Compassionate use of orphan drugs

Abstract

BackgroundEU regulation 726/2004 authorises manufacturers to provide drugs to patients on a temporary basis when marketing authorisation sought centrally for the entire EU is still pending. Individual Member States retain the right to approve and implement such ‘compassionate use’ programmes which companies will usually provide for free. Nevertheless some companies have opted not to partake in such programmes, in effect restricting access to drugs for patients in need. Here we survey the state of compassionate use programmes in the EU with particular reference to the rare disease field, and provide legal and ethical arguments to encourage their increased compassionate use in the EU and beyond. We contend that if enacted, these recommendations will be mutually beneficial to companies as well as patients.MethodsRequests for information from the European Medicines Agency were made under the UK Freedom of Information Act 2000. Legal, ethical and economic/pragmatic analysis identified means by which provision of therapy in compassionate use programmes might be increased.ResultsMore than 50 notifications of compassionate use programmes have been submitted to the EMA by Member States since 2006. About 40 % relate to orphan drugs. As there is a compulsory register of programmes but not of outcomes, their success is difficult to evaluate but, for example, the French programme expedited treatment for more than 20,000 (orphan and non-orphan) patients over a period of three years.ConclusionCompelling self-interested, legal and ethical arguments can be mounted to encourage manufacturers to offer therapies on a compassionate use basis and these are often equally applicable to provision on a humanitarian aid basis. The EU’s compassionate use programmes are instrumental in ensuring continuity of access to drugs until approval and reimbursement decisions are finalised. We propose the creation of a registry of drugs offered on a compassionate use basis; further transparency would allow such programmes to be evaluated and direct patients to sources of treatment.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-015-0306-x) contains supplementary material, which is available to authorized users.

Special Treatment? Flexibilities in the Politics of Regenerative Medicine’s Gatekeeping Regimes in the UK

“…It is certainly notable that many of the regenerative medicine products currently under development or in the marketplace concern rare diseases, and many of these have or seek 'orphan' status, which, while indicating relatively small potential patient populations clearly are seen to represent a business model for some companies which can offer a novel product for unmet needs at a viable, high price. Orphan status brings with it various fee waivers in the market regulation process and, if a marketed product finally results, gives the manufacturer a ten-year monopoly over its production (Hyry et al, 2015). Although a discourse of 'early access for patients' has become routine in the gatekeeping policy networks described here, this cannot be dismissed as purely rhetorical.…”

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“…Вве-дение ОЗ в правовое обеспечение здраво- ФАРМАЦЕВТИЧЕСКИЕ НАУКИ  охранения происходило не одномомент-но, а на протяжении последних 25-30 лет. В разной степени в большинстве этих стран также действуют национальные планы управления редкими болезнями, государ-ственные и частные программы помощи па-циентам с редкими заболеваниями [43] и иные, помимо фармакотерапии, меры поддержки [44]. Тем не менее, в ряде стран мира до сих пор не установлено каких-ни-будь законодательных инициатив по сти-мулированию разработки и производства орфанных ЛС.…”

An overview of Compassionate Use Programs in the European Union member states

“…Previously, the information available publicly on CUP, especially on legislation was limited and in many cases outdated when compared with the available grey literature (6,12,13). However, the current systematic search addresses these gaps by including data from grey literature and peer-reviewed journal articles.…”

“…Many pharmaceutical companies are developing new therapies for patients with rare, life-threatening genetic diseases and are also facilitating access to such drugs (5). Lately, Genzyme Corporation has donated imiglucerase to hundreds of severely affected patients with Gaucher's disease in three large-scale international CUP (6,7). Such efforts by pharma majors to improve early access to life-threatening disease drugs makes it essential to understand CUP and the processes involved.…”

“…CUP are early access programs intended to facilitate the availability of new medicines to patients suffering with life threatening disorders or diseases in the EU member states (Figure 1) (6). In general, CUP are considered in the early stages of the product development cycle where patients get pre-launch access to the investigational drugs or drugs not yet authorized in the country.…”