Academic development of ATMPS in Belgium : an exploratory study of the legal and strategic options

Abstract

139 p.ill.,LIST OF FIGURES .4 -- LIST OF TABLES 4 -- LIST OF ABBREVIATIONS 5 -- SCIENTIFIC REPORT .7 -- GLOSSARY 7 -- 1 INTRODUCTION 10 -- 1.1 COMPLEX MEDICINAL PRODUCTS 10 -- 1.2 HIGH HOPES AND SCIENTIFIC UNCERTAINTIES 10 -- 1.3 COSTLY MEDICINES AND LIMITED ACCESS .10 -- 1.4 CHALLENGING LEGAL ENVIRONMENT .11 -- 1.5 DIFFICULT TRANSLATION TO THE PATIENT .12 -- 1.6 EMERGING FUNDING AND BUSINESS MODELS .12 -- 2 AIM, SCOPE & LIMITATIONS 13 -- 3 METHODOLOGY 14 -- 4 CHAPTER 1 - LEGAL FRAMEWORK FOR ATMPS IN EUROPE 15 -- 4.1 DEFINITION AND APPLICABLE LEGAL FRAMEWORK 16 -- 4.2 DIFFERENTIATION WITH REGULATIONS ON HUMAN TISSUES AND CELLS 18 -- 5 CHAPTER 2 - ATMP LANDSCAPE IN BELGIUM 21 -- 5.1 ACCESS TO ATMPS 21 -- 5.1.1 Via (reimbursed) centrally authorised ATMPs 21 -- 5.1.2 Via clinical trials .28 -- 5.1.3 Via Hospital Exemption .32 -- 5.2 GMP MANUFACTURING CAPACITY IN BELGIUM .32 -- 6 CHAPTER 3 - AUTHORISATION ROUTES FOR ATMPS 35 -- 6.1 INTRODUCTION .36 -- 6.2 CLINICAL TRIALS 36 -- 6.2.1 Challenges and consequences .37 -- 6.2.2 Existing support mechanisms and possible improvements 39 -- 6.3 CENTRALISED MARKETING AUTHORISATION .40 -- 6.3.1 Challenges and consequences .41 -- 6.3.2 Existing support mechanisms and possible improvements 42 -- 6.4 HOSPITAL EXEMPTION 43 -- 6.4.1 EU framework 43 -- 6.4.2 Challenges and consequences (BE) 47 -- 6.4.3 Existing support mechanisms and possible improvements 55 -- 6.5 DIFFERENCE WITH OTHER EXCEPTIONS 57 -- 7 CHAPTER 4 - EMERGING CASES OF ACADEMIC DEVELOPMENT PATHWAYS IN EUROPE 57 -- 7.1 INTRODUCTION .58 -- 7.2 TUMOR INFILTRATING LYMPHOCYTES (TILS) IN ADVANCED MELANOMA – THE NETHERLANDS (AND DENMARK) 58 -- 7.3 CAR-T CELLS FOR ALL - SPAIN 60 -- 7.4 STRIMVELIS GENE THERAPY FOR ADA-SCID - FONDAZIONE TELETHON ETS (ITALY) 66 -- 7.5 LESSONS LEARNED FROM THE CASE STUDIES 66 -- 7.5.1 Strimvelis 66 -- 7.5.2 ARI-001 and TILs .67 -- 8 CHAPTER 5 - STRATEGIC AND LEGAL OPTIONS FOR THE ACADEMIC DEVELOPMENT OF ATMPS 68 -- 8.1 INTRODUCTION .69 -- 8.2 PUBLIC FUNDING: NECESSITY AND APPROACHES .70 -- 8.2.1 Types of public funding for ATMP projects .70 -- 8.2.2 Funding conditions to ensure accessibility and affordability and social responsibility 72 -- 8.2.3 State aid 74 -- 8.2.4 Other competition law concerns 83 -- 8.3 INTELLECTUAL PROPERTY STRATEGY 85 -- 8.3.1 Introduction .85 -- 8.3.1 Patentability of cell-and gene based technologies 85 -- 8.3.2 IP strategies under the academic pathway .90 -- 8.4 TRANSLATIONAL EXPERTISE 91 -- 8.5 MANUFACTURING FACILITIES AND EXPERTISE .95 -- 8.5.1 ATMP manufacturing is complex .96 -- 8.5.2 Automatisation in the manufacturing and decentralisation of production 97 -- 8.5.3 ATMP manufacturing is costly 100 -- 8.5.4 Manufacturing routes for Academia .100 -- 9 CHAPTER 6 - DISCUSSION AND CONCLUSIONS 102 -- 9.1 DISCUSSIONS WITH EXPERTS AND STAKEHOLDERS .102 -- 9.2 CONCLUSIONS ON THE LEGAL AND STRATEGIC OPTIONS FOR ACADEMIC DEVELOPMENT OF ATMPS IN BELGIUM 107 -- APPENDICES 109 -- APPENDIX 1. ATMP LANDSCAPE 10APPENDIX 1.1. OVERVIEW OF ATMPS ON THE EUROPEAN MARKET SINCE 2009 TILL SEPTEMBER 2024 .109 -- APPENDIX 1.2. CLINICAL TRIALS ACTIVITY WITH ATMPS IN BELGIUM 112 -- APPENDIX 1.3. CLINICAL TRIAL PATHWAY OF AUTHORISED ATMPS, IMPLICATION OF ACADEMIA 120 -- APPENDIX 1.4. DESIGN FEATURES OF PIVOTAL CLINICAL TRIALS FOR THE APPROVED ADVANCED THERAPY MEDICINAL PRODUCTS IN THE EU 121 -- APPENDIX 2. STATE AIDS DECISION-THREE 123 -- APPENDIX 3. COMPASSIONATE USE AND SPECIAL NEEDS FOR ATMPS 124 -- REFERENCES 127