As 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 Edith Frénoy, Director Market Access/HTA, European Federation of Pharmaceutical Industries and Associations. Total Orphan Drugs would like to take this opportunity to thank Edith Frénoy for speaking to us.
Delivering market access and reimbursement from the industry perspective means defining and discussing value, according to Edith Frénoy, Director Market Access/HTA, European Federation of Pharmaceutical Industries and Associations
From an industry perspective, getting fast access to needed medicines for patients with rare diseases is a priority. Systems to provide access for every patient, including pricing and reimbursement systems must be established to support this goal. For medicines to obtain orphan designation in the marketing authorization procedure in the EU environment, they must demonstrate that there exists no satisfactory alternative or that they provide a significant benefit over existing therapies. But that is just the first step in the process. “The fact remains that in Europe, having a marketing authorization is not sufficient to provide access to patients.” said Frénoy. “Because healthcare systems in Europe, which are primarily publicly- funded, have put in place additional mandatory procedures to set prices and levels of reimbursement.”
EU marketing authorization is done by the European Medicines Agency as one common approval across the 28 EU member states. Orphan designation is linked to a range of incentives in order to support research and development in rare diseases, including market exclusivity.
Pricing and reimbursement decisions are the competence of the 28 individual member states of the EU and each of the member states of the EU has a different system. “Pricing and reimbursement levels provide an important signal to companies on where they should focus their research investments. Another question is how to price a medicine so that it will be affordable for different countries.” said Frénoy. According to Frénoy, orphan disease patients should have access to all medicines that have been approved, particularly given that this is a specific group of patients who have a high unmet need. “Can we develop our systems so that all medicines are accessible and to ensure that the criteria used to support these approval decisions are sound and that budgets at national level are appropriate?” said Frénoy.
In order to accelerate access to medicines for rare diseases and to streamline national processes for pricing and reimbursement decisions, EU member states recently discussed the Transparent Value Framework, a collection of consensus criteria to guide member states in their evaluation and pricing of orphan medicines. These address questions such as what alternatives are available, what kind of clinical improvement is demonstrated, what is the patient response rate, and what is the degree of certainty.
From a European perspective, assessing these aspects of medicine value is challenging, according to Frénoy, because orphan diseases often involve an extremely small patient population, where often the natural history of the diseases is not well known. This makes better sharing of key information sets and international collaboration of experts a necessity. Various initiatives are now being established in Europe, including expert reference networks to share expertise, initiatives to connect patient registries across national borders, and increased dialogue on procedures that involves regulators, patients, payers and industry.
Another important thing to consider, according to Frénoy, is that Europe needs to seriously discuss the concept of solidarity. “If you look at the access that is achieved for patients, you see very striking gaps between access to medicines across European member states.” said Frénoy. “Ensuring that the conditions in Europe are conducive to differential pricing would support equal access for European patients.” This involves creating vetting systems that appropriately measure the value of orphan medicines, developing budgets that allow for appropriate access, and developing mechanisms to ensure “fairness” in budgeting for orphan medicines. “In the end, everybody has a role to play.” Frénoy said.
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 wendywolfsondotcom.wordpress.com and she can be contacted at email@example.com
Read more interviews in this orphan drug series
Partnering from the pharma perspective
How to deliver value to patients
Perspective from patient advocates in Sweden
Interview: Surmounting barriers to patient access
Unique opportunities to develop cures for orphan diseases in the Middle East shouldn’t be overlooked
Where do the current and future challenges lie in bringing new orphan drugs to market?
Bringing Biotech Spirit to a Non-Profit Environment
Paving the way for a whole new medical paradigm
Tailoring your reimbursement strategy, step by step, country by country
An Interview With Pfizer on the Orphan Drug Industry: Part I
An Interview With Pfizer on the Orphan Drug Industry: Part II
An Interview with the Chair of the IRDiRC
An Interview with an Orphan Drug Payer