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September 2022

EURORDIS Chief Executive Officer addresses Health Ministers at WHO to improve access to medicines for rare diseases


For the first time, Yann Le Cam, Chief Executive Officer of EURORDIS-Rare Diseases Europe, addressed 26 European Health Ministers at the WHO Regional Committee for Europe on their role in improving access to medicines for people with rare diseases.

Yann Le Cam spoke at the WHO Regional Committee for Europe Meeting in Tel Aviv on Monday 12 September. He addressed 26 Health Ministers from across the wider European region on the role that they could play in improving access to medicines for people living with rare diseases. This is an important milestone for the whole rare disease community, whose voice was heard at the level of global decision makers. 

At the meeting, delegates adopted a WHO Europe Statement, giving a mandate to WHO Europe to create a Joint Stakeholder Platform and take concrete actions on access to effective, novel, and high-priced medicines. Given the current geopolitical and economic circumstances, leaders from around Europe believe that now is the right time, and the WHO is the right forum to be discussing and pushing these actions that EURORDIS has pushed for a long time. 

This statement builds on the work carried out by the Oslo Medicines Initiative – WHO in 2021, aiming at fostering multi-stakeholder dialogue to reshape political discourse and create partnerships to build a movement for change. EURORDIS participated actively in the Initiative, through responses to consultations, interviews, meetings with advisors, participation in webinars, and in sharing its proposals. 

Addressing the delegates, including Director General of the WHO, Dr Tedros Adhanom Ghebreyesus, Regional Director of WHO-Europe Dr Hans Kluge, 26 European Health Ministers and 600 other participants, Yann Le Cam said 

“Among the novel, effective, high-priced medicines, those for rare diseases are impacted and a growing part. Science and industry investments are delivering lifesaving medicines or disease transforming therapies for unmet medical needs, particularly gene and cell therapies.”

It is expected that in the next 5 years, 20-30 new transformative therapies will be approved to treat 10 or 50, or 500 patients, or a few hundreds of thousands. Their timely availability will depend on the political will that governments are able to muster in favour of more collaborative policies, to drive more equitable outcomes.

The current fragmented European market doesn’t work for these new medicines. Only a few countries will be served. We are ready for failure, drama and media scandals, and having thousands of patients while they are waiting. Their challenges can only be addressed through European collaboration.”

The concrete proposals put forward by the rare disease community included: 

  • Collaborating now on the very low prevalence diseases and complex treatments
  • Organising structured access for very rare diseases to go beyond our entrenched barriers
  • Using the unique European solutions at hand, such as a centralised EMA and HTA framework, European Reference Networks for rare diseases, and evidence emerging from pilot trials on the collection of Real-World Evidence and between payers. 

In order to succeed in this ambitious endeavour, all policy makers at national, European and global levels should work together towards greater solidarity, transparency and sustainability, promoting open decision-making and sustainable industry and health systems.

“What we need now is for Member States’ buyers to come together. To have a one-stop shop. To have one European negotiator, using the strength of their collective bargaining power”,  added Le Cam. “Circumstances are unique and offer opportunities which cannot be missed. You have the power in this room to form the ‘Coalition of the WIlling’, to develop a European Model of Care with greater solidarity between the Member States, more equity across citizens in Europe which in turn will make Europe more attractive, whilst leaving no one behind.”

The adoption of this WHO Statement is a positive step forward, and EURORDIS looks forward to participating in the Joint Stakeholder Platform in the months to come to deliver on the promises of the milestone document.

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