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Incorporating Patient Perspective Into Benefit-Risk Assessments

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Incorporating Patient Perspective Into Benefit-Risk Assessments 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 Wissenschaftliche Prüfungsarbeit zur Erlangung des Titels “Master of Drug Regulatory Affairs“ der Mathematisch-Naturwissenschaftlichen Fakultät der Rheinischen Friedrich- Wilhelms-Universität Bonn vorgelegt von Marleen Lipinski aus Rathenow Bonn September 2020 Betreuerin and 1. Referentin: Dr. Birka Lehmann 2. Referentin: Dr. Ingrid Klingmann Acknowledgements I would like to thank my supervisor and primary advisor Dr. Birka Lehmann for her willingness to supervise and review this master thesis. Thank you very much for your encouraging emails. Also, I would also like to thank my secondary advisor Dr. Ingrid Klingmann for taking over the second review. I would like to thank Dr. Jasmin Fahnenstich for her patience and outstanding support during the journey to the finalisation of this master thesis. A very special thanks goes to Dr. Elmar Wegener for his patience and motivation throughout the entire writing phase of this master thesis. Table of Content List of Figures ......................................................................................................................................... II List of Tables .......................................................................................................................................... II Abbreviation ........................................................................................................................................... III 1 Introduction .................................................................................................................................... 1 2 General Aspects of Patient Participation in Regulatory Decision-Making ...................................... 4 2.1 Concept of Public Participation .............................................................................................. 4 2.2 Patient Participation in Regulatory Decision-Making Processes ........................................... 8 2.3 Potential challenges of Patient Participation ....................................................................... 11 3 Regulatory Framework governing Patient Participation in Benefit-Risk-Assessment in the EU .. 13 3.1 Legal Framework ................................................................................................................. 13 3.2 Overview of Initiatives and Programs at the EMA ............................................................... 15 3.3 Pilot Project on the Involvement of Patients in Benefit-Risk Assessments ......................... 21 3.4 Patient Perspective in the Benefit-Risk Assessment by the EMA ....................................... 22 4 Regulatory Framework governing Patient Participation in Benefit-Risk-Assessment in the US .. 29 4.1 Legal Framework ................................................................................................................. 29 4.2 Overview of Initiatives and Programs at the FDA ................................................................ 32 4.3 Patient-Focused Drug Development Initiative ..................................................................... 34 4.4 Patient Perspective in the Benefit-Risk Assessment by the FDA ........................................ 37 5 Evaluation of Patient Perspective incorporated in the BRA of Medicinal Products ...................... 41 5.1 Objective .............................................................................................................................. 41 5.2 Method ................................................................................................................................. 41 5.3 Results ................................................................................................................................. 43 6 Discussion .................................................................................................................................... 53 References ........................................................................................................................................... 59 Annex .................................................................................................................................................... 72 I List of Figures Figure 1 Key Milestones of EMA Interaction with Patients and Consumers ........................................ 15 Figure 2 Overview of Patient Involvement in EMA Activities and Scope of Representation ................ 17 Figure 3 EMA Regulatory Science to 2025 - 5 Goals for Human medicines Regulation ..................... 20 Figure 4 Statement on Patient Experience Data and Related Information .......................................... 40 Figure 5 Selection Process for Medicinal Products approved by the EMA .......................................... 44 Figure 6 Characteristics: Type of Medicinal Products EMA (n=118) ................................................... 45 Figure 7 Characteristics: Special Regulatory Pathways EMA (n=118) ................................................ 45 Figure 8 Number of SAGs and AHEGs per Year (n=23) ..................................................................... 46 Figure 9 Number of SAGs and AHEGs per Type of Medicinal Product (n=23) ................................... 46 Figure 10 Number of SAGs and AHEGs per Special Regulatory Pathways (n=7) .............................. 47 Figure 11 Selection Process for Medicinal Products approved by the FDA ........................................ 48 Figure 12 Characteristics: Type of Applications FDA (n=137 .............................................................. 49 Figure 13 Characteristic: Special Regulatory Pathways FDA (n=137) ................................................ 49 Figure 14 Number of FDA Advisory Committees and SGE Consultations per Year (n=17) ................ 50 Figure 15 Number of Advisory Committee and SGE Consultations per Type of Application (n=17) ... 50 Figure 16 Number of Advisory Committees and SGE Consultation per Special Regulatory Authorisation (n=32) ............................................................................................................................. 51 Figure 17 Inclusion of PFDD "Voice of the Patient" Reports (n=24) .................................................... 51 Figure 18 Patient Experience Date Statement (Application submitted after June 2017) (n=88) .......... 52 List of Tables Table 1 IAP2's Public Participations Spectrum ...................................................................................... 5 Table 2 Formalized Public Participation Methods .................................................................................. 6 Table 3 EMA´s Level of Stakeholder Involvement ................................................................................. 9 Table 4 Patient Involvement in EMA Activities ..................................................................................... 18 Table 5 Medicinal Products involved in the EMA Pilot to involve Patients in B/R Discussions ............ 21 Table 6 Procedures with SAG or Ad-Hoc Expert Group Involvement .................................................. 24 Table 7 EMA Overview on Assessment Reports ................................................................................. 25 Table 8 Overview on the Content of the European Public Assessment Report ................................... 27 Table 9 Presentation of SAG Involvement in the EPAR - Public Assessment report ........................... 27 Table 10 Initiatives for Patients to Engage With FDA .......................................................................... 33 Table 11 FDA-led Patient-Focused Drug Development (PFDD) Meetings .......................................... 35 Table 12 FDA Benefit-Risk Framework ................................................................................................ 38 Table 13 Patient Perspective in the Framework of Benefit-Risk Assessment ...................................... 39 Table 14 Additional FDA Reports covering special regulatory Grants ................................................. 42 Table 15 Relevant Chapters in the Assessment Reports ..................................................................... 43 Table 16 Search Criteria for the Manual Research .............................................................................. 43 II Abbreviation 21st CC Act 21st Century Cure Act AHEG Ad-Hoc Expert Group BLA Biologics License Applications BRA Benefit-Risk Assessment B/R Benefit-Risk CAT Committee for Advanced Therapies CBER Center for Biologics Evaluation and Research CDER Center for Drug Evaluation and Research CHMP Committee for Medicinal Products for Human Use COMP Committee for Orphan Medicinal Products CTTI Clinical Trials Transformation Initiative DIA Drug Information Association EC European Commission EMA European Medicines Agency ENCePP European Network of Centres for Pharmacoepidemiology and Pharmacovigilance EPAR European Public Assessment Report EU European Union EUPATI European Patients’ Academy on Therapeutic Innovation EURORDIS Rare Diseases Europe FD&C Federal Food, Drug, and Cosmetic Act FDA Food and Drug Administration FDARA Food and Drug Administration Reauthorization Act FDASIA Food and Drug Administration Safety and Innovation Act FY Fiscal Year HTA Health Technology Assessment IAP International Association for
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