- Vertex Pharmaceuticals' cystic fibrosis business grew more than 40% in 2018, partially because of reimbursement victories in countries like Australia, Denmark and Sweden. One country has been particularly difficult to lock down, however, and Vertex doesn't see that changing in the near-term.
- In the U.K., the National Institute for Health and Care Excellence (NICE) has sparred with Vertex over the cost of its two-drug regimens. Now, Vertex is concerned that if it gains approval for one of its investigational triplets, the therapy will also face challenges getting U.K. reimbursement despite having strong clinical data.
- "Even those results, when put into the NICE model, don’t come out with a valuation which we think in any way gets close to valuing the transformative nature of these medicines," said Stuart Arbuckle, Vertex's chief commercial officer, on a fourth quarter earnings call Tuesday. "So even for the triple, we are going to continue to need to work with England to change the assessment methodology for all medicines."
Vertex's cystic fibrosis portfolio, consisting of Kalydeco (ivacaftor), Orkambi (ivacaftor/lumacaftor) and Symdeko (ivacaftor/tezacaftor), raked in almost $870 million in the fourth quarter, easily beating consensus estimates.
Symdeko, the newest of those drugs, first gained U.S. approval last February and has since enjoyed a strong launch. Executives noted on Tuesday's earnings call how Symdeko was the key driver of revenue growth both in the fourth quarter and across 2018. The therapy's net revenue respectively totaled $294 million and $769 million for those periods.
Across the pond, Symdeko has made waves of a different sort. It's been a source of contention between U.K. regulators and Vertex executives, two groups that haven't seen eye to eye on the value of the biotech's cystic fibrosis medicines.
Last year, NICE recommended the U.K.'s National Health Service not cover Orkambi on the grounds of cost-effectiveness. It also suspended a review of Symkevi (the branded name of Symdeko in Europe) after Vertex refused to provide evidence for a NICE appraisal.
"We support the need for robust and fair medicines appraisal in England, but believe that NICE's single technology appraisal has not kept pace with changes in medicine and has significant limitations in how it captures and values the full benefits of precision medicines such as tezacaftor/ivacaftor," a Vertex spokesperson wrote in an email to BioPharma Dive last August.
While the arguments go on, Vertex now worries the pushback will extend to its closely watched investigational triplets. The biotech has two different three-drug regimens in late-stage testing, and plans to file one for approval in the U.S. by mid-year.
Vertex estimates that an approved triplet, along with the rest of its portfolio, would allow around 90% of the world's cystic fibrosis population to be eligible for one of its products. Currently, the company says around 18,000 patients take one of its cystic fibrosis drugs.
Yet high price tags on those therapies have been a barrier to access for some patients, and a hard pill to swallow for countries like the U.K. and France.
"We expect the pressure to reimburse these medicines to increase with the approval of Symdeko/Symkevi in Europe, and then to [intensify] again once the triples are approved," SVB Leerink analyst Geoffrey Porges wrote in a Feb. 6 investor note.