While all rare disease products must get a European marketing authorization via a centralized process, whether or not they receive reimbursement is the decision of each member state or healthcare system, according to Celgene’s Kevin Loth. In the past 12 years 80 orphan medicinal products have received European marketing authorization and over 1000 products received an orphan designation. Meeting the requirements of the regulatory authorities involves early dialogue, gathering scientific advice, and close cooperation with the regulatory authorities—seemingly common sense stuff. “There is another set of discussions that we need to go through for every healthcare system to make sure those products actually get made available to patients.” Loth said.
According to Loth, reimbursement strategy must address what is needed to demonstrate value in an individual healthcare system. It is simplistic to look only at country GDP or what proportion a country spends on healthcare within Europe as each healthcare system is run and financed differently. For example, healthcare systems in the UK have been heavily influenced by cost-effectiveness and health technology assessment. Thus in developing your reimbursement dossier you would emphasize cost effectiveness data. An Italian healthcare system may want a demonstration of clinical effectiveness, or interested in registries to show that your product is delivering clinical results. Other countries may be interested in total budget impact. “So those are three very different strategies, irrespective of the GDP of the country.” said Loth. “For example, you have low GDP countries applying cost effectiveness, and high GDP countries interested in total budget impact.”
The investment in rare diseases varies as well. Some countries have made rare diseases a public health priority. Others have invested heavily other areas like cancer or mental health. According to Loth, as more therapies become available for a rare disease, the market dynamic changes. Rare diseases cover a huge span of conditions; some are more common than others, or have existing therapies, well-recognized healthcare or social interventions. This is a different proposition than if the disease is not well understood, has no treatment and is perceived as having a cost to a system.
The EMA has expanded the role of patient representatives on their regulatory committees and expert advisory groups. Industry is also trying to work more closely with patients in earlier stages of drug development, according to Loth. Rather than retrospectively demonstrating the benefits of a product to patients and using abstract endpoints, companies are using endpoints upfront with which patients can immediately identify. If that is done early and prospectively with the approval and buy-in regulator and reimbursement authorities, it is easier to not only meet the regulatory requirements to get market authorization but more closely demonstrates the value required to get reimbursement and the support of patients.
Loth has a few suggestions for policy development:
1. Expand the network of patient reference centers
Centers for rare diseases are being set up by the 27 European member states to implement national health plans for rare diseases. This is a well-established concept for other diseases areas such as oncology. But for some diseases that are extremely rare, it would be helpful to have European reference centers that connect national centers of rare diseases to each other and to centers in other countries, to give patients access to the best possible diagnosis, access to expertise and treatment.
2. Address patient access early in the process
Stakeholders, whether regulators, the company or payors, acknowledge that patients need to be more closely involved in drug development, drug licensing and reimbursement early on.
3. Solve systemic problems in parallel when possible
Rare disease regulation is still very new in Europe. These interrelationships have to be thought of together and worked on in parallel rather than thought of as a series of consecutive hurdles to overcome. For policymakers, it is no longer sufficient to develop a drug and then a marketing authorization as that means nothing in terms of patient access, given it can be many years until a patient can access a drug.
About the company
Celgene International Sàrl, located in Boudry, Switzerland, is a wholly-owned subsidiary and international headquarters of Celgene Corporation. Headquartered in Summit, New Jersey, Celgene Corporation, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit www.celgene.com.
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