EU orphan drug regulations haven’t “failed” but reforms needed, says European rare disease advocacy group

Two parents hold their child, who has spinal muscular atrophy 1. Source: EURORDIS - Rare Diseases Europe.

Last week, a report published in The BMJ slammed European drug regulations for allowing biopharmaceuticals to reap "billions" in profits on new drugs for rare diseases, but failing to incentivize sufficient R&D into new cures and treatments. A leading patient advocacy group, however, says that while the regulations may be in need of reform, they have helped advance treatments for many debilitating and deadly rare diseases.

European legislation on orphan drugs is ‘not a failure’. European legislation on drugs for rare diseases is “far from perfect”, but it is not a ‘failure’ given the amount of private sector investment it has attracted in the past two decades, says Yann Le Cam, CEO of EURORDIS – Rare Diseases Europe, a non-profit alliance of some 930 rare disease patient organisations across 72 countries, in response to the BMJ report.

Le Cam said the report’s conclusions are simplistic, ignoring the big achievements that have been made since the regulations were put in place two decades ago.

“Twenty years ago, pulmonary arterial hypertension used to be fatal. Today, 7 treatments are approved in Europe, largely because of EU and US orphan drug regulations. The life of patients has changed,” said Le Cam.

EURORDIS - Rare Diseases Europe

Unsubstantiated conclusions. Last week’s investigative report claims that only 18-24 of the 142 orphan drugs that had been approved between 1999 and 2017 were developed as a result of the European Union Orphan Regulation, which grants new drugs for rare diseases “market exclusivity” for at least a decade - a benefit stronger than standard patent protections. However, there is “no data” to back up this “opinion”, said Le Cam.

European Member States need to pull their weight. Le Cam stressed that European Member States need to adopt a more collaborative approach to post-marketing evaluations, paving the way for coordinated price negotiations over such drugs by countries as well. This would hit several birds with one stone, such as:

  1. Improving knowledge about how well new drugs really work in patients with rare diseases. Many drugs for rare diseaes are ‘stabilizing’ rather than ‘curative’, and by nature they may be approved after relatively few trials. So more coordinated post-market assessment can see which drug work the best among the patients living with a ‘dreadful disease’, said Le Cam.

  2. Allowing European Member States to re-negotiate drug prices based on their value in patients. Today, it is impossible to price an orphan drug based on its “real-life impact in patients” because the data simply isn’t there.

  3. Improve access to orphan drugs in smaller countries and specific demographic groups. Currently, Member States tend to negotiate orphan drug prices on their own, rather than in regional blocs. As a result, smaller nations may have to wait for years to gain access to an orphan drug, even though a larger country right next door already has it available.

“We can blame the private sector as [the BMJ] report does, but we also need to blame member states for their fragmented approach to generating evidence on orphan drugs after they’re approved”, said Le Cam. “As a result, we still don’t know the real-life value of treatments in patients and are unable to adjust prices accordingly.”

“Today, data collected in the region is not robust enough to allow different treatments to be compared between each other….Pooling data across Europe with the same research questions and protocols would be good science and better use of your money.”

Quick facts on rare diseases. Rare diseases are defined as ones that affect less than 5 in every 10,000 people.

  • There are approximately 6000 rare diseases worldwide.

  • It is estimated that at least 30 million people in the EU are affected by a rare disease.

Health Policy Watch EU Orphan Drug Regulations Haven’t “Failed” But Reforms Needed, Says CEO Of European Rare Disease NGO