In June 2021, the United States Food and Drug Administration (FDA) published a 60-day notice requesting public comment on the proposed collection of information, announcing its plan to conduct two studies regarding patient understanding of accelerated approvals. Recently, on March 24, 2022, the agency released another proposed collection of information, reaffirming its intent to move forward and addressing some of the concerns raised by Industry in response to the June 2021 proposal.
In August 2021, the Pharmaceutical Research and Manufacturers of America (PhRMA) sent in a comment in response to the proposal, applauding the general intent of the FDA to study consumer understanding of accelerated approvals but simultaneously voicing concerns over the specifics of the planned course of action. PhRMA believes that FDA should focus more on communications between healthcare professionals and patients and less on promotional materials.
The March 24, 2022, announcement is essentially a duplication of the June 2021 proposal with respect to the background and plans for the upcoming studies. However, it also includes responses to PhRMA’s letter. For one, PhRMA had concerns over whether the proposed research would duplicate previous work by the FDA and requested that the agency publish the full results of the prior studies before beginning work on these proposed studies. FDA noted that the agency does “not plan to duplicate the prior research, although there often is value in that undertaking. Rather, the present research seeks to replicate the previous study in a new patient population and extend the previous study by testing additional versions of the disclosure.” FDA also noted that by focusing on what cancer patients and their caregivers gather from consumer information about accelerate approvals can be greatly beneficial.
PhRMA also raised concern over risks associated with overemphasizing accelerated approval and that “new required disclosures” might be a deterrent to patients seeking treatment. In response, the FDA said that the proposal does not “establish a mandate or propose a new rule,” but instead is just a data collection for research.
As noted above, PhRMA suggested the agency modify the plan to focus more on prescriber-patient communication, not direct-to-consumer promotions and advertisements, noting that the shared decision-making and conversations with healthcare providers is an important part of the therapeutic process. FDA responded, acknowledging the importance of prescriber-patient interaction, but reiterated its position that direct-to-consumer messaging can help shape those interactions and therefore, the agency will focus study on that.
PhRMA further suggested that the FDA screen study participants for patients with acute lymphoblastic leukemia (ALL) who have actually received accelerated approval products. However, FDA notes that patients are not likely to know whether their therapy was approved under an accelerated approval pathway but the FDA will explore allocating quotas for “a broad range of cancers” in its recruitment efforts, and might restrict enrollments to patients who receive systemic therapies.
PhRMA also suggested that study participants be told that the drug in question has received accelerated approval (and the regulatory basis for this type of approval), so they can understand the disclosures. However, the FDA noted that “[c]onsumers encountering DTC websites for accelerated approval products would not have this background information, so giving this information to participants would defeat the purpose of testing what perceptions these consumers form from the website disclosures.”
Next Steps
The FDA plans to proceed with conducting the studies. However, because the research is the very beginning of the path to potential policy changes, any changes are likely years away.
Any comments or recommendations in response to the proposal should be submitted no later than April 25, 2022.