WEBINAR: The orphanization of medicine: Are we seeing fundamental changes in the development, use and governance of medicines

At this webinar, Prof. Paul Martin will outline how a marked shift in the development, use and regulation of medicine has happened during the past couple of decades possibly turning the ’rare’ into the ’new normal’. Prof. Martin’s talk is followed by a response by Dr. Steffen Thirstrup who is Chief Medical Officer at the European Medicines Agency (EMA). The webinar is organized jointly by the Danish Center for Social Science Research – VIVE and the Global Pharmaceuticals and Society (GPSS) Network.

 

When: 24th of August at 3:00-4:30 pm CET

 

Where: Online – a zoom link will be sent out in advance to all who register for the event

 

Registration: Please sign up before the 23nd of August at 3 pm CET through this link.

 

Abstract for Prof. Martin’s talk:
A series of major transformations and transitions are underway in the production, regulation, marketing and use of medicines. This impacts on both the pharmaceutical industry itself, as well as the organisation of healthcare and the relationship between companies, professionals and patients. These changes centre around the rapidly increasing importance of advanced therapies and so-called “orphan drugs” for rare diseases, named after the 1983 US Orphan Drug Act (ODA). Over the last two decades orphan drugs have moved centre stage within the pharmaceutical sector and now constitute more than half of new drug approvals by the US FDA each year. Advanced therapies (ATMPs) based on tissue engineering, cell and gene therapy represent the next generation of therapeutic innovation and many also target rare diseases. Accompanying the development of these new technologies have been a number of key changes, including: 1) Novel industry business models that seek to create highly priced products for niche markets; 2) New regulatory regimes including expedited review processes, conditional approval and new forms of evidence; 3) Innovative technology assessment and reimbursement models; 4) The establishment of services and infrastructures to support the use of highly specialised orphan drugs and complex advanced therapies; 5) Changing relationships between the pharmaceutical industry and rare disease patients; and 6) New discourses of disease aetiology that place greater emphasis on the genetic origins of disease. Collectively these changes can be seen as constituting a transition in the sociotechnical regime associated with the pharmaceutical sector that has much wider implications beyond the field of rare diseases. It raises profound questions about access, equity and the long term sustainability of healthcare systems and the industry itself. This paper will establish a robust conceptual framework based on the notion of “orphanisation” to analyse these changes and their implications for the future of the pharmaceutical industry, the governance of innovation, the structure of healthcare systems and the biopolitics of contemporary medicine.

 

 

Abstract for Dr. Thirstrup’s response:
Medicines to treat, prevent, or diagnose rare diseases are considered orphan drugs by regulatory authorities such as US-FDA and EMA when fulfilling certain criteria. These criteria are, however, not identical among different authorities. To obtain orphan drug designation in EU the condition must fulfil the following criteria: (1) the disease is life-threatening or chronic debilitating, (2) the prevalence in EU is less than 5 in 10.000, and (3) there is not other medicine approved for the condition or if there is, the new medicines will have to add a significant benefit to the patients. Medicines developers can apply for orphan designation during development and if granted this will give access to scientific advice and other services at EMA with a fee reduction. If the product in the end obtains a marketing authorisation, and still fulfils the requirement for an orphan designation, the product will have 10 years’ market exclusivity in EU. EU regulators have generally been reluctant to accept subsets of a disease as being an orphan condition (i.e., not allowing for salami slicing the indications) unless the subsets are individually recognised as clinically distinct conditions. Currently there is around 150 orphan drugs authorised in EU and despite each of them are targeting small populations the cumulative cost of treating rare diseases is a challenge for many national health care systems.