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Why are some conditions more suitable for gene and cell therapies than others?
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Glossário

Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Antibody
Antigen
Base editors
Cas9 nuclease
Chimeric Antigen Receptor (CAR)
Chromosome
Clinical trial

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ATMPs specific "Hospital exemption" pathway

Introduction

The ‘Hospital exemption’ is specific to ATMPs. It has been established by the ATMP regulation (Article 28), which modifies the European Union (EU) general pharmaceutical legislation (Article 3.7 Directive 2001/83/EC). “Advanced therapy medicinal products which are prepared on a non-routine basis according to specific quality standards, and used within the same Member State in a hospital under the exclusive professional responsibility of a medical practitioner, in order to comply with an individual medical prescription for a custom-made product for an individual patient” are excluded from the scope of EU general pharmaceutical legislation. This means that they are not submitted to the standard regulatory pathway as other ATMPs, i.e. the mandatory centralised procedure of marketing authorisation of ATMPs and its linked requirements.

Moreover, some requirements for exempted ATMPs are established by European Union law which requires Member States to implement them at National levels:

  • The manufacturing of exempted ATMPs shall be authorised by the competent authority of the relevant Member State.
  • National traceability and pharmacovigilance requirements as well as specific quality standards for exempted ATMPs have to be equivalent to those applicable to non-exempted ATMPs that are fully regulated at the EU level.

Apart from such an EU definition and the above-mentioned EU requirements, exempted ATMPs are regulated at National levels. It has resulted in a huge heterogeneity of rules applicable to exempted ATMPs across Member States.

On such a basis, the main issue at stake is the potential competition between two regulatory pathways’ types: the mandatory centralised marketing authorisation and the linked requirements for non-exempted ATMPs as governed by EU law, and the various National regulations at National level for exempted ATMPs. Indeed, in the spirit of the ATMP regulation, the National rules on exempted ATMPs should not undermined the EU rules related to the quality and safety of non-exempted ATMPs (Recital 6 of the ATMP regulation).

Actors, including the European Commission, the EMA, and National medicines agencies are aware of the issues raised by hospital exemption for ATMPs. In that context, the European Commission has started on new study on this topic at the end of 2023, the 2023 European Commission’s proposal for a revision of the general pharmaceutical legislation covers the hospital exemption, and a revision of the ATMP regulation can be considered. Nevertheless, the issues at stake are politically very sensitive. Strong conflicting views may slow down any revision of the EU legislation, which process is based on a co-decision by the European Parliament (representing EU citizens) and the council of Ministers (composed of representatives of the Governments of each Member State of the EU) on the basis on the proposal from the European Commission. 

Main actors

National Competent Authorities: National Competent Authorities, which are often national medicines agencies, are in charge of implementing national laws for exempted ATMPs.

Manufacturer of an exempted ATMP: The legal entity authorized to manufacture an exempted ATMP in compliance with national law.

Medical practitioner: the person with medical qualifications who has the exclusive professional responsibility to provide an individual medical prescription for an exempted ATMP to be administered to an individual patient.

Individual patient: the patient to which an exempted ATMP is administered in accordance with an individual medical prescription in a hospital under the exclusive professional responsibility of a medical practitioner.

Hospital: Within a Member State, the health establishment in which an exempted ATMP is prepared and administered to an individual patient.

Definitions

Hospital exemption/ Exempted ATMPs: “Advanced therapy medicinal products which are prepared on a non-routine basis according to specific quality standards, and used within the same Member State in a hospital under the exclusive professional responsibility of a medical practitioner, in order to comply with an individual medical prescription for a custom-made product for an individual patient” are exempted from the application of general EU pharmaceutical legislation (Directive 2001/83/EC and Regulation 726/2004) and the ATMP regulation. (Article 3.7 of Directive 2001/83/EC and article 28.2 of the ATMP regulation)

Non-exempted ATMPs: Advanced therapy medicinal products which are intended to be placed on the entire EU market and either prepared industrially or manufactured by a method involving an industrial process. As such they are covered by the general EU pharmaceutical legislation (Directive 2001/83/EC and Regulation 726/2004) and the ATMP regulation. In particular, they have to obtain a marketing authorisation (Centralised Procedure for ATMPs) to be marketed in the EU.

