Orphan medicines legislation General pharmaceutical legislation 7 May - 30 July 2021 28 Sept. - 21 Dec. 2021 303 answers including 12 public authorities from 9 Member States* 478 answers including 31 public authorities from 15 Member States** IMPROVING ACCESS TO INNOVATIVE MEDICINES: IMPROVING ACCESS TO INNOVATIVE MEDICINES: PROPOSALS FROM NATIONAL AUTHORITIES PROPOSALS FROM NATIONAL AUTHORITIES TO REVISE THE EUROPEAN LEGISLATION TO REVISE THE EUROPEAN LEGISLATION A u r é l i e M a h a l a t c h i m y [ 1 ] , F l o r e n c e L a n s o n [ 2 ] , B l a n k a B a r t o s [ 3 ] , F l o r e n c e T a b o u l e t [ 4 ] MAIN PROPOSALS FROM NATIONAL AUTHORITIES Proportionate to the level of UMN (e.g. variable duration of market exclusivity) Focused on neglected therapeutic areas Conditional to certain obligations Be transparent on R&D costs and actual prices Launch in all or most Member States UMN re-evaluated after commercialization Use experiences developped from covid-19 crisis Joint procurement Flexibility in clinical trials conduct On the incentives granted to sponsors On the criteria for orphan designation To be adjusted to avoid "salami slicing" (segmenting therapeutic indications) To be taken into account with the severity of the disease (level of unmet medical need - UMN) Prevalence : < 5 in 10,000 persons in the EU To be clarified and graduated Significant benefit : no EU satisfactory method of diagnosis, prevention or treatment, or drug significant benefit Many isolated proposals : difficult to identify a consensus among the authorities Request of better cooperation between all agencies and more EMA support BUT emphasis on respecting the EU and Member States competences sharing > Orphan single status transformed into a mosaic of statuses Complexities : how to quantify ? which indicators ? > Future EU considerations of national authorities proposals and answers from other respondents ? Patient access to medicines Health systems sustainability Innovation support EU legislation Objectives to be balanced 80% of rare diseases are of genetic origin in the EU = Many advanced therapies have obtained the orphan medicine status under European regulation (EC) n° 141/2000 of 16 December 1999 on orphan medicial products Provides orphan designation and incentives (market exclusivity for 10 years) Ensure the relevance of incentives granted to industry (to avoid abuses) Reward risk-taking and innovation more Improve equitable patient access in the EU Needs to be revised. Why? Public consultations on revision of European legislation DISCUSSION AND CONCLUSION [1] Permanent Researcher in law at the French National Centre for Scientific Research (CNRS, research fellow), Law Faculty, UMR 7318 International, Comparative and European law (DICE) CERIC, CNRS, Aix Marseille University, Toulon University, Pau & Pays de l’Adour University, Aix-en-Provence, 13628, France. ; [2] CERPOP (Center for Epidemiology and Research in POPulation health), UMR 1295 Inserm, University of Paul Sabatier- Toulouse 3, Toulouse, 31062, France. ; [3] PhD Student in law, CREDIMI, University of Burgundy, Dijon, 27877, France. ; [4] Professor in Pharmaceutical Law and Health Economics, CERPOP, UMR 1295 Inserm, University of Paul Sabatier- Toulouse 3, Toulouse, 31062, France. Acknowledgement: This work has been supported by ANR-funded I-BioLex project (ANR-20-CE26-0007-01, coord. A. Mahalatchimy). *AT, DE, DK, ES, FI, FR, HU, NL, SE ; **AT, BE, CZ, DE, DK, EE, ES, FI, FR, HU, IS, MT, NL, PT, SE