In the JournalsPerspective

Physicians lack clarity on FDA approvals, ‘breakthrough therapy’

Physicians demonstrated limited understanding of the FDA approval process and the meaning of the “breakthrough therapy” designation, according to recent research in JAMA.

“Although the term ‘breakthrough’ leads consumers to overly optimistic beliefs about drug effectiveness, it is not known how physicians understand this term — or more generally, what FDA approval means,” Aaron S. Kesselheim, MD, JD, MPH, of the division of pharmacoepidemiology and pharmacoeconomics at Brigham and Women’s Hospital, and colleagues wrote. “A national survey of board-certified internists and specialists revealed substantial deficits in knowledge of the meaning of FDA approval.”

The researchers surveyed a cohort of 1,148 physicians, contacted through the American Board of Internal Medicine’s diplomate list. The survey included three questions about FDA approval and five questions about breakthrough therapies. The researchers also included a hypothetical situation, in which physicians were asked to choose between prescribing two new drugs, one of which was labeled “breakthrough therapy,” while, in fact, both met the criteria for breakthrough therapies. Physicians also were randomly selected to give their opinions on one of four mock FDA press releases for a hypothetical drug.

Study results showed that 73% of respondents mistakenly believed that FDA approval meant that the drug’s effectiveness was comparable to that of other approved drugs, while 70% mistakenly believed that FDA approval required a statically significant and clinically important outcome. Likewise, 52% of respondents incorrectly believed that drugs labeled as breakthrough therapy required randomized trials to claim the designation.

Furthermore, 94% of respondents preferred the hypothetical drug that was labeled as a “breakthrough therapy” over the nonlabeled version.

“The misconceptions identified may lead physicians to overprescribe newly approved drugs — particularly breakthrough therapies — and inadequately communicate how well these drugs work to the patients who will use them,” Kesselheim and colleagues wrote. – by David Costill

Disclosure: Kesselheim reports no relevant financial disclosures. Please see the full study for a list of all other authors’ relevant financial disclosures.

Physicians demonstrated limited understanding of the FDA approval process and the meaning of the “breakthrough therapy” designation, according to recent research in JAMA.

“Although the term ‘breakthrough’ leads consumers to overly optimistic beliefs about drug effectiveness, it is not known how physicians understand this term — or more generally, what FDA approval means,” Aaron S. Kesselheim, MD, JD, MPH, of the division of pharmacoepidemiology and pharmacoeconomics at Brigham and Women’s Hospital, and colleagues wrote. “A national survey of board-certified internists and specialists revealed substantial deficits in knowledge of the meaning of FDA approval.”

The researchers surveyed a cohort of 1,148 physicians, contacted through the American Board of Internal Medicine’s diplomate list. The survey included three questions about FDA approval and five questions about breakthrough therapies. The researchers also included a hypothetical situation, in which physicians were asked to choose between prescribing two new drugs, one of which was labeled “breakthrough therapy,” while, in fact, both met the criteria for breakthrough therapies. Physicians also were randomly selected to give their opinions on one of four mock FDA press releases for a hypothetical drug.

Study results showed that 73% of respondents mistakenly believed that FDA approval meant that the drug’s effectiveness was comparable to that of other approved drugs, while 70% mistakenly believed that FDA approval required a statically significant and clinically important outcome. Likewise, 52% of respondents incorrectly believed that drugs labeled as breakthrough therapy required randomized trials to claim the designation.

Furthermore, 94% of respondents preferred the hypothetical drug that was labeled as a “breakthrough therapy” over the nonlabeled version.

“The misconceptions identified may lead physicians to overprescribe newly approved drugs — particularly breakthrough therapies — and inadequately communicate how well these drugs work to the patients who will use them,” Kesselheim and colleagues wrote. – by David Costill

Disclosure: Kesselheim reports no relevant financial disclosures. Please see the full study for a list of all other authors’ relevant financial disclosures.

    Perspective
    Richard F. Jacobs

    Richard F. Jacobs

    The recent JAMA paper studying the level of understanding and the impact of a compound being approved by the FDA as a “breakthrough therapy” demonstrates a heightened need for better communication and education in medicine on the approval process for drugs through the FDA. This designation does not imply or support any evidence based trials or study data on the effectiveness of these new compounds compared to traditional, long used or much less expensive drugs or treatments. It also does not provide any comparative data on safety. These designations are further extremely limited to those of us who treat children. Better educational venues and communications with practicing physicians is needed so the highly informed public really understands these processes when they visit their physicians looking for answers and informed dialogues.

    • Richard F. Jacobs, MD, FAAP
    • Infectious Diseases in Children Chief Medical Editor

    Disclosures: Jacobs reports no financial disclosures.