Non-routine basis: this expression is not expressly defined in EU binding law. Thus, it has been subject to various interpretations at the National level. Nevertheless, it can be deducted from existing provisions on ATMPs in EU law, that an ATMPs prepared on a non-routine basis is not “prepared industrially or manufactured by a method involving an industrial process”. Nevertheless, in this context of not being prepared industrially, it remains possible to have a restrictive or a wider approach of what means “non-routine basis”. The interpretation of this expression is also linked to the one of “custom-made product”.

Custom-made product for an individual patient: This expression is not expressly defined in EU binding law. Thus, it has been subject to various interpretations at the National level. For instance, some Member States have established an upper limit number of patients to be treated while others have not but patients must be treated one by one (as in France, the French version of “custom-made product” being literally “a product specially designed for a particular patient” in the French version of the ATMP regulation).

Either prepared industrially or by a method involving an industrial process: This expression is not expressly defined in EU binding law. Nevertheless, the European Commission has tried to clarify ‘the industrial process’ in its Staff working document during the legislative process of adoption of the ATMP regulation: “This should cover, inter alia: Any `mass production' of advanced therapy products for allogeneic use (batch production, `off the shelf' products etc.); any advanced therapy product for autologous use (i.e. using cells/tissues from a single patient and re-implanting after manipulation into same patient) which, although being patient-specific by definition, is manufactured in accordance with a standardised and industrial process.” (European Commission, Staff Working document, Annex to the proposal for a regulation on advanced therapy medicinal products impact assessment).

Challenges

Implementation of the hospital exemption rules is highly heterogeneous across Member States. Divergences relate to the interpretation of “specific quality standards”, clinical evidences to be provided, type of eligible hospital exemption approval holders (e.g., public only, or possible for private entities), patients’ eligibility, supervision, meaning of “non-routine basis” (e.g., based on scale or frequency, with an upper limit of patients’ number or for one patient at a time), the intended purpose (e.g., early development of ATMPs before the start of clinical trials, treatment of patients outside clinical trials such as under compassionate use schemes, for unmet medical needs, or as an alternative pathway to marketing authorisation), duration of the approval (e.g., 1 year, several years or no end given that there is no EU legislative provision stating that the hospital exemption should no longer be possible when a non-exempted ATMP has obtained a centralised marketing authorisation).

As a consequence of these heterogeneous rules, a major challenge relates to the interplay between and respective indications of nationally regulated exempted ATMPs and centrally authorised ATMPs. The 2014 European Commission’s report (COM(2014) 188 final) highlighted the need to clarify the role that hospital exemption data could play for the marketing authorisation of non-exempted ATMPs. The European Commission as well as some members of the EMA’s Committee for Advanced Therapies (CAT) have regularly stated in gene and cell therapies congresses that it is not allowed to use the hospital exemption data for clinical trials applications. However, some CAT members (representing National medicines agencies) have stated the opposite, sometimes in the same congresses. For the latter, all data available including those coming from hospital exemption national pathways should be used to support marketing authorisation applications of non-exempted ATMPs, given the current difficulties to prove long-term efficacy for many of these medicines. In that context, the developers of exempted ATMPs, CAR-T Cells ARI-0001 et ARI-0003, in Spain are exploring whether they could obtain a marketing authorisation for their products in the future. This is a significant example of the current challenge and its potential evolution.

While the hospital exemption is necessary for patients to access exempted ATMPs that are not intended to be marketed, and are prepared for a few patients only on the basis of named prescriptions, in one Member State, such regulatory pathways should not compete with the centralised marketing authorisation pathway which gives non-exempted ATMPs access to the market of the 27 Member States of the EU, and thus potentially to all EU patients. The right balance has to be found.

Opportunities and incentives

A European Commission Study on hospital exemption has started in September 2023 and should end in January 2025. It should provide a mapping of the hospital exemption pathways in the EU Member States, a study of the interplay of the hospital exemption with other legal frameworks (such as, centrally authorised ATMPs, Clinical trials, Substances of Human Origin, Medical Devices, orphan medicines, and other derogatory pathways that are not specific to ATMPs but may be used for ATMPs such as compassionate uses pathways) in order to adopt recommendations for supporting implementation of the new EU pharmaceutical legislation (i.e., best practices regarding data collection & reporting; template for national implementation report; analyses, rationale & recommendations for a potential revision of the ATMP regulation).

