Review of the ATMP Regulation

Transcription

1 Review of the ATMP Regulation Joint DIA/MHRA Workshop on ATMPs London, 18 November 2011 Dr. Christian K Schneider Committee for Advanced Therapies (CAT) European Medicines Agency (EMA), London, UK I attend this conference as an individual expert, and do not represent the CHMP/CAT. The views expressed here are my personal views, and may not be understood or quoted as being made on behalf of the CHMP/CAT or reflecting the position of the CHMP/CAT. 2 Has Regulation 1394/2007 been successful? What has worked? What if anything needs to be changed? Key points of the legislation: Positive spirit ( nascent field, new opportunities ) Implementation of the CAT as expert committee Definitions of what ATMPs are Specific requirements (Annex I Dir. 2001/83/EC) Assessment of combined ATMPs Post-authorisation follow-up of efficacy as an option New procedures: Classification, Certification Fee reduction for MA (hospitals, SMEs) Hospital exemption 1

2 Our environment: The academic gap Basic research Regulation 1394/2007 ( ATMP regulation ) Marketing authorisation Development prior to approval Schneider CK and Schäffner-Dallmann G (2008). Nat Rev Drug Discov 7(11):

3 Issues that were encountered during CAT procedures CAT prepares the draft opinion, CHMP adopts final opinion: Requires well-organized collaboration. Can be challenging when the timetable is short (e.g. in re-examination procedures). But, works remarkably well. 5 Marie-Helene Pinheiro, EMA Issues that were encountered during CAT procedures Requirements according to Annex I to Directive 2001/83/EC: The same standard for all? Double standards? Or just different ways to the same standards? 6 both will drive you home!! 3

4 Annex I to Directive 2001/83/EC: How to handle minimally manipulated ATMPs? (e.g., bone marrow in non-homologous use) Starting materials Validation Identity Purity Potency Mechanism of Action 7 How much can we ask for? 8 4

5 9 What happens upon licensing of such product? Minimally manipulated ATMPs could be seen as easy to manufacture Doctors could use autologous bone marrow and prepare it themselves (under hospital exemption?) Doctors would then make use of the data generated by the ATMP manufacturer Should there be an ATMP data protection? 10 5

6 ATMP Classification Article 17: Any applicant developing a product based on genes, cells or tissues may request a scientific recommendation of the Agency with a view to determining whether the referred product falls, on scientific grounds, within the definition of an advanced therapy medicinal product.( ) 11 Borderline GTMP on the horizon Molecular Therapy, 17, ,

7 How to interpret the definition of GTMP? Example: Borderline case of bacteria expressing a human gene Gene therapy medicinal product means a biological medicinal product which has the following characteristics: (a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence to/in human cells or human genome? Or not?; How to avoid disharmonization: Classification by CAT is a recommendation only. Certification: What is it? Art. 18: Small and medium-sized enterprises developing an advanced therapy medicinal product may submit to the Agency all relevant quality and, where available, nonclinical data required in accordance with modules 3 and 4 of Annex I to Directive 2001/83/EC, for scientific evaluation and certification. Early stage development vs. Annex I: Is the Annex I written from a Marketing Authorisation perspective? EMA procedureal guidance EMA/CAT/418458/2008/corr.: Not binding for future MA; not a Scientific Advice; not binding for National Agencies for Clinical Trial Authorisations. 14 7

8 Certification What is a certification? (a personal interpretation) i 16 Art. 28: the so-called hospital exemption Additional exclusion under very specific conditions e.g.: Non-routine basis of production [what is this?] Specific quality standards Used in same MS in hospital (manufacturing authorized by Comp. Authority of MS) Custom-made made product for individual patient Under the exclusive professional responsibility of a practitioner National rules on the use of cells on ethical grounds An alternative Marketing Authorisation procedure? Creation of a second market? 8

9 In God we trust, the rest bring data! W. Edwards Deming Pioneer in Quality Philosophy, W. Edwards Deming is widely held to have been one of the leaders who helped create the Total Quality Movement. Deming s 14 points and his book Out of the Crisis are key documents in the development of Quality Systems for Business management. Dr. Deming is best known for his revolution in the quality and economic productions in Japan where from 1950 onward he taught top management and engineers, methods for management of quality. These teachings dramatically altered the economy of Japan. In recognition of his contributions the Union of Japanese Science and Engineering (JUSE) instituted the annual Deming prizes for achievement in quality and dependability of product. 9

10 Efficacy data (Marketing Efficacy!) ( Experience Proof of efficacy!) Which data can be used? How to deal with Companys claims like No reports on serious adverse events so far, so a very well tolerated and safe product? Conclusions 20 ATMP Regulation is a milestone Problems arise where the legislation comes to its border to national legislations or other provisions: Hospital exemption Classification Regulation/handling of GMOs Problems arise in daily practice with the new procedures and special product scenarios Problems arise for academia when not organized as SME. 10