Recent research from the London School of Economics and Political Science (LSE), highlighted in The Lancet Oncology, suggests that patients with cancer may prefer to wait longer for new cancer drugs to be approved, provided there is greater certainty regarding their effectiveness. This publication addresses critical concerns surrounding the U.S. Food and Drug Administration's (FDA) accelerated approval pathways.

The Dilemma of Drug Approval

When considering new cancer drugs, the FDA faces a challenging trade-off: whether to prioritize speed in drug approvals or to delay in order to confirm a drug's clinical benefits. In recent years, the accelerated approval process has faced significant criticism due to its implications for patient outcomes. Evidence indicates that a considerable number of drugs approved through this pathway do not extend the lives of cancer patients.

Study Overview

The LSE researchers conducted a study involving over 850 participants across the United States, all of whom had either been diagnosed with cancer or had a close relation who was affected by it. The participants were asked to make hypothetical choices concerning various cancer drugs, allowing the researchers to model their preferences and determine the extent of their willingness to wait for greater certainty concerning the clinical benefits of these medications.

Key Findings

  • Participants expressed a willingness to wait up to 22 additional months to ensure a drug is effective before FDA approval.
  • Those with personal experiences with cancer favored faster drug access when alternative treatments were limited.
  • However, many indicated a preference for rigorous evaluation over expedited access if it guaranteed greater effectiveness.

Perspectives from Researchers

As noted by Robin Forrest, the lead author of the study, “People with experience of cancer value faster access to new cancer drugs, particularly when they have limited treatments available. But when faster access comes at the expense of certainty that a drug extends survival, then this might not always be the case.”

Dr. Huseyin Naci, the study's senior author, emphasized that “these results show that patients are not willing to access new cancer drugs at any cost,” indicating that some respondents would favor extended wait times for robust clinical evidence instead of immediate approval.

Recommendations for Future Approvals

Given the findings of this study, the authors advocate for:

  • Increased transparency: Providing clearer justifications for the use of accelerated drug approvals.
  • Focus on patient-centered outcomes: Placing greater emphasis on the unique preferences and experiences of patients in the drug approval process.

The Importance of Meaningful Treatment

Professor Ajay Aggarwal, co-author of the study and a Consultant Clinical Oncologist, remarked on the importance of providing treatment options that genuinely improve survival rates and quality of life. He reiterated the need for a robust evaluation process to ensure that patient hopes and expectations align with the actual benefits offered by new drugs.

“As an oncologist, providing new treatment options for patients with cancer that offer meaningful improvements in survival and quality of life is critical, however expediency should not come at the cost of robust evaluation especially when we know many drugs often fail to offer the benefits that patients themselves hope for.” – Professor Ajay Aggarwal

Conclusion

In summary, this study sheds light on the complex relationship between drug approval processes and patient preferences in oncology. Patients exhibit a willingness to trade waiting time for certainty of treatment efficacy, highlighting the need for FDA practices that prioritize patient interests while ensuring safety and effectiveness.

References

For further reading, please refer to the study: Robin Forrest et al, Preferences for speed of access versus certainty of the survival benefit of new cancer drugs: a discrete choice experiment, published in The Lancet Oncology (2024).

[1] Lifespan.io