A patient-driven campaign has been mounted across multiple countries, including India, for access to cystic fibrosis (CF) therapy - Trikafta, from the American biotech company Vertex Pharmaceuticals.

The international “Right to breathe” campaign seeks to “challenge Vertex’s monopoly, utilising a variety of legal and campaigning tactics to build pressure on corporations and politicians to deliver affordable access to CFTRs (a new class of medicines) for all cystic fibrosis patient,” a note from the campaigners said.

Campaigns have been mounted in South Africa, Ukraine, Brazil and India, led by ‘Vertex save us’, a global community of CF patients and families, and coordinated by Just Treatment, a UK-based campaigner for patient rights, with support from other international organisations.

Although Vertex does not have a presence in India, patient families recently wrote to the Centre, seeking access to the combination drug Trikafta or Kaftrio, that treats this life-shortening genetic disorder. CF causes sticky mucus to build up in the lungs, and digestive system, and the undiagnosed often die in their infancy, the letter said, urging the Government to consider patent provisions available in the law to make the drug accessible.

Responding to businessline on whether Vertex was exploring initiatives to make their drug accessible in India, a company spokesperson said, “In some low-to-middle-income countries such as India where access challenges are significant and where we do not currently have a presence, we are actively evaluating options that may facilitate access to our medicines including a product donation program, which we began piloting last year.”

In India, the patient families estimate the number of identified people living with CF to be between 600-1200. Due to the non-availability of diagnostics and under-reporting, the total number could be 40,000, their letter said. The only option for families to obtain these medicines was through a personal use license from the drug regulatory authority (CDSCO), the letter said.

“Most importantly, these medicines are highly priced and beyond the reach of patients and their families. Two of these drugs, Orkambi and Trikafta, are priced at $272,623 and $326,239 respectively,” it added.

Legal options

According to the campaigners, papers had been filed with the South African High Court seeking a compulsory licence that would allow - if granted - the supply of more affordable generic versions of the CF drug. Petitions to revoke or suspend Vertex’s patents were also submitted to governments in India, Ukraine, and Brazil, they added.

The letter from patient families in India pointed out that Orkambi and Trikafta are fixed-dose combinations (*see box). And while these therapies were not patented, individual components of these medicines were under patent protection in India, the letter said, preventing generic manufacturers from making it.

The CF patients and families coalition sought the revocation of Vertex’s patents under Sections 66 and 100 of the 1970 Patent Act. “The revocation of the patent is justified on at least two counts, including Vertex’s failure to work their patents (i.e. supply the relevant medicine) in the Indian market, and their unaffordable high prices,” a campaign document explained.

Cystic Fibrosis Therapy 
Orkambi is a combination of lumacaftor/ivacaftor
Trikafta is elexacaftor/tezacaftor/ivacaftor.