Moreover, in April 2023, the European Commission has initiated the legislative process for a major reform of the EU general pharmaceutical legislation. In that context, the European Commission’s proposal for a new Directive on the Union code relating to medicinal products for human use, and repealing Directive 2001/83/EC (COM(2023) 192 final) includes several new provisions relating to the hospital exemption (Article 2 of the proposal for a directive).

First, it includes a set of measures for the annual collection and reporting of data on the use, safety and efficacy of exempted ATMPs by the hospital exemption approval holder to the national competent authority, the review of such data and their annual transfer to the EMA by the national competent authority, as well as the setting up and maintaining of a repository for such data by the EMA. (Article 2.4 to 2.6 of the proposal for a directive)

Second, the EMA should provide a report on the implementation of hospital exemption on the basis of contributions from Member States in order to examine whether an adapted framework should be established for certain less complex ATMPs that have been developed and used under the hospital exemption (3 years after adoption of the new Directive and then every 5 years). (Article 2.8 of the proposal for a directive)

Finally, the proposal for a directive also provides for the European Commission to adopt further legislative acts to implement these provisions regarding:

  • The details of the application for the National approval of HE “including the evidence on quality, safety and efficacy” for approval
  • the format for collection and reporting of data on the use, safety and the efficacy of ATMP under HE
  • the modalities for the exchange of knowledge between HE approval holders within the same Member State or different Member States
  • the modalities for preparation and use of exempted ATMP on a non-routine basis. (Article 2.7 of the proposal for a directive)

In its first reading, the European Parliament has proposed the following amendments to article 2 of the European Commission proposal for a directive, mainly:

  • The EMA Report on the implementation of hospital exemption to be made available to the public.
  • By derogation, “Member States may authorize the cross-border exchange of advanced therapy medicinal products prepared under the hospital exemption in justified cases of medical need and in the absence of other solutions for the individual patient.”
    • “A second medical practitioner and a hospital pharmacist in the receiving Member State shall be designated for the exclusive professional responsibility of the use and collection of follow-up data for the advanced therapy medicinal product.”
    • “Information about the cross-border exchange shall be submitted to the competent authorities of both Member States, and shall be shared in the public repository”

The European Parliament has also proposed to add in the recitals before the articles of the future directive that “Competent authorities should support academic institutions and other non-profit entities through the requirements of the hospital exemption clause.” (Recital 18)

Practical steps

1/ To clarify if the product is legally qualified as an exempted ATMPs.

2/ To identify and implement the applicable National regulatory framework

3/ To interact with the National competent authority to find support on 1/ and 2/.

Given the current challenge and potential evolution regarding exempted ATMPs, it may be relevant for developers to establish and maintain a list of establishments that prepare and distribute exempted ATMPs, if it is not available at the EMA level depending on the final version of the EU general directive on medicinal products for human use at the end of the legislative process for its revision. The latter should take a few years.

Interactions with regulators

Given that exempted ATMPs are regulated at national levels, developers of exempted ATMPs can contact the national competent authorities of their respective Member State in order to obtain regulatory support.

National competent authorities for exempted ATMPs are generally medicines agencies, most of which have notably innovation offices, and provide opportunities for early interactions.

The list of National Medicines Agencies is available here.

European Union Legislation

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, p. 67-128, CELEX number: 32001L0083

Regulation (EC) No 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) No 726/2004 (Text with EEA relevance), OJ L 324, 10.12.2007, p. 121-137, CELEX number: 32007R1394

European Union guidance and documents

Proposal for a Directive of the European Parliament and the Council on the Union code relating to medicinal products for human use, and repealing Directive 2001/83/EC and Directive 2009/35/EC, 26.4.2023, COM(2023) 192 final.

General Secretariat of the Council, Proposal for a DIRECTIVE OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL on the Union code relating to medicinal products for human use, and repealing Directive 2001/83/EC and Directive 2009/35/EC - Outcome of the European Parliament's first reading (Brussels, 10 to 11 April 2024), 2 July 2024, 11754/24.

European Commission, report from the Commission to the European Parliament and the Council in accordance with Article 25 of Regulation (EC) No 1394/2007 of the European Parliament and of the Council on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004, 28/03/2014, COM(2014) 188 final.

