Curious about Patient Experience Data (PED) and its role in regulatory approvals? As highlighted in a recent scientific publication and industry survey, Real World Data (RWD), and Patient Experience Data (PED), in addition to classic clinical trial data, are sometimes necessary to complete medical registration files.

However, they can pose challenges for companies seeking approvals. Authorities like the Food and Drug Administration (FDA) in the US or the European Medicines Agency (EMA) in Europe are increasingly demanding these types of data to approve products or confirm conditional approval, emphasizing the importance of addressing patients’ needs. 

1. From Patient Experience Data (PED) to Real-World Evidence (RWE)

A. What is Patient Experience Data (PED)? 

Patient Experience Data (PED) refers to information gathered from patients. Various methodologies collect these data, such as capturing patients’ insights into their health status, symptoms, disease progression, treatment preferences, quality of life, or the impact of healthcare through validated patient-reported outcomes that are quantitative data. Qualitative information can also be obtained through focus groups, surveys, or interviews to gain a broader understanding of patients’ experiences. 

 B. What is Patient Reported Outcomes (PRO)?  

Patient Reported Outcomes (PRO) refer to measures based on a report directly from patients, without interpretation by a clinician or anyone else, about the patient’s health condition. 

C. What is Real-World Data (RWD)? 

Real-World Data (RWD) comprises information about patient health from everyday medical practice. Various sources, such as electronic health records, medical claims, information from product or disease registries, and data acquired through digital health technologies, contribute to RWD. All these sources share the commonality of being obtained in real-life situations. 

D. What is Real Word Evidence (RWE)?  

Real-World Evidence (RWE) refers to clinical proof obtained outside conventional clinical trials through the analysis of RWD regarding the utilization, advantages, or risks of a medical product. 

From a regulatory decision-making point of view, those data about patient health from everyday life are very important because they allow to gain more insights about the patient’s perspective and point of view but also it can be interesting in terms of safety information for instance.    

Today, health authorities’ communication on the need of those patient data is improving but not everything is yet clear. 

2. Insights from “Current Practices and Challenges When Submitting Patient Experience Data for Regulatory Decisions by the US Food and Drug Administration: An Industry Survey” methodology, results, and perspectives.   

A study, published in December 2023, involved 50 participants from the industry (84%) and contract research organizations (16%) to understand practices and challenges related to the submission of patient experience data. 

 A. Study methodology

Conducted by a pharmaceutical industry research team, the study underwent pretesting with ten industry and Contract Research Organization (CRO) members to assess readability and comprehension. It was then distributed to large professional special interest groups. 

The study consisted of an online survey with two parts: one focused on the gathering, submission, and utilization of PED by the FDA in regulatory decision-making, and the second part was a best-worst exercise to identify potential PED initiatives. Eligibility criteria for participants were based on their experience with PED, requiring more than 2 years of experience. 

 B. Study results

The study indicates that to enhance the gathering and submission of high-quality PED to the FDA for regulatory decision-making, improving dialogue between the FDA and the industry is essential.   

It remains unclear to applicants for approval what information is precisely expected at the regulatory level. As not all PED are significantly important in regulatory decision-making, it is the industry’s role to emphasize the significance of their data, such as patient preferences. Improving dialogue with the FDA is deemed useful for achieving this.   

Meeting the FDA more frequently and the availability of expanded FDA guidance on the development of PED or Clinical Outcome Assessments would also be beneficial. 

 C. Main challenges highlighted 

The challenges mostly cited in PED collection was:  

• The difficulty of justifying the effort involved in collecting that data without being sure of the agency’s utilization of such data in regulatory decision-making.  

• The lack of harmonized process to understand what is needed by the FDA or EMA for instance is also a challenge, a sort of PED evidentiary standards would be useful.  

Participants of the survey also considered that the agency should justify the reasons for not using PED data when they are provided by the applicant. The participants recommend to the authorities to provide a sort of template with categories such as “data not fit for purpose”, “data incomplete” or “not addressing the regulatory decision”, “lack of data quality”, to be aware of the expectancies of the agencies.  

D. Future priorities and goals  

Developing guidance on the development of PED and a dedicated meeting pathway are among the highest priorities according to the study results, because being able to create and consequently utilize more helpful PED is important. Plus, according to the study participants PED should be considered as an integral component such as efficacy or safety data which is in line with authorities’ expectations. 

3. How to answer the agencies’ requests of gathering and submitting high-quality PED or RWD ?  

The role of patient-centered data is crucial for regulatory agencies, despite some improvements needed in terms of the use of PED and RWE information. 

 A. Solutions for obtaining patients data 

Various solutions exist for acquiring patient data as requested by health authorities. Companies, such as CROs specialized in generating such data through studies conducted directly with patients, offer viable options. Carenity, with over a decade of expertise, is one of these companies dedicated to meeting agencies’ requests for gathering and submitting high-quality PED or RWD.   

In collaboration with the industry, in high-prevalence and rare diseases, Carenity conducts studies to provide significant data, such as patient preferences or treatment preferences. Many results from Carenity’s studies, part of various day-to-day projects, are regularly submitted to health authorities and can prove valuable in completing submission dossiers. 

 B. Practical use of PRO  

The utilization of validated Patient Reported Outcomes (PRO), a current practice at Carenity, is also noteworthy for regulatory agencies. PRO reveals patients’ perspectives without biases from healthcare professionals. Additionally, studies can be conducted to define value in health, identify disease burden and unmet needs, or gain patient perspectives through discrete choice experiment studies. Carenity offers real-world studies that complement pivotal clinical data for submission for reimbursement. Beyond quantitative data, Carenity can provide qualitative data through interviews and their analysis. 

4. Conclusion  

Regulatory agencies express a desire to enhance the utilization of Patient Experience Data (PED), despite existing obstacles in transparency within regulatory decision-making. It is crucial to persist in efforts and consistently provide agencies with both PED and Real-World Data (RWD). Companies possessing the capability to generate such data, such as Carenity, emerge as dependable partners for the industry in meeting the regulatory agencies’ demand for high-quality data. 

Sources:  

https://www.ema.europa.eu/en/documents/other/executive-summary-patient-experience-data-eu-medicines-development-and-regulatory-decision-making-workshop_en.pdf 

https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence 

https://link.springer.com/article/10.1007/s40271-023-00653-8#Sec2  

https://www.fda.gov/media/124063/download

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