An initiative by the U.S. Food and Drug Administration aimed at granting patients quicker access to promising oncology treatments appears to falter when it comes to long-term benefits. The accelerated approval program may allow cancer medications to reach the market faster, but new evidence questions their effectiveness in the long run.
Recent research indicates that a significant number of cancer drugs, which had received accelerated approval, fail to show substantial improvement in patient outcomes within five years.
Dr. Ezekiel Emanuel, a University of Pennsylvania cancer specialist and bioethicist not associated with the study, states, “Five years post accelerated approval should yield conclusive results.” He highlights the dilemma of prescribing drugs without verified efficacy to numerous patients.
Originally introduced in 1992 primarily for HIV medications, the program now primarily serves oncology drugs, accounting for 85% of the program’s approvals.
The accelerated approval pathway permits the FDA to green light drugs with preliminary positive data for severe or life-threatening conditions. In return, pharmaceutical companies are expected to conduct thorough testing to obtain full approval subsequently.
While patients benefit from earlier access to drugs, it comes at the cost of potential inefficacy. The task of withdrawing ineffective drugs falls to the FDA or the pharmaceutical companies, and at times, the FDA accepts less robust evidence for complete approval.
Findings of the new study reveal out of 46 cancer drugs receiving accelerated approval from 2013 to 2017, 63% advanced to regular approval despite only 43% demonstrating clinical benefit in follow-up trials.
Published in the Journal of the American Medical Association, the study was also a topic at the American Association for Cancer Research annual meeting in San Diego.
The study’s co-author Dr. Edward Cliff of Harvard Medical School emphasizes the uncertainty surrounding these drugs and the importance of communicating this to cancer patients.
Accelerated approval drugs often represent the sole therapeutic option for individuals with rare or advanced-stage cancers, explains Dr. Jennifer Litton from MD Anderson Cancer Center, who did not participate in the study. Litton underscores the necessity for clinicians to explain the evidence without creating false hopes.
Congress has recently revisited the accelerated approval process, granting the FDA greater power and simplifying drug withdrawal procedures when pharmaceutical firms do not fulfill obligations.
These amendments mean the FDA “can expedite the process to revoke an accelerated approval drug when required,” explains FDA representative Cherie Duvall-Jones via email. The FDA now mandates that confirmatory trials start at the time of initial approval, thus accelerating the confirmation process.
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FAQ Section
- What is the accelerated approval program?
The accelerated approval program is an FDA initiative that allows for earlier approval of drugs that show potential benefit for serious or life-threatening diseases based on preliminary evidence. This program was established to ensure quicker access to critical medications for patients in need. - What are the requirements for full approval after receiving accelerated approval?
After a drug receives accelerated approval, the pharmaceutical company must conduct additional rigorous testing to prove the drug’s clinical benefit. If the drug meets the necessary criteria, it can obtain regular, full approval from the FDA. - Can drugs that receive accelerated approval later be withdrawn?
Yes, the FDA or the drug company must withdraw drugs that do not prove to be effective or show clinical benefit in the post-approval period. Recent legislative changes have made it easier for the FDA to expedite the withdrawal process if needed. - Why might there be cause for concern with the accelerated approval of cancer drugs?
There is concern because research shows that many drugs granted accelerated approval do not confirm clinical benefits within five years. This raises questions about the therapeutic value of these drugs and the information conveyed to patients regarding their potential benefits.
Conclusion
The accelerated approval program by the FDA was conceived with the intention of offering hope to patients with life-threatening diseases by delivering treatment options more swiftly. However, as recent studies have suggested, the efficacy and benefit of many cancer drugs that have received accelerated approval remain unproven after several years on the market. As the FDA and pharmaceutical companies face ongoing scrutiny, it is critical for them to ensure that the drugs’ potential benefits outweigh the risks and uncertainties, adequately conveying this information to patients. Moreover, the recent changes to the approval process could signal a more stringent, transparent, and rapid response to drugs that fail to meet their initial promise.