Access to advanced therapy medicinal products in the EU: Where do we stand?

European Journal of Health Law

Volumn 18, Issue 3, 2011, Pages 305-317

  Mahalatchimy, A ^a,b  

^a Faculté de Médecine Centre Hospitalo universitaire   (France)

^b Laboratoire PACTE   (France)

Author keywords

access; Advanced Therapy Medicinal Products (ATMP); European Union (EU); genes; market

Indexed keywords

ARTICLE; CELL TRANSPLANTATION; EUROPEAN UNION; GENE THERAPY; HEALTH CARE DELIVERY; HEALTH CARE POLICY; HUMAN; LEGAL ASPECT; PUBLIC HEALTH; TISSUE ENGINEERING;

CELL TRANSPLANTATION; EUROPEAN UNION; GENE THERAPY; HEALTH POLICY; HEALTH SERVICES ACCESSIBILITY; HUMANS; NATIONAL HEALTH PROGRAMS; TISSUE ENGINEERING;

EID: 80053476354     PISSN: 09290273     EISSN: 15718093     Source Type: Journal    
DOI: 10.1163/157180911X574146     Document Type: Article

References (53)

1. Regulation (EC) N°1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) N°726/2004, OJ L324, 10.12.2007, pp. 121-137.
2. Article 1 § 2 and Annex I of Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use, OJ L 311, 28.11.2001, pp. 67-128;
3. As lastly amended by Directive 2004/27/EC of the European Parliament and of the Council of 31 March 2004 amending Directive 2001/83/EC on the Community code relating to medicinal products for human use, OJ L136, 30.04.2004, pp. 34-57.
4. Commission Directive 2003/63/EC of 25 June 2003 amending directive 2001/83/EC, OJ L 159 of 27.06.2003, pp. 46-94.
5. Annex, Part IV, 2.1 of Commission Directive 2009/120/EC of 14 September 2009 amending Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use as regards advanced therapy medicinal products, OJ L 242 of 15.09.2009, pp. 3-12.
6. Annex, Part IV, 2.2 of Commission Directive 2009/120/EC of 14 September 2009 amending Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use as regards advanced therapy medicinal products, OJ L 242 of 15.09.2009, pp. 3-12.
7. Article 2.1 (b) of Regulation (EC) N°1394/2007, supra note 1.
8. http://www.ema.europa.eu/htms/human/advanced-therapies/recommendations. htm
9. For instance, the Committee for Advanced Therapy recognized as a gene therapy medicinal product "a product consisting of a Salmonella typhi strain genetically modified to secrete a fusion protein of the prostate specific antigen (PSA) and a protein leading to an increased antigenicity, intended for the treatment of prostate cancer": http://www.ema.europa.eu/pdfs/human/cat/ 10968110en.pdf.
10. For instance, the Committee for Advanced Therapy recognised as a somatic cell therapy medicinal product "a product consisting of autologous tolerogenic dendritic cells derived from peripheral blood monocytes, intended for the treatment of rheumatoid arthritis": http://www.ema.europa.eu/pdfs/ human/cat/66775809en.pdf.
11. For instance, the Committee for Advanced Therapy recognised as a somatic cell therapy medicinal product "an autologous cell therapy product, intended for the treatment of Crohn's disease": http://www.ema.europa.eu/ pdfs/human/cat/3891010en.pdf.
12. For instance, the Committee for Advanced Therapy recognised as a somatic cell therapy medicinal product "a product consisting of allogenic natural killer cells activated with a lysate from a cell line which is established from a patient with acute monoblastic leukaemia, intended for the treatment of acute myeloid leukaemia": online at: http://www.ema.europa.eu/pdfs/human/cat/ 3891010en.pdf.
13. On 17 December 2009, the EMA released for consultation the "development of a guideline on the risk-based approach according to annex I, part IV of Directive 2001/83/EC applied to advanced therapy medicinal products": CHMP/CPWP/708420/09, online at: http://www.ema.europa.eu/pdfs/ human/cpwp/70842009en.pdf.
14. EMA, ChondroCelect, European Public Assessment Report, Summary for the public, EMEA/691866/2009, EMEA/H/C/878, online at: http://www.ema.europa.eu/ema/ index.jsp?curl=pages/medicines/human/medicines/000878/human-med-000698. jsp&murl=menus/medicines/medicines.jsp&mid=WC0b01ac058001d125.
15. The CAT is the sixth Committee within the EMA. It was established in accordance with Regulation (CE) No1394/2007 on ATMP to provide notably assessment and recommendations regarding ATMP, online at: http://www.ema.europa. eu/htms/general/contacts/CAT/CAT.html.
16. Online at: http://www.ema.europa.eu/docs/en-GB/document-library/ Committee-meeting-report/2011/05/WC5000106258.pdf.
17. Article 168 of the Treaty on the functioning of the European Union, OJ C83, 30.03.2010, pp. 47-199.
18. T.K. Hervey and J.V. Mchale, Health Law and the European Union, Cambridge: Cambridge University Press, 2004, p. 469.
19. V. Michel, "La compétence de la Communauté en matière de santé publique", Revue des Affaires Européennes - Law & European Affaires, 2 (2003-2004) 157-194.
20. Article 5 al. 2 EU Treaty, OJ C83 of 30.03.2010, pp. 13-45.
21. Committee on Legal Affairs, Report of the European Parliament, 7 February 2007, on the proposal for a regulation of the European Parliament and of the Council on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) N°726/2004 (COM(2005)0567-C6-0401/2005-2005/0227(COD)), A6-0031/2007 final, p. 73;
22. See also F. Bellivier, L. Brunet, M.A. Hermitte et al., "Les limitations légales de la recherche génétique et de la commercialisation de ses résultats: le droit français", Revue Internationale de Droit Comparé 2 (2006) 275-318;
