Findings come as drug manufacturer Vertex reports annual profits of $2.72 billion

New research presented at the 2021 North American Cystic Fibrosis Conference shows that an estimated 157,970 patients are living with cystic fibrosis (CF) worldwide, of whom 67% have been diagnosed and only 12% receiving the best available treatment by the end of 2020.

A new class of ground-breaking treatments known as CFTR modulators were first synthesised in 2013 and hailed as “transformative therapies”. Now, such drugs represent an opportunity to increase quality and length of life for 90% of CF patients. However, prices have been set between $270,000-310,000 per year for a condition requiring a lifetime of treatment.

106,000 patients from 94 different countries were estimated to have been diagnosed with CF, conversely, at the end of 2020 the best available treatment, a triple combination drug known as Trikafta, was only available in six countries.

Within the same timeframe, despite the limited rollout the manufacturer, Vertex Pharmaceuticals, reported record-breaking annual profits of $2.72 billion and a 55% year-on-year increase in product revenues.

The high treatment costs pose dilemmas to even the most robust of health systems, sparking controversy and lengthy negotiations between health systems and Vertex in Australia, the UK and Canada at the cost of patients.

The analysis revealed a much higher patient population than previously thought, particularly in low-income countries, where the manufacturer has largely not sought approval for any CFTR modulator therapies. At current list prices the cost of treating the entire diagnosed population would amount to over $32 billion per year.

Equivalent analyses for HIV/AIDS show much higher rates of both diagnosis and treatment. Similarly prohibitive drug prices for HIV treatment were decreased by over 99%, allowing millions access to lifesaving treatments in low- and high-income countries alike. With another triple combination treatment expected to advance to phase three trials this year, the authors call for urgent action to bring prices down and replicate the success seen with HIV.

This is particularly crucial now as Vertex has recently stated that they will no longer take applications for ‘compassionate use’ Trikafta across Europe and most of the world where there is currently no access, removing the only lifeline for hundreds of desperately ill and dying CF patients.