Pharmacists' Knowledge and Perceptions of FDA Approval Standards and the Breakthrough Therapy Designation

Area of Science:

• Pharmacology
• Regulatory Science
• Health Outcomes

Background:

• The United States Food and Drug Administration (FDA) established the breakthrough therapy designation (BTD) to accelerate access to drugs for unmet medical needs.
• Understanding healthcare professionals' knowledge of regulatory pathways is crucial for effective drug implementation.

Purpose of the Study:

• To assess pharmacists' knowledge of FDA drug approval standards.
• To evaluate pharmacists' awareness and perceptions of the BTD.
• To identify potential knowledge gaps in understanding expedited drug approval mechanisms.

Main Methods:

• A questionnaire was distributed to pharmacists in advanced clinical practice identified through a professional pharmacy organization.
• The survey assessed knowledge of FDA approval standards and the BTD.
• Data from 226 respondents were analyzed.

Main Results:

• While most pharmacists answered questions about FDA approval standards (58.1%) and BTD criteria (78.1%) correctly, only 24.1% reported familiarity with the BTD.
• A majority perceived breakthrough drugs as major advances (62.8%) and preferred them hypothetically (88.5%).

Conclusions:

• Pharmacists demonstrate adequate knowledge of FDA approval standards and BTD criteria but lack awareness of the designation's implications.
• There may be an overestimation of the benefits of drugs with a BTD among pharmacists.
• Future research should focus on pharmacist understanding of regulatory mechanisms for expedited drug approval.