23. C.-M. Romeo Casabona, "La recherche et la thérapie avec des cellules souches embryonnaires. Quel est le cadre juridique pour l'Europe ?", Revue Générale de droit Médical 9(2003) 151-166.
24. COM (2000) 285 final.
25. OJ L 301, 20.11.2007, pp. 3-13.
26. COM (2007) 630 final.
27. S. Hennette-Vauchez, "L'émergence d'un droit communautaire de la biomédecine", Revue Trimestrielle de Droit Européen 45 (1) (2009) 21-45.
28. Communication from the Commission to the European Parliament, the Council, the Economic and Social Committee and the Committee of the Regions, "Life sciences and biotechnology - A strategy for Europe", COM (2002)27.
29. European Commission proposal for a Regulation of the European Parliament and of the Council on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) N®726/2004, COM(2005)0567, 16.11.2005, Explanatory Memorandum, 2.1, p. 3.
30. The new approach aims "to recast technical harmonisation in Europe on a new basis by only harmonising the essential requirements of products and by applying the 'general reference to standards' formula and the principle of mutual recognition in order to eliminate technical obstacles to the free movement of goods", Europa, http://europa.eu/legislation-summaries/ internal-market/single-market-for-goods/technical-harmonisation/l21001a-en.htm.
31. Applications for authorisation [⋯] would be submitted to and processed by the competent authorities of the Member States, passed on to a central scientific committee for evaluation, and eventually approved by the Community", Commission staff working document - Annex to the proposal for a regulation on advanced therapy medicinal products impact assessment - COM (2005) 567 final/SEC/2005/1444/.
32. Regulation (EC) N®726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency, OJ L 136, 30.04.2004, pp. 1-33.
33. Directive 93/42/EEC of the Council concerning medical devices, OJ L169, 12.07.1993, pp. 1-43.
34. Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use, OJ L311, 28.11.2001, pp. 67-128.
35. Directive as amended by Commission Directive 2003/63/EC, OJ L159, 27.06.2003, pp. 46-94
36. Directive 2004/27/EC, OJ L136, 30.04.2004, pp. 34-57.
37. Directive 2004/23/EC of the European Parliament and of the Council on setting standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells, OJ L102, 7.04.2004, pp. 48-58.
38. Commission staff working document - Annex to the proposal for a regulation on advanced therapy medicinal products impact assessment - COM (2005) 567 final/ SEC/2005/1444/.
39. A.K.D. Bock, E. Rodriguez-Cerezo, "Human tissue-engineered products-Today's markets and future prospects." Joint Research Centre-Institute for Prospective Technological Studies. European Commission. Technical Report EUR 21000 EN. October 2003. Online at: ftp://ftp.jrc.es/pub/ EURdoc/eur21000en.pdf.
40. Commission staff working document- Annex to the proposal for a regulation on advanced therapy medicinal products impact assessment - COM (2005) 567 final/SEC/2005/1444/.
41. Whereas (6) and Article 28, 2) of Regulation (CE) N°1394/2007.
42. See M. Blanquet M. and N. De Grove-Valdeyron, "Les enjeux et les apports du règlement communautaire concernant les médicaments de thérapie innovante", Revue des Affaires Européennes - Law & European Affaires 6 (2006) 689-721.
43. Directive 2004/23/EC of the European Parliament and of the Council on setting standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells, OJ L102; 7.04.2004, pp. 48-58.
44. Article 36 of the Treaty on the functioning of the European Union (OJ C83, 30.03.2010, pp. 47-199) provides prohibitions or restrictions on imports or/and exports of goods can be justified on grounds of public morality, public policy or public security; the protection of health and life of humans, animals or plants; the protection of national treasures possessing artistic, historic or archaeological value; or the protection of industrial and commercial property.
45. European Court of Justice, 20 February 1979, Rewe-Zentral AG v. Bundesmonopolverwaltung fur Branntwein, C-120/78, Rec. 1979, p. 649. For example, consumers' protection could be invoked.
46. European Court of Justice, 30 November 1995, Gebhard v. Consiglio dell'Ordine degli Avvocati e Procuratori di Milano, C- 55/94, Rec. 1995, pp. 1-4165.
47. Without being exhaustive, it seems interesting to point out the ECJ notably recognised, in the Kohll and Decker case law [European Court of Justice, 28 April 1998, Kholl, C-158/96, and European Court of Justice, 28 April 1998, Decker, C-120/95], a patient's right to access to medical treatment in another Member State without prior authorisation and to be reimbursed in accordance with the tariffs of the state in which the patient is insured.
48. See J.-L. Clergerie and C. Karathanasi, "La mobilité des patients et le remboursement des dépenses médicales dans l'Union européenne", Revue de Droit Sanitaire et Social 3 (2009) 463-479.
49. A. Denis, L. Mergaert, C. Fostier et al., "A comparative study of European rare disease and orphan drug markets", Health Policy 97 (2010) 173-179.
50. Directive 89/105/ECC of 21 December 1988 relating to the transparency of measures regulating the pricing of medicinal products for human use and their inclusion in the scope of national health insurance systems, OJ L 40 of 11.02.1989, pp. 8-11.
51. Eurordis (European Organisation for rare diseases) proposal for orphan drugs, online at: http://www.eurordis.org/.
52. OJ C 364, 18.12.2000, pp. 1-22.
53. http://europa.eu/lisbon-treaty/index-fr.htm.