Landmark Legal Battle for Cystic Fibrosis Medicine Ends in Price Reduction for South African Patients

Cheri Nel, an investment banker from Johannesburg, recently ended her high-profile legal challenge against Vertex Pharmaceuticals regarding the exorbitant cost and availability of Trikafta, a life-changing cystic fibrosis medication. Recognized as a 'miracle' treatment, Trikafta has the potential to vastly improve the lives of 90% of cystic fibrosis patients by managing symptoms and slowing the disease's progression.

Nel sought a compulsory license from the Gauteng Division of the High Court in Pretoria to allow for the importation of generic Trikafta—only to find that Vertex had not registered Trikafta with the South African Health Products Regulatory Authority, nor had it appointed a local distributor. Consequently, South African patients had to resort to traveling to Argentina to purchase a generic version named Trixacar.

With Trikafta's US price tag over $300,000 annually, fears grew that a similar cost would inhibit access for many South Africans. While the Argentinian generic version was cheaper at approximately $60,000, it was still unaffordable for most, and its import faced potential patent infringements.

The lawsuit by Nel aimed to facilitate the direct import of generic Trikafta to South Africa, mitigating the need for costly travel, and sparking significant international media attention. The pressure appeared to have a tangible effect. In recent developments, Vertex designated Equity Pharmaceuticals as its local importer through Section 21 authorizations, and while the price for a year's supply remains high at about R400,000, it is substantially lower than initially feared.

Additionally, Vertex has been in discussions with South African medical schemes, though the exact nature of these discussions remains opaque. Discovery Health highlighted that their comprehensive and executive plans offer a substantial cystic fibrosis medication benefit, aligning with Vertex's recent statements; over 100 South African patients have already been prescribed Trikafta.

Although Nel dropped the case, the impact is evident; the reduced cost of Trikafta in South Africa and the speed of patient uptake stand testament to her efforts. Moreover, generic Trikafta from Argentina has seen price increases due to hyperinflation, making Vertex's South Africa offering more competitive.

Nel and supporting advocacy groups now pivot their efforts towards encouraging government procurement of the drug for public health sector patients. Vertex, meanwhile, maintains that Section 21 authorization—bypassing complete registration—remains the quickest path to sustainable access.

In summary, while registration of Trikafta in South Africa remains unconfirmed, Vertex's strategic choices appear influenced by non-transparent pricing flexibility. Nevertheless, the case demonstrates the intricate play between legal action, advocacy, and pharmaceutical pricing, ultimately leading to potential gains in patient access and affordability.