A two-year debate in the UK around access to Orkambi, a life-extending cystic fibrosis (CF) therapy developed by Vertex Pharmaceuticals and priced at £105,000, recently reached the Parliament floor, surfacing a number of outstanding questions around affordability and the cost of innovation. Meanwhile, in the face of continued denial of access, patients in the UK have started the “CF Buyers’ Club” to gain access to generic versions of Vertex’s portfolio of CF drugs, despite the lack of approval by the European Medicines Agency (EMA) and National Health Service (NHS) in the UK, as well as potential intellectual property (IP) violations.
While the CF Buyers’ Club is controversial in its current form, it may be worth asking: is there an opportunity for originator brands to engage in shaping the Buyers’ Club model to be a sustainable alternative when full reimbursement is not an option?
The generic alternatives mentioned above are developed and shipped by manufacturers in Argentina, where IP laws protecting patents on new chemical entities are much less rigid. The Buyers’ Club then purchases directly from them at a significantly reduced price to branded Orkambi. This importation and use of non-approved drugs raises issues around IP laws, patient safety, and the implications of undercutting the price of innovation in the UK.
However, with a bit of compromise from all sides, could a new orphan drug reimbursement mechanism which sits outside of the rigid ICER-based system (and incorporates learnings from the UK’s Cancer Drug Fund) actually benefit all parties? We believe yes.
Looking closer at the Orkambi example – Vertex is offering the product directly to patients for ~£8,000/month (a slight discount to the ~£8,750/month price which was denied by NHS). The Argentinian manufacturers are reportedly offering it to patients for ~£1,600/mo. If Vertex and the CF Buyers’ Club asked NHS to cover even 75% of the branded acquisition price, the result would be the following:
- Patients save 75% on the branded drug which has been reviewed by EMA and sourced from approved suppliers – the premium to generic is limited to ~£400/month
- NHS is able to provide access to Orkambi at a more cost-effective 31.4% discount to Vertex’s original price and avoid legal battles over IP and importation
- Vertex retains 91.4% of original price and is able to use Buyers’ Club as an opportunity to more effectively engage with CF patient community
Challenges undoubtedly exist to establishing this mechanism, but cost-effectiveness markets like the UK, Canada and Australia will eventually need to shift their approach to reimbursing orphan disease if they hope to sustain innovation. Harnessing the proactivity of patient Buyers’ Clubs may just be the disruption that creates that opportunity.