatory Science
• Patient-Reported Outcomes

Background:

• The US Food and Drug Administration's Accelerated Approval pathway allows faster patient access to new cancer drugs.
• This pathway assumes patients will accept uncertainty about clinical benefit for quicker access.
• Little is known about patient perspectives on this trade-off.

Purpose of the Study:

• To explore patient views on the trade-off between faster drug approval and certainty of clinical benefit in the US.
• To understand the conditions under which patients find this trade-off acceptable.

Main Methods:

• Qualitative study using semistructured online interviews.
• Participants: 30 adult patients with breast cancer, recruited via purposive sampling for diversity in cancer stage, treatment status, and demographics.
• Analysis: Thematic analysis of interview transcripts.

Main Results:

• Faster approval was acceptable when no alternative treatments were available or benefits were potentially transformative.
• Patients prioritized survival and quality of life when clinical benefit was uncertain, weighing risks and treatment burdens.
• Many preferred increased access to clinical trials over faster regulatory approval to address unmet needs and generate evidence.

Conclusions:

• A discrepancy exists between patient-acceptability criteria for accelerated approval and current FDA practices.
• Aligning regulatory approvals with patient values may necessitate using accelerated approval primarily for drugs addressing significant unmet needs or offering substantial improvements over existing therapies.