Rare disease community calls for radical change to improve patients’ access to medicines

EURORDIS position paper on access to rare disease therapies in Europe

EURORDIS and its over 700 member patient organisations have launched a new position papercalling for urgent change to ensure patients’ full and fast access to rare disease therapies in Europe.

The ‘Breaking the Access Deadlock to Leave No One Behind’ paper is a contribution from EURORDIS and its members and follows a reflection process initiated at the EURORDIS Symposium on Improving Patient Access to Rare Disease Therapies, held in February 2017.

It sets out a new four-pillar approach to tackling the challenges that prevent patients’ access to care and medicines, as well as the ambition to have 3 to 5 times more new rare disease therapies approved per year, 3 to 5 times cheaper than today by 2025.

Why is a new approach needed to improve access?

Today’s science and technology create unprecedented opportunities to address the unmet medical needs of people living with a rare disease. Before this potential can be translated into actual health benefits, issues around access are creating a deadlock.

The majority of people living with a rare disease have delayed or no access to the medicine they need, or no medicine even exists. If a therapy is approved for market but does not reach those who need it, it has failed in its primary purpose.

In the paper, EURORDIS calls for a new model based on a collective conversation involving all stakeholders (patients, the pharmaceutical industry, national competent authorities, national health ministries, researchers, scientists and regulators).

Yann Le Cam, EURORDIS Chief Executive Officer, commented, “When it comes to access, we refuse to take no for an answer. We will not cave under the weaknesses of the current model. We, rare disease patients, are not the problem: we are part of the solution”.

He added, “We need to close the gap between innovation and access. All stakeholders have an urgent collective responsibility to shape a new approach that will accelerate the translation of major scientific advancements into new therapies.”

Four pillars to success

The new position paper sets out a four-pillar approach that encompasses:

A new blueprint to cut costs and fast-track R&D (read more);
Early dialogue and cooperation between healthcare systems on the determination of value of a medicine and on patient access (read more);
A transparent European cooperation framework between national healthcare systems for the determination of fair prices and of sustainable healthcare budget impacts (read more); and
A continuum approach to evidence generation linked to healthcare budget spending (read more).

The paper also dispels misconceptions around the pricing of orphan medicines and sets out the facts on marketing authorisation and pricing of rare disease medicines.

Eva Bearryman, Communications Manager, EURORDIS