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OVERVIEW OF REGULATORY INITIATIVES IN THE EUROPEAN UNION TO STIMULATE RESEARCH AND ACCELERATE ACCESS TO ORPHAN DRUGS AND OTHER HIGH MEDICAL NEED PRODUCTS

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Baran-Kooiker A., Czech M., Kooiker C.

Acta Poloniae Pharmaceutica - Drug Research

2019

vol. 76

issue 1

3-17

The European Union (EU) is continuously working towards further promoting and facilitating registration and development of OMPs. The goal of this article is to present a broad overview of existing legislation, programmes and incentives in the EU that allow for accelerated access of orphan drugs and high medical need products, as well as give some historical context and how these programs are interconnected. The authors conducted an explorative review of publicly available sources of regulatory intelligence policy and information on orphan drug and rare disease policies as well as information on accelerated assessment programs in the EU. The authors identified and looked into the following programs and initiatives: orphan drug legislation, accelerated assessment, conditional approval, Priority Medicine (PRIME), compassionate use programs, off-label use and RTU, Medicines Adaptive Pathways to Patients (MAPPs), ADAPT-SMART, HTA initiatives (EUnetHTA, Parallel Consultation), STAMP, IMI and IMI2 and the European Framework Programmes (incl. FP7 IDEAL, Asterix and Inspire). It can be concluded that promoting innovation and early access to medicines has been firmly put on the agenda in the EU, with various programs and initiatives, either national or EU-wide. Hurdles in regulatory science remain present, especially for rare diseases, but fundamental research as done in FP7 could lead to more efficient orphan drug development. Barriers in the field of HTA, pricing and reimbursement still need to be addressed to enhance patient access. The true effectiveness of all these programs will only become clear over time.

Bridging East with West of Europe – a comparison of orphan drugs policies in Poland, Russia and the Netherlands

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Baran A., Czech M., Kooiker C., Hołownia M., Sykut- Cegielska J.

Acta Poloniae Pharmaceutica - Drug Research

2018

vol. 75

issue 6

1409-1422

The goal of this article is to provide an in-depth review of rare disease policies and the reimbursement of ODs in 3 European countries, two EU members (Poland, the Netherlands) and a non-EU one (Russia). A review of publicly available information on rare disorder policies and HTA processes was performed. Experts were consulted for unclear or scarce information. Russia has a five times higher frequency threshold for its rare disease definition than Poland and the Netherlands (both using the EU definition). The Netherlands has vastly increased its disease registries by instituting 300 expert centres via its National Plan, in Poland there are only 6 registries while in Russia one central registry exists. All 3 countries have an HTA process in place, however, the Russian one is relatively undeveloped. The access to ODs in the Netherlands is the broadest with 80 out of 83 EMA approved ODs reimbursed in 2015; Poland reimbursed 49, whereas Russia reimbursed 4 on the federal level and 43 in Moscow region. In all countries new rare disease policies are under development. The availability of healthcare systems solutions and the reimbursement of ODs differs greatly in all 3 countries, mainly in Russia. Even though both states are EU member with common regulations and access to EMA approved drugs, marked differences between Poland and the Netherlands in the range of policies, access to treatments and screening programs exist.

APPLICABILITY OF THE EVIDEM MULTI-CRITERIA DECISION ANALYSIS FRAMEWORK FOR ORPHAN DRUGS – RESULTS FROM A STUDY IN 7 EURASIAN COUNTRIES.

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Baran-Kooiker A., ATIKELER K., GAITOVA K., HOLOWNIA-VOLOSKOVA M., TURCU-STIOLICA A., Kooiker C., PINIAZHKO O., CZECH M.

Acta Poloniae Pharmaceutica - Drug Research

2019

vol. 76

issue 3

581-598

Several Multi-Criteria Decision Analysis (MCDA) models for use in health technology assessment (HTA) have been developed over the years, including some for orphan drugs (OD). However, there is no general consensus yet on MCDA structure and which criteria should be included and implementation of MCDA into HTA practice has been slow so far. Our study tested the criteria preferences and possibilities for implementation of the EVIDEM MCDA framework for OD with a diverse group of 140 stakeholders in Kazakhstan, Netherlands, Poland, Romania, Russia, Turkey and Ukraine (KZ,NL,PL,RO,RU,TR,UA). The research elicited stakeholder preferences (weighting) for EVIDEM domains, criteria and sub-criteria to measure their relative importance. Correlations of work place and HTA/rare diseases experience with weighting results were investigated. Results showed that the ‘Need for intervention’ domain was assessed as the most important in: RO/NL/RU/TR, in KZ/PL the ‘Type of benefit of intervention’ and in UA ‘Economic consequences of intervention’. ‘Population Priorities’ was uniformly given a low priority. Further research, sharing of experiences and multi-stakeholder discussion is necessary to define a path forward for a robust and sustainable improvement of MCDA models and its application in orphan drug HTA. Model simplification and clarification of outcomes would be beneficial.

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3 Acta Poloniae Pharmaceutica - Drug Research

2 2019

1 2018

3 Kooiker C.

2 Baran-Kooiker A.

2 Czech M.

1 ATIKELER K.

1 Baran A.

1 CZECH M.

1 GAITOVA K.

1 HOLOWNIA-VOLOSKOVA M.

1 Hołownia M.

1 PINIAZHKO O.

1 Sykut- Cegielska J.

1 TURCU-STIOLICA A.

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OVERVIEW OF REGULATORY INITIATIVES IN THE EUROPEAN UNION TO STIMULATE RESEARCH AND ACCELERATE ACCESS TO ORPHAN DRUGS AND OTHER HIGH MEDICAL NEED PRODUCTS

Bridging East with West of Europe – a comparison of orphan drugs policies in Poland, Russia and the Netherlands

APPLICABILITY OF THE EVIDEM MULTI-CRITERIA DECISION ANALYSIS FRAMEWORK FOR ORPHAN DRUGS – RESULTS FROM A STUDY IN 7 EURASIAN COUNTRIES.