European Commission, Pharmaceutical Committee, Hospital exemption for ATMPs (implementation of Art 28(2) of the ATMP regulation): update on feedback received by the Commission, 28/03/2012, PHARM 600.

European 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/

Relevant literature

  1. Barriers for the evaluation of advanced therapy medicines and their translation to clinical practice: Umbrella review - Aguilera-Cobos, Lorena, María Piedad Rosario-Lozano, Angela Ponce-Polo, Juan Antonio Blasco-Amaro, and David Epstein. Health Policy 126, no. 12 (1/12/2022): 1248-1255.
  2. Cell therapy medicinal product regulatory framework in Europe and its application for MSC-based therapy development - Ancans, Jains. Frontiers In Immunology 3 (2012): 253. 
  3. Clinical research and drug regulation in the challenging times of individualized therapies: A pivotal role of clinical pharmacology - Antonio Gomez-Outes a, Aranzazu Sancho-Lopez b., Antonio J. Carcas Sansuan c, and Cristina Avenda˜no-Sola b. Pharmacological Research 199 (1/01/2024): 107045. 
  4. On CAR-Ts, decentralized in-house models, and the hospital exception. Routes for sustainable access to innovative therapies - Arnaudo, Luca. Journal of Law and the Biosciences 9, no. 2 (2022): 1-19. 
  5. The European Hospital Exemption Clause—New Option for Gene Therapy? - Buchholz, Christian J., Ralf Sanzenbacher, and Silke Schuele. Human Gene Therapy 23, no. 1 (01/2012): 7-12.
  6. Developing cell therapies as drug products - Ciccocioppo, Rachele, Patrizia Comoli, Giuseppe Astori, Francesca del Bufalo, Malvina Prapa, Massimo Dominici, and Franco Locatelli. British Journal of Pharmacology 178, no. 2 (2021): 262-279. 
  7. Regulating Advanced Therapy Medicinal Products through the Hospital Exemption: An Analysis of Regulatory Approaches in Nine EU Countries -  Coppens, Delphi G. M., Helga Gardarsdottir, Marie L. De Bruin, Pauline Meij, Hubert G. M. Leufkens, and Jarno Hoekman. Regenerative Medicine 15, no. 8 (08/2020): 2015-2028. 
  8. Advanced therapy medicinal product manufacturing under the hospital exemption and other exemption pathways in seven European Union countries - Coppens, Delphi G. M., Jarno Hoekman, Marie L. De Bruin, Ineke C. M. Slaper-Cortenbach, Hubert G. M. Leufkens, Pauline Meij, and Helga Gardarsdottir. Cytotherapy 22, no. 10 (1/10/2020): 592-600. 
  9. The puzzling situation of hospital exemption for advanced therapy medicinal products in Europe and stakeholders' concerns - Cuende, Natividad, Christelle Boniface, Christopher Bravery, Miguel Forte, Rosaria Giordano, Martin Hildebrandt, Ander Izeta, and Massimo Dominici. Cytotherapy 16, no. 12 (1/12/2014): 1597-1600. 
  10. Patient access to and ethical considerations of the application of the European Union hospital exemption rule for advanced therapy medicinal products - Cuende, Natividad, Rachele Ciccocioppo, Miguel Forte, Jacques Galipeau, Laertis Ikonomou, Bruce L. Levine, Alok Srivastava, and Patricia J. Zettler. Cytotherapy 24, no. 7 (1/07/2022): 686-690.
  11. A management model in blood, tissue and cell establishments to ensure rapid and sustainable patient access to advanced therapy medicinal products in Europe - Delgadillo, Joaquín, Erja Kerkelä, Allison Waters, Emile van den Akker, Chantal Lechanteur, Etienne Baudoux, Nicola Gardiner, John De Vos, and Joaquim Vives. Cytotherapy 25, no. 12 (1/12/2023): 1259-1264.
  12. EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation - Detela, Giulia, and Anthony Lodge. Molecular Therapy - Methods & Clinical Development 13 (14/06/2019): 205-232.
  13. Unproven cell interventions in Poland and the exploitation of European Union law on advanced therapy medicinal products - Dulak, Józef, and Marlena Pecyna. Stem Cell Reports 18, no. 8 (8/08/2023): 1610-1620.
  14. Accelerating Patients’ Access to Advanced Therapies in the EU: Molecular Therapy Methods & Clinical Development - Elsanhoury, Ahmed, Ralf Sanzenbacher, Petra Reinke, and Mohamed Abou-El-Enein. Molecular Therapy - Methods & Clinical Development 7 (15/12/2017): 15-19.
     
