Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Incorporating Patient Perspective into Benefit-Risk Assessments of a Medicinal Product - An Inventory of the Regulatory Approaches established in the European Union and the United States ***

Marleen Lipinski (Abschlußjahr: 2020)

Summary
Language: English
Regulators worldwide consider it increasingly important to include the patient perspective in the benefit-risk assessment to inform regulatory decision-making. It is widely acknowledged that patients suffering from a disease may weigh risks differently than a regulator who takes a decision based on the scientific data provided by the applicant. More recently, the EMA and the FDA have implemented various programs and initiatives with the aim to enhance the interaction with patients and to incorporate patient’s perspectives and preferences into the regulatory decision-making process.
The present master thesis provides an inventory of regulatory approaches by the EMA and the FDA to incorporate patient perspectives in the benefit-risk assessment process of a medicinal product. It further includes a theoretical model for public participation which is presented in order to establish an interconnection between patient participation and regulatory decision-making processes. The potential challenges associated with the inclusion of patient perspectives in the benefit-risk assessment are highlighted. EPARs published on EMA´s website and review documents published on FDA´s website for medicinal products were assessed to

1. identify the type of methods which are primarily used by both agencies to involve patients in benefit-risk assessment process.
2. understand for which type of medicinal products the EMA and FDA primarily consider the involvement of patients
3. analyze how transparent patient involvement has been documented in regulatory assessment reports which are publicly available.

It was found that both agencies primarily consult with patient representatives either in their function as a member of a Scientific Expert Group or as an Ad-hoc Expert Group at the EMA or via Advisory Committees requested by the FDA. In 19% (n=23) of the cases analyzed, the EMA requested feedback from the Scientific Expert Group or an Ad-hoc Expert Group and the majority of the medicinal products did not have a special regulatory designation. However, there remain uncertainties due to limited transparency. In only 9 of 23 cases patient representation was documented in the public assessment report. The FDA requested feedback from an Advisory Committee concerning the respective medicinal product in only 10% (n=14) of the cases. Patient representatives were involved in all identified Advisory Committees. In the U.S., the vast majority of the products analyzed qualified for priority review.
As a final outcome of this thesis, it can be confirmed that both agencies, EMA and FDA engage with patients during the benefit risk assessment process of medicinal products. However, there is still limited documentation available concerning these interactions. Based on the findings in this thesis, it can be concluded that the FDA appears to be providing more structured information concerning patient participation compared to EMA.
Pages: 58
Annexes: 02, Pages: 22