Interview: Speeding the path of assessment for rare disease drugs in Europe

In Market Access by Freya Smale

download, Dr. Segolène Aymé, Orphanet, Speeding the path of assessment for rare disease drugs in EuropeAs part of a series of interviews conducted with leading industry experts in the run up to the World Orphan Drug Congress in Geneva, Total Orphan Drugs was delighted to sit down with Dr. Segolène Aymé, Orphanet Emeritus Research Director at the French Institute of Health and Medical Research (INSERM) and founder of Orphanet ( Total Orphan Drugs would like to take this opportunity to thank Dr. Segolène Aymé for speaking to us.

Earlier collaboration part of policy fixes for orphan drugs, according to Dr. Segolène Aymé, Orphanet Emeritus Research Director at the French Institute of Health and Medical Research (INSERM)  

Two initiatives are underway in Europe to coordinate the system that approves orphan drugs and assist companies in avoiding misunderstandings on what is expected by regulators. The Clinical Added Value of Orphan Medicinal Products (CAVOMP) Information Flow, and the Mechanism of coordinated access to orphan medicinal products (MoCA-OMP). As companies develop their regulatory dossier, they are encouraged to share information with the people who will be involved later in the process of market authorization. According to Dr. Segolène Aymé, it is better to start working with regulators as early n the process of developing orphan drugs as possible.  “It is a mutual process.” said Aymé.  “Even the regulators have to better understand what happens at the company level. Both sides benefit from this early contact.” 

By developing a network of agencies willing to collaborate to make a common assessment, it is hoped that patient access to orphan drugs will improve, and distribution of the drugs will be more equitable and efficient.  “We propose to really speed the path of scientific assessment and the decision process that is generally left to the countries, which is an essential part of the evaluation.” said Aymé.  “To save time, our goal is to have a recognition that this assessment is good enough to be used at a national level.”

According to Ayme, it is no longer acceptable for countries with economic constraints to be asked to pay for drugs without clearly demonstrated benefit. “I think that the trend that everybody knows that we want to support innovation, and we have to pay for innovation.” said Aymé. “Repurposed drugs are not innovation so should not be seen in the same way.”   

According to Aymé a judicious approach to classifying drugs for reimbursement and reimbursing only for truly novel, innovative drugs is essential with an increasing number of entrants into a maturing orphan drug market. There are real limits to countries’ ability to pay, so effectively either the market price should be capped or fewer patients will have access to medications.

“In the discussions that I hear around me multiple people think there should be limitations in terms of size of the market where the drug will lose its orphan drug status when it is an extension to or when it is exactly the same drug.” said Aymé.

According to Aymé, countries with publicly funded health systems do not want to overly protect products that would have been successfully developed otherwise without regulation. “We have to be careful because there are people who oppose this regulation.” said Aymé. “I think that it is for the industry to be reasonable and to accept capitation in a country where the government says that that there is a maximum that it can pay for this drug.” But according to Aymé, capitation of either the number of patients or number of drugs is intrusive. She instead favors deciding when a market size converges to become a normal market large enough for products judged on conventional criteria.    

“The other idea which is floating around, is that it is shocking to see that the prices do not decrease after the 10 years exclusivity.” said Aymé. “People accept to overpay because of the difficulties of innovation. After a certain time normally, the companies should have had enough benefit to recover and be able to invest in other research. I don’t know if it is 10 years or 15 years but this has to happen.”

Aymé likens the increasingly crowded orphan drug field to the area of hemophilia drugs where there are too many drug entrants that were too similar to each other. “That particular market is maturing.” said Aymé. “The first drugs appeared in 1997. Fifteen years later, there are many contenders.”  

“At least we know the problems, and know the directions of what to go and almost nothing is solved, but we are on track.” said Aymé. “We have the diagnosis and treatment. The orphan drug regulatory framework being developed in Europe is now being applied elsewhere. “The situation is bad for the patients in some countries.” said Aymé. “They cannot access some drugs.  I try to promote the solutions we have identified in Europe to get ideas and examples in South America and Asia. It is inspiring when you see that some countries have successfully established.”

imageSpecial thanks go to Wendy Wolfson who conducted the interview on behalf of Total Orphan Drugs.

Wendy Wolfson covers innovations at the intersection of medicine, science and healthcare as a columnist for Chemistry & Biology, a publication of Cell Press. She has contributed to magazines including Science, Nature Biotechnology, the Lancet, and Red Herring. Her work can be found at and she can be contacted at