  15. Commensuration and Proliferation: Similarity and Divergence in Law's Shaping of Medical Technology - Faulkner, Alex. Law, Innovation and Technology 4, no. 2 (2012): 165-184. 
  16. Unlocking the value of innovative medicines: Insights from the advanced therapy medicinal products (ATMP) innovation systems in Germany and Sweden - Fischer, Piret, Thomas Reiss, Jörg Mahlich, Erwan Gicquel, Heike Aichinger, Liliya Pullmann, and Tanja Bratan. Health Policy and Technology 12, no. 2 (1/06/2023): 100744.
  17. European Regulatory Tools for Advanced Therapy Medicinal Products - Flory, Egbert, and Jens Reinhardt. Transfusion Medicine and Hemotherapy 40, no. 6 (29/10/2013): 409-412. 
  18. Focus Hospital Exemption on Developing Innovative and Safe Treatments for Patients - ‘Focus Hospital Exemption on Developing Innovative and Safe Treatments for Patients’. Regenerative Medicine 8, no. 2 (1.03/2013): 121-123. 
  19. EU Marketing Authorisation of Orphan Medicinal Products and Its Impact on Related Research - Gerke, Sara, and Shaun D. Pattinson. European Journal of Health Law 24, no. 5 (2017): 541-564. 
  20. Advanced Therapy Medicinal Products Challenges and Perspectives in Regenerative Medicine - Goula, Aspasia, Vasiliki Gkioka, Efstathios Michalopoulos, Michalis Katsimpoulas, Michel Noutsias, Eirini Faidra Sarri, Catherine Stavropoulos, and Alkiviadis Kostakis. Journal of Clinical Medicine Research 12, no. 12 (12/2020): 780-786.
  21. An assessment of the hospital exemption landscape across European Member States: regulatory frameworks, use and impact - Hills, Allison, Jacquelyn Awigena-Cook, Ken Genenz, Mareike Ostertag, Simon Butler, Anne-Virginie Eggimann, and Annie Hubert. Cytotherapy 22, no. 12 (1/12/2020): 772-779.e1.
  22. Hospital Exemption for Advanced Therapy Medicinal Products: Issue in Application in the European Union Member States - Ivaskiene, Tatjana, Mykolas Mauricas, and Justinas Ivaska. Current Stem Cell Research & Therapy 12, no. 1 (2017): 45-51.
  23. Is Hospital Exemption an Alternative or a Bridge to European Medicines Agency for Developing Academic Chimeric Antigen Receptor T-Cell in Europe? Our Experience with ARI-0001 - Juan, Manel, Julio Delgado, Gonzalo Calvo, Esteve Trias, and Alvaro Urbano-Ispizua. Human Gene Therapy 32, no. 19-20 (10/2021): 1004-1007.
  24. Processing and Ex Vivo Expansion of Adipose Tissue-Derived Mesenchymal Stem/Stromal Cells for the Development of an Advanced Therapy Medicinal Product for use in Humans - Labedz-Maslowska, Anna, Agnieszka Szkaradek, Tomasz Mierzwinski, Zbigniew Madeja, and Ewa Zuba-Surma. Cells 10, no. 8 (08/2021): 1908.
  25. Creating Conditions for the Success of The French Industrial Advanced Therapy Sector - Lirsac, Pierre Noel, Olivier Blin, Jérémy Magalon, Pierre Angot, Estelle de Barbeyrac, Pascal Bilbault, Elisabeth Bourg, et al. Therapies, XXX Rencontres nationales de Pharmacologie et Recherche clinique, pour l’Innovation et l’Évaluation des Technologies de Santé Tables rondes Giens – 5 au 6 octobre 2014, 70, no. 1 (1/01/2015): 83-94.
  26. Encountering Challenges with the eu Regulation on Advance Therapy Medical Products - Mansnérus, Juli. European Journal of Health Law 22, no. 5 (28/10/2015): 426-461.
  27. Creating European Markets through Regulation: the Case of the Regulation on advanced therapy medicinal products - Marton Varju and Judit Sandor. European Law Review 41, no. 1 (2016): 25-43.
  28. Access to CAR-T Therapies in Ireland: Legal, Ethical & Broader Policy Considerations - McMahon, Aisling. Maynooth University, (25/01/2024).
     
