How much should a life-saving drug cost?

My former chairman used to tell the story of when he was a resident on rounds in the 1950s, he would hear a pounding sound in some of the patient rooms. When he looked in to discover the source, it was a nurse pounding the back of a patient who was stretched across the bed with his/her head hanging over the edge. The nurse was trying to dislodge the mucus in the lungs of the patients, who had cystic fibrosis. While cystic fibrosis is a multisystem genetic disorder, the main life-threatening complication is pulmonary infections caused by a build-up of secretions in the lung.

Enter Vertex, a Boston-based pharma company with a three-drug cocktail called Trikafta to treat cystic fibrosis. Trikafta works for about 90% of cystic fibrosis patients. Its transformational value is being recognized by the awarding of the $3 million Breakthrough Prize in Life Sciences, shared by three Vertex scientists who will donate the prize money to charity. But while this revolutionary advance deserves accolades, access to Trikafta may be limited by cost. The list price for Trikafta—a drug that has to be taken for the patient’s entire life—is $300,000/year, although the drug costs Vertex ca. $6,000 per patient annually.

With transformational therapies emerging on a regular basis these days, the tension between drug costs and access is a large and growing problem worldwide. Pharma invests a huge amount of money in drug research, and many or most potential drugs fail somewhere in the pipeline. Naturally, they want to recoup their investments and plow at least some of their profits into future drug development. On the other side of the equation is the patient population. Most cystic fibrosis patients world-wide can’t afford the sticker price of this life-preserving medication.

While it has opposed the development of cheaper generics, Vertex claims it has made the drug available for free to 6,500 patients and has launched a program to donate Trikafta to patients in twelve low-income companies.

I don’t know enough about the business model of Vertex and other pharma companies to say where the line is for fair profits. Striking the balance between recovering cost and investment on the one hand and patient access on the other has been a challenge for a long time, and it’s only going to continue.

Vertex scientist win award for Trikafta