EU Compassionate Use Programmes (CUPs)
- First Online:
- 119 Downloads
For some patients, compassionate use programmes (CUPs) are the only way to access promising medicinal products that are not yet authorized and for which clinical trials are either not available or in which these patients cannot participate. For such patients, the CUP benefits can be very important. Although an EU regulatory framework exists, there is no single, centralized EU-wide procedure for CUPs. Each Member State has its own procedures and CUP approvals are governed by national regulatory authorities. This article provides an overview of the regulatory frameworks in the EU for CUPs and includes a discussion of the different types of programme available, eligibility criteria, key points for industry to consider and related pharmacovigilance obligations.
There are two major types of CUPs, commonly referred to as named patient compassionate use programmes (NP-CUPs) and cohort compassionate use programmes (Coh-CUPs). NP-CUPs are typically initiated by physicians for an individual patient in great need of a medicinal product, which will be administered under the physician’s responsibility. Companies usually have little influence on this type of CUP. However, companies can try to anticipate these demands and define in advance a set of criteria allowing safe access and administration to these patients. Coh-CUPs are usually defined programmes initiated by the manufacturer to allow access for a group of patients to an unauthorized medicinal product. Both NP-CUPs and Coh-CUPs are allowed under strict conditions only. A key point for a successful CUP is to manage stakeholders’ expectations and to have a good level of communication with patients and physicians, although it is strictly forbidden to promote the use of unauthorized medicinal products. In addition, a CUP needs to be well controlled, and risks carefully anticipated particularly with regards to safety aspects. In terms of financing, there is no general rule with regards to the possibility of charging for these medicinal products under CUP. It depends on the country and on the type of programme. For some countries, it is often on a case-by-case basis. Thus, this should also be factored in to the budget. Importantly, in some countries, such as France, the price of a medicinal product established in a CUP will influence future price negotiations after the medicinal product is granted marketing authorization, as well as the timing of these negotiations. In such cases, it can be difficult to obtain a higher price than was applied in the CUP and it is wise to conduct pharmacoeconomic studies prior to proposing the price for the product during the CUP. Finally, if a CUP is anticipated, companies should plan their internal and external resources. Important resources include a project manager supported by a medical person, as well a person in charge of the pharmacovigilance aspect and an EU importer/distributor.
There are many factors to take into account when a medicinal product is expected to be solicited in a CUP, whether NP-CUP or a Coh-CUP. Careful planning for a CUP is critical and should take into account the perspective of the treating physician and the patient, as well as the national regulatory frameworks.
- 1.Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating tomedicinal products for human use (OJ L 311, 28.11.2001, p. 67) as amended (consolidated version 30/12/2008) [online]. Available from URL: http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=CONSLEG:2001L0083:20081230:EN:PDF [Accessed 2010 Jul 23]
- 2.Regulation (EC) No 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, 30.4.2004, p. 1) [consolidated version: 20/04/2009; online]. Available from URL: http://ec.europa.eu/health/files/eudralex/vol-1/reg_2004_726_cons/reg_2004_726_cons_en.pdf [Accessed 2010 Jul 23]
- 3.European Medicines Agency Committee for Medicinal Products for Human Use (CHMP). Guideline on compassionate use of medicinal products, pursuant to article 83 of Regulation (EC) No 726/2004 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2009/10/WC500004075.pdf [Accessed 2010 Aug 16]
- 4.European Medicines Agency. Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring adressed to Member States for IV Tamiflu: available for compassionate use [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Other/2010/05/WC500090250.pdf [Accessed 2010 Jul 23]
- 5.European Medicines Agency. Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring adressed to member states for IV Zanamivir available for compassionate use [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Other/2010/02/WC500074124.pdf [Accessed 2010 Jul 23]
- 6.VOLUME 9A of the rules governing medicinal products in the European Union — guidelines on pharmacovigilance for medicinal products for human use: version September 2008 [online]. Available from URL: http://ec.europa.eu/health/files/eudralex/vol-9/pdf/vol9a_09-2008_en.pdf [Accessed 2010 Jul 23]