Bringing Patient Experience to Regulatory Decision-Making: Communication Pathway Needed


Takeda’s Jessica S. Scott examines the steps already being taken to bring greater patient engagement to drug development and the regulatory decision-making process in the USA; as well as what more needs to be done in this crucial area.


Steps Taken by FDA

Under the Food and Drug Administration Safety and Innovation Act[1] (FDASIA), the US FDA mandates Patient Engagement (PE) in drug development efforts. FDA has since expanded its PE efforts across all medical products.[2] Furthermore, §1137 of FDASIA, “Patient Participation in Medical Product Discussions”, directs the Secretary of Health and Human Services to:

  • Develop and implement strategies to solicit the views of patients during the medical product development process; and
  • Consider the perspectives of patients during regulatory discussions

FDA has since expanded its PE efforts and encourages the collection of meaningful patient experience data (PED) to augment traditional clinical trial data targeted for regulatory submission. Such expanded authorization came under Section 3001 of the 2016, 21st Century Cures Act, requiring FDA to make a public statement about the PED considered in the approval of a drug application [3]. In its recent Guidance [4]FDA encourages stakeholders to have early interactions with FDA and obtain feedback from the relevant FDA review division when considering collection of patient experience data related to the burden of disease and burden of treatment.”


Industry Momentum

To meet the regulatory mandates mentioned above, many sponsors have embarked on PE efforts that may provide insightful outcomes to inform research and development (R&D) efforts and regulatory submissions. These efforts vary by organization in breadth of adoption, purpose, and methods. Sponsors are seeing value from the activities, however, some inconsistencies and limitations have been observed in the adoption of these regulatory requirements and recommendations in terms of the type of PED used, the context and the rationale.

As shown in a recent analysis of FDA Reported Use of Patient Experience [5], of the 33 new drug approvals where the sponsor-submitted PED was part of the regulatory review, the vast majority of PED (73.2%) were limited to clinical outcome assessments (COAs), mostly consisting of Patient Reported Outcomes (PROs). In contrast. the use of Non-COAs, such as Qualitative Studies, PFDD Stakeholder meeting summary reports, Patient Preference Studies, etc., was very limited. The rationale for the use primarily of COAs (and especially PROs) could be a result of the familiarity and quantitative nature of COAs for both the sponsors and the FDA, while the FDA also tends to consider non-COA less for drug approval reviews.

However, COAs are less likely to provide transparency in patient preferences, for example, in terms of the trial design, where learnings can have a significant impact on future trial design protocols for the R&D pipeline. Having the patient’s perspective in the development of clinical endpoints, quality of life outcome measures, and biomarkers to assess response to therapies, will ensure that sponsors are measuring meaningful outcomes, and that these measures will be acceptable to regulators in assessing clinical benefit to patients.

Related to this, the use of PED is mostly restricted to NDA/BLA submissions. This means that communications between FDA and individual sponsors are generally product-specific, resulting in the outcomes and best practices from company-specific submissions remaining siloed rather than being shared widely. Even when outcomes are shared in the public domain, they are often too late to inform asset development for future products which may already be in the late-stage, R&D pipeline. Early interaction with FDA regarding the collection of PED, prior to the design of clinical trial protocols, is an important example of how PED would inform the development of inclusion/exclusion criteria, clinical endpoints, surrogate endpoints, use of placebo versus active ingredient, enrichment strategies, and other requirements under the 505 (b) and 351 (a) pathways.

However, currently there are no clear pro-active communication pathways and data sharing processes between the sponsor and FDA during the early stages of drug development to better inform the qualitative and/or quantitative PED that should be collected. This knowledge is critical when collecting to inform regulatory decision-making on these issues. Feedback from patients, caregivers, and advocacy groups during R&D could inform regulatory, in addition to internal, decision-making. Indeed, sponsors are likely to engage in more PE activities where the value extends beyond internal use and benefits other stakeholders as well.


Opportunity for Greater Connectivity

By developing a concerted effort that leverages the expertise and best practices from multiple sponsors and stakeholders, there is an opportunity to bridge the gap that exists among FDASIA mandates, 21st Century Cures Act requirements, recommendations within guidances and early product development efforts. In fact, FDA recommends that stakeholders engage with appropriate subject matter experts (e.g., patients, qualitative researchers, survey methodologists, statisticians, psychometricians, patient preference researchers) when designing and implementing studies to evaluate the burden of disease, burden of treatment, and perspectives on treatment benefits and harms [6].

Creating PE efficiencies with streamlined and focused collection of critical PED will also benefit patients. Efficient collection and use of PED will ensure that patients’ experiences are being leveraged in both current and future drug development, particularly since sponsors are the major contributors of PED.

I propose we work collaboratively to improve clarity and create a pathway to bridge this gap to support early, proactive communication. Such an effort could truly reflect the voice of the patient in drug development and regulatory decision-making.



[1] Food and Drug Administration Safety and Innovation Act (FDASIA):

[2] Medical products refer to drugs, biologics, and medical devices

[3] Upton F. H.R. 6—21st Century Cures Act. 114th Congress (2015- 2016) July 13, 2015.

[4] Guidance: “Patient Engagement in the Design and Conduct of Medical Device Clinical Investigations:”

[5] Kieffer et al, FDA Reported Use of Patient Experience Data in 2018 Drug Approvals, DIA, 2019

[6] Guidance: “Patient Engagement in the Design and Conduct of Medical Device Clinical Investigations:”



Illustrative photo by Quang Nguyen Vinh from Pexels

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