Increased Patient Engagement by FDA and Its Impact on Industry
​[Posted on: Thursday, March 16, 2017]

This week FDA announced its plans to open a new Office of Patient Affairs (OPA) to enhance future patient engagement by providing a more transparent, accessible, and robust experience for patient communities. However, how will this office impact regulatory decisions is not clear. According to the notice in the Federal Register, this idea came about from comments received by FDA from patients. This adds to several other initiatives FDA implemented in recent years to collect patient perspectives such as Patient Focused Drug Development, Patient Representative Program, and the Patient Engagement Advisory Committee. Most of the patient-centric initiatives will likely come under the new OPA. One may wonder, what impact, if any, does such patient engagements at FDA, have on the products regulated by it or the developers of such products. In recent years, a few controversial product approval decisions were made based on robust patient engagement by the companies. This has lead to companies using patient engagement as a critical component of their product approval strategy. In recent years, thanks to social media, patients have also become very vocal in airing their opinions about regulatory processes. Patient support groups frequently attend FDA’s advisory committee meetings for related products, and even fund development of new products. So, this new initiative by FDA should help consolidate all these patient efforts. While this may be new for the FDA, such initiatives are not new in the regulatory domain. The European Medicines Agency (EMA) have had active patient engagement programs for more than 12 years called the Patients’ and Consumers’ Working Party (PCWP) with very similar mandate as FDA’s new initiative. In addition, EMA has a separate program for engaging physicians and other healthcare providers called the Healthcare Professionals Working Party (HCPWP) which engages prescribers. Members of both the PCWP and HCPWP include most patient and professional organizations, respectively, along with EMA administrators and reviewers. PCWP and HCPWP meet 4-5 times each year to discuss various topics of interest. EMA reviewers participate in these meetings to understand the perspectives of the stakeholders. From the EMA experience, one can learn that patient engagement fills an important void at the regulatory agency and helps humanize regulatory decisions. However, their direct impact on regulatory decisions is not clear. For sure companies developing products of interest to a given community, use these communities to build public support for their products both for regulatory approval and future market access. However, regulatory decisions are primarily based on the data generated by the sponsor. Patient engagement can only play a supportive role at best. This may change a bit due to the current political environment that favors lowering the threshold of product approval. Such lowering of regulatory requirements may be balanced by the increase in patient advocacy for products that have a higher perceived benefit to the patient even if the statistical data is not strong enough for direct FDA approval.