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

Balasubramanian, Gayathri; Morampudi, Suman; Chhabra, Pankdeep; Garg, Arun; Zomorodi, Behsad

doi:10.5582/irdr.2016.01054

Cited by 58 publications

(66 citation statements)

References 12 publications

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“…According to Balasubramanian et al, 20 out of 28 EU member states had an established compassionate use programme (CUP) (82). A CUP exists in all EU countries selected for this review, except Poland (work on implementation of a national CUP is ongoing) 23 (82).…”

Section: Rare Disease Policies and Access To Orphan Drugsmentioning

confidence: 99%

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Czech¹,

Baran-Kooiker

Atikeler

et al. 2020

Front. Public Health

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Section: Rare Disease Policies and Access To Orphan Drugsmentioning

confidence: 99%

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Czech¹,

Baran-Kooiker

Atikeler

et al. 2020

Front. Public Health

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“…Furthermore, most, but not all, EU member states have an established compassionate use program, with different regulations and access criteria, adding to disparity within Europe. 17 …”

Section: Regulatory and Systemic Issuesmentioning

confidence: 99%

Access to Affordable Orphan Medicines in Europe: An EHA Position Paper

et al. 2020

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O rphan medicinal products (OMPs) are pharmaceuticals designed for the treatment of rare diseases, a family of more than 6000 medical conditions, each of which affects a small to ultra-small population of patientstypically less than 1 in 2000 people. 1 The June 2009 European Council Recommendation on action in the field of rare diseases emphasizes that "the principles and overarching values of universality, access to good quality care, equity and solidarity [...] are of paramount importance for patients with rare diseases." Although important advances have been made in the OMP field during the last decade, several deadlocks still impede optimal accessibility. 2 Disparities in the availability of OMPs in the EUA survey conducted in 12 Eurasian countries on rare disease policies and orphan drug reimbursement systems showed that inequality in patient access to new OMPs still exists due to variations in national healthcare budgets, health insurance, and reimbursement systems. 3 Fragmentation of reimbursement policies translates into unequal access to treatment. The number of reimbursed OMPs ranged from >100 in Germany, France, Italy, and the Netherlands to zero in Armenia. For instance, France reimburses 116 orphan drugs, England 68, Scotland 55, and Wales 47. A survey of access to OMPs in the United Kingdom, France, Germany, Italy, and Spain found that since the implementation of the OMP Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorization in the European Union. 4 These OMPs are most widely accessible in Germany and France, while in the other countries between 30 and 60% of OMPs are reimbursed. In addition, in some countries, such as Italy, differences in treatment availability and access to medicines, diagnosis, and care can occur regionally, depending on each regional health management system. The cost of OMPs is clearly a major factor explaining the disparities among different European countries. 5 The complex issue of drug pricing is dealt with in an accompanying European Hematology Association (EHA) position paper, 6 but here we will briefly consider the issues related to OMP prices. In addition, differences in normative approaches to rare diseases and OMPs between countries also play an important role.

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“…While these regulations differ by country, in principle, expanded access treatment can be performed in patients with serious, including life‐threatening diseases who cannot be treated satisfactorily with authorized drugs. In addition, the use of an investigational drug must be supported by sufficient evidence of its safety and efficacy, and a doctor must obtain informed consent from the patient .…”

Section: Expanded Access and Off‐label Applications Of Drugs: Generalmentioning

confidence: 99%

Ethics codes and use of new and innovative drugs

Borysowski

Ehni

Górski

2019

Brit J Clinical Pharma

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Treatment with new and/or innovative drugs with uncertain safety and efficacy profile is associated with substantial ethical concerns. The main objective of this paper is to present guidance on the use of such drugs contained in: (i) major international codes and guidelines pertaining to medical ethics and biomedical research; (ii) national codes of medical ethics and professional conduct of the USA, Canada, Australia, New Zealand, the UK, Ireland, France and Germany. Out of the four international codes and guidelines analysed, only the Declaration of Helsinki addresses the question of the use of unproven drugs. Among national codes, only two (USA and New Zealand) explicitly allow for use of new or innovative drugs. Moreover, treatment with unproven drugs seems to be permissible under the French code, though this is not stated explicitly. The remaining codes do not contain any articles on the use of new and innovative drugs. An update of existing articles, as well as the addition of new guidelines to the codes, should be considered in view of the rapid pace of development and introduction to clinical practice of new drugs. This work is relevant to innovative off‐label applications of approved drugs and expanded access to investigational drugs.

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

Cited by 58 publications

References 12 publications

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Access to Affordable Orphan Medicines in Europe: An EHA Position Paper

Ethics codes and use of new and innovative drugs

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