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:

• 2004 Survey – 12 orphan drugs in 26 countries
• 2007 Survey - 22 orphan drugs in 28 countries
• 2010 Survey – 60 orphan drugs in 10 countries

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.

Working Party for European collaboration

EURORDIS, industry and academic leaders in the field of orphan drugs developed a proposal to the European Commission and the European Medicines Agency (EMA) to establish a Working Party for European collaboration toward common scientific assessment of the clinical added value of orphan drugs ([1] CAVOD).

The proposed Working Party would facilitate collaboration amongst EU level authorities and Member States in order to make the most of already existing information, to help national health authorities make their pricing and reimbursement decisions, thus improving effective access to orphan drugs by rare disease patients.