Study: Patients favor accelerated approval for oncology drugs without alternatives
Patients with breast cancer said faster approval times for oncology products, as a trade-off for evidentiary certainty, is most permissible in situations where there are no treatment alternatives, the results from a recent qualitative study suggest.
However, the participant answers were more nuanced. Most favored treatments that improved overall survival while also preserving function and quality of life over treatments that with less certain evidentiary benefit and said they were willing to wait for these kinds of treatments to come to market.
“When clinical benefit was uncertain, study participants emphasized survival and quality of life as priority outcomes and weighed the added risks of adverse effects and treatment burden,” Robin Forrest, of the department of health policy at the London School of Economics and Political Science, and colleagues wrote in their study.
The participants also said having greater access to clinical trials may be a better way to bridge the gap for unmet treatment needs, rather than approving oncology products more quickly.
“These findings reveal a mismatch between patient values and the common features of many FDA-approved cancer drugs,” Forrest and colleagues said.
Forrest and colleagues used a semi-structured interview process to ask 30 patients diagnosed with breast cancer about their preferences for oncology products that were approved more quickly than a standard product as a trade-off for products that had more evidentiary certainty. Participants in the study were between 20 years and 60 years old, all were women, and a majority were currently receiving treatment (70.0%) and had received at least one systemic therapy (90.0%), while less than half had metastatic breast cancer (43.3%).
Researchers interviewed participants about their perspectives on waiting and uncertainty, how they would choose between a product with a faster approval as opposed to one with more evidentiary certainty, what treatment outcomes were most meaningful to them, and asked whether they had ideas on ways for patients to receive faster access to treatments.
The researchers found that participants were aware of the US Food and Drug Administration’s (FDA) role in approving drugs but were less clear on the types of approval pathways or the expectations surrounding approval. Participants noted that reliance on physician expertise was important, as even established drugs provided some level of uncertainty for treatment effectiveness.
In terms of waiting and uncertainty, participants said that they preferred uncertainty to having no treatment options. A drug’s anticipated benefit influenced uncertainty, and most participants noted they were willing to accept uncertainty for drugs with a potential greater benefit. When other treatment options were available, participants said certainty became more important. Faster drug approval was not always better in all circumstances, they said, but the participants were split on whether FDA achieved an appropriate balance between faster drug approval and evidentiary certainty. Some participants also drew comparisons to using recently approved drugs with an uncertain clinical benefit and participating in a clinical trial.
“Participants readily understood the trade-off between faster approval and evidentiary certainty, with expectations that largely matched their clinical circumstances,” Forrest and colleagues explained. “Such an understanding contrasts with earlier evidence that patients with metastatic cancer often believe that treatment intent could be curative, which may reflect improved patient education and health literacy, improved access to information in recent years, or comparatively longer survival of patients with breast cancer that allows patients more time to engage with their prognosis.”
The researchers said there needs to be more information clearly communicating uncertainties of approved oncology drugs in light of these findings, which might also improve clinician decision-making.
“The FDA should consider reclassifying existing accelerated approvals as available therapy, thereby limiting pathway eligibility to drugs that genuinely address unmet needs,” researchers said.”
