Improving patient access to orphan drugs in Europe

Read also the 2007 Eurordis survey on orphan drugs

A good medication for rare disease patients is a medication that is both available in the country where they live and affordable. If one of these two factors is missing, the drug is of little use. Unfortunately, for cost reasons, this happens far too often in the EU of Europe showing orphan drug availability | carte de l'Europe | mapa de Europa | cartina dell'Europa | Diagramm von Europa A 2004 Eurordis survey on the availability of twelve orphan medicinal products having received a market authorisation in the EU shows that, one year later, Denmark was the only country where all twelve products were available to patients. The number varies greatly in other countries, with the new EU Member states and Luxemburg lagging behind with less than four products available. Too often, when the products are available, patients cannot afford them. In Belgium for example, Busilvex, a conditioning treatment used prior to conventional haematopoietic progenitor cell transplantation, costs more than 4,400 euros and is not reimbursed at all. 'This situation is not only inequitable because patient access to orphan drugs depends on the country where they live, but it is also totally unethical,' argues Yann le Cam, CEO of Eurordis. 'European regulations benefiting patients exist, but they are not applied.' The legal delay for placing medicinal products on the EU market is 180 days [Council Directive of 21/12/1998]. However, the average delay is 189 days at the moment, and some drugs in some Member states take up to 700 days before being placed on the market. 'This means that orphan drugs are not available to patients within the legal time limit. It also means they are made available in a timeframe and under conditions of access than are worse than for other drugs,' says Yann le Cam. And, worst of all, it means that patients cannot access drugs that would be of benefit to them; as a consequence, their lives are threatened.

Euros Several factors contribute to this shocking situation. Orphan drug designation, protocol assistance and marketing authorisation are centralised processes, but therapeutic value assessment, pricing and reimbursement for these innovative products remain the Member states' responsibility. In the case of the therapeutic value assessment, Member states don't have the expertise to do it. Each state negotiates pricing separately with the pharmaceutical companies. There is a tendency for companies to start negotiating with Member states that grant them 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 to avoid having to make the drug available and pay for it. Although the situation could be seen to be 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 of negotiations that take up to four years!

Eurordis has been denouncing this situation since its first survey on orphan drug availability, which was presented to the Commission's Transparency Committee in 2004 and is mentioned in the Commission's report on five years of the Orphan Drug Legislation. To improve patient access to orphan drugs, Eurordis is suggesting establishing an EU working group for orphan drug transparency made of volunteer Member states, which would have the expertise to assess the therapeutic added value of each orphan drug and negotiate an EU ex-factory reference price with the marketing authorisation holder. The group would start working straight after the CHMP1 gives its opinion, without the need to wait for the Commission's decision. It would provide an opinion that all Members states could immediately use for their pricing and reimbursement negotiations. The benefits for Member states would be a lower negotiated price and the respect of the legal delay for orphan drug availability; for pharmaceutical companies, it would mean earlier return on investment and more visibility and predictability of returns; for patients - and that's the whole point - earlier availability of orphan medicinal products. The proposal has been presented to the Commission, the COMP2 and the industry.


graph showing orphan drug availability by country | graphique montrant la disponibilité de médicaments orphelines par pays | gráfico que demuestra la disponibilidad de los medicamentos huérfanos | grafico che mostra la disponibilità dei farmaci orfani | gráfico que mostra a disponibilidade de medicamentos orfãos | Diagramm, welches die Verwendbarkeit der Arzneimittel für seltene Krankheiten zeigt

What’s next? Discussions are currently underway with some Member states; the proposal will be presented soon to the Medicine Evaluation Committee (MEDEV3); and discussions will take place with pharmaceutical industry federations and the European Commission. Eurordis’ objective is to obtain the agreement of a few Member states to move the proposal further - a proposal that is flexible and remains open to suggestions, as long as patient access to orphan medicinal products remains the central and final objective.

1Committee for Medicinal Products for Human Use (EMEA)
2 Committee for Orphan Medicinal Products (EMEA)
3The MEDEV is an informal committee of Belgium experts representing the social insurance organisations and national bodies responsible for the assessment and reimbursement of medicines.

This article appeared in the April 06 issue of our newsletter.

Author: Jerome Parisse-Brassens
Photo credits: © European Community, 2006
Graphics: © Eurordis

Page created: 19/08/2009
Page last updated: 13/03/2012
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