  29. New challenges in advanced therapies - Megías-Vericat, Juan Eduardo, Ana Bonora-Centelles A, Tomás Palanques-Pastor, Cristóbal Eduardo Aguilar Gallardo, Manuel Guerreiro, Inés Gómez Seguí, Javier De La Rubia, and José Luis Poveda Andrés. Farmacia Hospitalaria, Nuevos roles y retos del farmacéutico de hospital, 48 (1/07/2024): TS21-27. 
  30. Frontiers | Polish regulatory system regarding ATMP hospital exemptions - Pachocki, Jan, and Frances Verter. Frontiers in Immunology 15 (13/05/2024).
  31. Business oriented EU human cell and tissue product legislation will adversely impact Member States’ health care systems - Pirnay, Jean-Paul, Alain Vanderkelen, Daniel De Vos, Jean-Pierre Draye, Thomas Rose, Carl Ceulemans, Nadine Ectors, Isabelle Huys, Serge Jennes, and Gilbert Verbeken. Cell and Tissue Banking 14, no. 4 (1/12/ 2013): 525-560. 
  32. Advanced Therapy Medicinal Products and the Changing Role of Academia - Priesner, Christoph, and Martin Hildebrandt. Transfusion Medicine and Hemotherapy 49, no. 3 (16/05/2022): 158-162. 
  33. A Room with a View (and with a Gene Therapy Drug): Gene Therapy Medicinal Products and Genetic Tourism in Europe - Raposo, Vera Lúcia. European Journal of Health Law 29, no. 3–5 (8/04/2022): 504-520. 
  34. Moving somatic gene editing to the clinic: routes to market access and reimbursement in Europe - Rigter, Tessel, David Klein, Stephanie S. Weinreich, and Martina C. Cornel. European Journal of Human Genetics 29, no. 10 (10/2021): 1477-1484.
  35. Exploring Solutions to Foster ATMP Development and Access to Patients - Salvatore, Vincenzo. European Journal of Health Law 27, no. 3 (2020): 259-273.
  36. Current challenges in cell and gene therapy: a joint view from the European Committee of the International Society for Cell & Gene Therapy (ISCT) and the European Society for Blood and Marrow Transplantation (EBMT) - Sanchez-Guijo, Fermin, Joaquim Vives, Annalisa Ruggeri, Christian Chabannon, Selim Corbacioglu, Harry Dolstra, Dominique Farge, et al. Cytotherapy 26, no. 7 (1/07/2024): 681-685.
  37. The hospital exemption pathway for the approval of advanced therapy medicinal products: an underused opportunity? The case of the CAR-T ARI-0001 - Trias, Esteve, Manel Juan, Alvaro Urbano-Ispizua, and Gonzalo Calvo. Bone Marrow Transplantation 57, no. 2 (1/02/2022): 156-159. 
  38. Frontiers | Advanced Therapy Medicinal Products and Exemptions to the Regulation 1394/2007: How Confident Can We be? An Exploratory Analysis - Van Wilder, Philippe. Frontiers in Pharmacology 3 (14/02/2012).
  39. Hurdles in clinical implementation of academic advanced therapy medicinal products: A national evaluation - Wilde, Sofieke de, Louise Veltrop-Duits, Merel Hoozemans-Strik, Thirza Ras, Janine Blom-Veenman, Henk-Jan Guchelaar, Maarten Zandvliet, and Pauline Meij. Cytotherapy 18, no. 6 (1/06/2016): 797-805. 
  40. EU Pharma Legislation Review Series: Pharmacy and Hospital Exemptions, Grant Castle, Peter Bogaert, Anna Wawrzyniak, Inside EU Life Sciences, 28 April 2023.
     
