Access to orphan drugs

A good medication for rare disease patients is a medication that is both available in the country where they live and affordable.

The drug is of little use if one of these two factors is missing. Due to costs, we unfortunately observe this far too often in the EU today.

Several factors contribute to this situation. Orphan drug designation, protocol assistance and marketing authorisation belong to a centralised procedure. However therapeutic value assessment, pricing and reimbursement for these innovative products remain within the realm of Member states' responsibility.

Member state pricing negotiations

In the case of the therapeutic value assessment, not all Member states have the expertise to perform such complicated evaluations. Each state negotiates pricing separately with the pharmaceutical companies. Sometimes companies tend to begin negotiations with Member states that grant a higher price - which is then used as a reference price in their negotiations with other countries.

Member states delay negotiations as much as possible in order to avoid having to make the drug available and therefore having to pay for it. Although the situation could appear in favour of both pharmaceutical companies and the Member states, it is not so.

Member states end up paying higher prices for the drugs, and companies lose part of their ten-year market exclusivity because negotiations may take up to four years.

EURORDIS began denouncing this situation in its first survey on orphan drug availability presented to the Commission's Transparency Committee in 2004. The Commission's report on five years of the Orphan Drug Legislation also mentions this survey.

EURORDIS has conducted 3 surveys on access to orphan drugs:


Improving access to orphan medicines

EURORDIS continually works to identify the main blocks encountered in patient access to orphan medicines and proposes concrete measures to overcome them.

This work was started by EURORDIS and others at the Working Group on Pricing of the EU Pharmaceutical Forum in 2007 (read the EU Pharma Forum Guiding Principles on Improving access to orphan medicines for all affected EU citizens).

The most important hurdle identified was the one on pricing and reimbursement. National authorities are often reluctant to reimburse often costly orphan drugs that require extensions of indications and a life-long need for treatment. Nevertheless, other factors also come into play:

  • The priority (or lack of) given to rare diseases and orphan drugs in the health budget
  • The extent to which authorities are able to monitor and manage use, in order to control budgets despite high prices.


Pricing and reimbursement: Rising to the challenge

To estimate how many new orphan drugs can be expected to receive marketing authorisation in the next decade, EURORDIS performed an analysis based on the US experience since the US Orphan Drug Act was adopted. This was initiated as a response to fears about a potential deluge of new orphan drugs and their cost to society. (Read EURORDIS Statement "Orphan drugs: rising to the challenge to ensure a better future for 30 million patients in Europe”).

The final conclusions and recommendations on Pricing and Reimbursement of the EU High Level Pharmaceutical Forum was that “while the EU Orphan Drugs Regulation 141/2000 has stimulated research and development of orphan medicinal products in the EU, equitable and timely access to approved Orphan Drugs for rare diseases patients remains an issue, and effective market access and utilisation vary strongly between and within Member States”.

The most important measure recommended by consensus by all Member States, industry and patient groups in the framework of the EU Pharmaceutical Forum – was to produce a common scientific assessment report for the clinical added value of orphan drugs. This report is intended to be used by Member States to base and accelerate their decisions on reimbursement.

EURORDIS has conducted several surveys on the real access to OMPs in EU countries to highlight where there are needs to be addressed.
Increasingly, EURORDIS has been involved in addressing the issue of pricing and reimbursement of OMPs and access to patients. We have participated in many levels of discussion within different EU platforms and fora, encouraging debate with all relevant stakeholders.
EMA high-level management, the COMP, the European Commission as well as the Task Force of pharmaceutical laboratories developing OMPs have contributed, together with EURORDIS and other patient groups, to the publication of policy documents calling for improving timely access to approved OMPs in all EU Member States. Workshops of the EURORDIS Round Table of Companies have been dedicated to the analysis of patients’ access to orphan products as well as to the elaboration of the CAVOMP Information Flow process in a meaningful and acceptable manner for all parties involved.
Towards additional European collaboration

In 2010, Ernst & Young was mandated by the European Commission to conduct a study into the feasibility of creating a mechanism for the exchange of knowledge of the clinical added value of OMPs (CAVOMP). The outcomes of the study and proposed scenarios were presented at the EUCERD in 2011.
Based on Ernst & Young’ study, the EUCERD members discussed the best mechanism to be put in place for the CAVOMP. The different stakeholders sitting on the EUCERD, namely experts as well as representatives from the 27 EU Member States, EEA countries, industry and EURORDIS, have discussed for more than one year, on the best mechanism for the CAVOMP Information Flow to be implemented at both EU and Member States (MS) levels.

EUCERD recommendation for a cavomp information flowThe adoption of the EUCERD Recommendation on the CAVOMP Information Flow to the European Commission and the Member States is the latest  corner stone in the long process of developing and marketing OMPs intended for patients living with rare, heterogeneous, severe and life threatening diseases.  Read the “EUCERD Recommendation on improving informed decisions based on the Clinical Added Value of Orphan Medicinal Products (CAVOMP) Information Flow to the European Commission and Member States”.









Page created: 14/03/2012
Page last updated: 16/01/2018
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