What Do Patients Want?

Thirty years ago, HIV advocates elevated the notion of patient involvement in the development of new medicines to entirely unprecedented levels by demanding input into multiple aspects of clinical development of HIV treatments.  That included not only in the way that drug companies conducted trials, but in the way FDA evaluated them. The result was the development of enhanced pathways for approval and faster development. Recently there has been renewed effort to more broadly bring the patient voice into greater play. In fact, being “patient-centeredness” has become a common goal along the entire healthcare spectrum – from the provision of healthcare services to the comparison of medical treatments to considerations in developing new medicine.

FDA is getting on board. In a March 2017 Federal Register Notice,  FDA announced that the agency was opening a public docket to solicit input into the creation of an Office of Patient Affairs within the agency. This is not like it was three decades ago. There are a good number of highly active patient organizations that are involved in a spectrum of disease and condition-specific advocacy. There are diseases that are common such as cancer, a rare such as Duchenne muscular dystrophy. In other words, today patient involvement means a greater volume and range of involvement and issues if any organization is truly seeking to be more patient-centric. That naturally leads to the question – what do patients want?

RX4Good is an organization uniquely suited to finding out. Formed in 2009 the organization is aimed at helping companies not only achieve a greater understanding of patient need, but how they can respond thoughtfully and strategically to it. This month, RX4Good issued a report that compiled the results of a survey of patient organizations to assess what the priorities should be for FDA in developing an Office of Patient Affairs. The report contains a good deal of specific information, but also lays out the top three priorities identified by patient groups as FDA moves forward.

The most immediate priority for FDA’s new office expressed by 4 out of 5 the organizations surveyed was the desire for patients to have a seat at the table in the clinical development phase, particularly so that clinically meaningful endpoints translate into meaningful patient outcomes and to minimize the outcome that a trial may have a limited value to patients.

The second most relevant goal cited by just over half of the respondents was for the support of expanded access programs. And just behind this was expediting recruitment and the sharing of outcomes data.

The third area of concern was for the generation of greater amounts of communication and information.

In fact, there are currently a number of ways that patient organizations can interact with the FDA process – such as participation on an FDA advisory committee or workshop.  But it is another noteworthy aspect of this report that a clear majority of the advocates surveyed have not participated in FDA workshops or advisory committees.  While there was some awareness of patient-centered efforts on behalf of the agency, only a small percentage felt that they were very familiar with them. That means the agency has its work cut out on the communications and outreach front – the “if you build it they will come” approach won’t be enough.

You can download a full copy of the report at the RX4Good Website to see the results to the specific questions posed by the organization to patient groups. Clearly there are no quick fixes, but as the report points out, there are plenty of opportunities for FDA to capitalize on what it has already started with respect to patient centeredness while the agency takes in more information and suggestions through the docket on what it still needs to do.

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