  41. Advanced therapies, hospital exemptions & marketing authorizations: the UK’s emerging regulatory framework for point-of-care manufacture, Bicudo, E.; Brass, I. (2023), Cell & Gene Therapy Insights 9(1): 101-120.
     
  42. Technology forecast: advanced therapies in late clinical research, EMA approval or clinical application via hospital exemption - Claudia Eder, and Claudia Wild, Journal of Market Access & Health Policy, Online publication (19 April 2019): 1600939.
     
  43. Master thesis - The Hospital Exemption, a regulatory option for unauthorised ATMPs, Anna Schnitger, 12 December 2014.

More

  1. Learned Societies

European Blood Alliance. EBA opposes restriction of Hospital Exemption of ATMPs, 14 March 2024.

European Hematology Association (EHA) and Association of European Cancer Leagues - ECL,Stakeholders call for protection of the EHA Hospital Exemption, 19 February 2024.

Salud por Derecho, The Hospital Exemption: increasing access to innovation and local production, November 2023

Delphi Coppens - KWF Kankerbestrijding, ATMP treatment under the Hospital Exemption, 23 November 2021.

Alliance for Regenerative Medicine, Recommendations for the use of Hospital Exemption, 10 October 2020.

Alliance for Regenerative Medicine, Alliance for Regenerative Medicine position on Hospital Exemption, 16 February 2017.
 

  1. Business Associations

EUCOPE, EUCOPE Position Paper - Hospital Exemption clause in ATMPs: Recommendations for good practice, February 2024.

EPTRI, EAHP, EUEYE and SIOPE, Joint Position_EPTRI_EAHP_EUEYE_SIOPE: Hospital Exemption in ATMP development within the Revision of the EU general pharmaceuticals legislation, November 2023.

European Federation of Pharmaceutical Industries and Associations, EFPIA - Joint Position Paper on the Hospital Exemption Scheme for ATMPs, 13 July 2023.

Sánchez-Guijo, Fermín, Cristina Avendaño-Solá, Lina Badimón and al., Role of Hospital Exemption in Europe: position paper from the Spanish Advanced Therapy Network (TERAV) | Bone Marrow Transplantation, , Bone Marrow Transplantation 58, no. 6 (June 2023): 727-728. https://doi.org/10.1038/s41409-023-01962-0.

Europabio, Application of the ATMP Hospital Exemption in the EU, 19 October 2020.

EUCOPE, Hospital Exemption for ATMPs: Our Recommendations, 6 May 2020.

EUCOPE, EUCOPE - Recommendations for good practice of Hospital Exemption clause in ATMPs, 21 April 2020.

Belgian Association for Tissue and Cell Banks, Hospital exemption ATMP, 24 April 2017.

European Biopharmaceutical Enterprises, Position paper on the Hospital Exemption, July 2016.
 

  1. Media

Marta Iraola Iribarren, Commission’s hospital exemption divides health sector, Euronews, 3 May 2024.
 

  1. Companies

Simmons & Simmons, EU pharma package: ATMPs under hospital exemption, 26 May 2023.

Inbeeo, European Hospital Exemption Pathways for ATMPs : Implementation and Impact  - Inbeeo Strategic Market Access & Pricing, 21 September 2023.

Meletsi, Angeliki – P4A, Hospital Exemption; still a big risk for pharma, 26 April 2023.

PharmaLex GmbH, Study on Hospital Exemption for ATMPs in Selected EU Countries – FINAL REPORT For the Federal Office of Public Health (FOPH) / Bundesamt für Gesundheit (BAG), Bern, Switzerland, January 2022.
 

  1. Events

European Commission, Implementation of the Hospital Exemption in the EU and its role in boosting innovation and patient access to innovative therapies, Online Event, 21 November 2024.

Acknowledgements

Published: 27/01/2025 by Valentin Roby

Authors:

Aurélie Mahalatchimy, EuroGCT WP4 Convenor, UMR 7318 DICE CERIC, Aix-Marseille University, CNRS, Aix-en-Provence- France

Christian Chabannon, Professor of Molecular Biology, MD, Centre de Thérapie Cellulaire. Institut Paoli-Calmettes Comprehensive Cancer Center & module Biothérapies du Centre d’Investigations Cliniques de Marseille, Inserm CBT-1409 –AMU –AP-HM -IPC

Research Pathways Overview

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