Access Mechanisms for Orphan Drugs: A Comparative Study of Selected European Countries

OHE Briefing, No. 52, October 2009

32 Pages Posted: 27 Aug 2015

See all articles by Martina Garau

Martina Garau

Office of Health Economics

Jorge Mestre-Ferrandiz

Office of Health Economics

Date Written: October 1, 2009


OBJECTIVES This study compares the pricing and reimbursement (P&R) arrangements implemented in selected EU countries to make coverage decisions on orphan medicinal products (OMPs) and investigates whether these measures have had an impact on their availability.

METHODS We collected evidence on P&R systems and specific OMPs policies in France, Germany, Italy, Spain, Sweden, the Netherlands and the UK (differentiating where relevant between constituent countries) through a literature review and a consultation of national experts.

We also collected data on coverage decisions on the first 43 OMPs approved by the European Medicines Agency (EMEA) in the first eight years of the Orphan Drug Legislation, which has been in force since 2000. Data sources for this analysis included an IMS database, health care bodies’ websites and a consultation of national experts.

RESULTS Criteria informing coverage decisions vary substantially across countries. However, the most recurrent factors deemed important when making decisions on OMPs were the severity of the illness and the lack of an adequate alternative treatment. The main concerns among decision-makers were around the limited evidence base available for OMPs at the time of their evaluation and the high cost per patient associated with OMPs.

We found that in six of the selected countries, the large majority (or all) of the EMEA-designated OMPs were considered eligible for reimbursement or prescribed within the National Health System (NHS). In countries where a formal health technology assessment (HTA) process is in place, OMP deliberations were varied. Within the UK, a large proportion of the Scottish HTA body’s decisions were either rejections (46%) or involved some restricted use (11%) i.e. recommended for use only in some subgroups of the licensed population. In England and Wales, the National Institute for Health and Clinical Excellence (NICE) has to date appraised only one OMP, which was recommended in both its indications.

Sweden is the country with the highest rate of rejection where almost 30% of the OMPs launched in the country were not reimbursed.

Specific policies for the implementation of post-launch studies for OMPs were identified in all countries but Germany. However, Italy and the Netherlands were particularly active in adopting innovative schemes to allow early access and evidence generation in real-world settings.

DISCUSSION With an increasing demand for HTA by health care decision makers, OMP manufacturers will have to provide an expanded evidence package to show the value for money of their products. This might be problematic given the high level of uncertainty around clinical evidence of OMPs, particularly near the time of launch. As more OMPs will obtain regulatory approval on an accelerated or conditional licensing basis by licensing bodies such as the EMEA, this issue is likely to exacerbate. Also, additional pressures on national health care budgets will present a challenge in terms of the affordability of these drugs in the medium to long term.

Given the low and patchy distribution of rare diseases across regions/countries, more cooperation at the European and international level is required to develop robust evidence. Post-marketing data collection to inform subsequent re-assessment can represent the way forward to address uncertainty and knowledge gaps.

Suggested Citation

Garau, Martina and Mestre-Ferrandiz, Jorge, Access Mechanisms for Orphan Drugs: A Comparative Study of Selected European Countries (October 1, 2009). OHE Briefing, No. 52, October 2009, Available at SSRN:

Martina Garau (Contact Author)

Office of Health Economics ( email )

12 Whitehall
London, SW1A 2DY
United Kingdom

Jorge Mestre-Ferrandiz

Office of Health Economics ( email )

12 Whitehall
London, SW1A 2DY
United Kingdom

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