A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies.

Any method of determining 'value' should provide a fair incentive and outcome for industry, payers, regulators, and especially patients. Questioning of high prices occurs when patients, payers, and policy makers hold divergent views on the value and uncertainty of therapies.

Key for patients is that high prices need to be justified, and companies need to partner authentically with patients.

This 2019 publication via @nature provides a comprehensive look at a patient's view on fair pricing and equitable access. The article focuses on emerging gene therapies - a scientific technique that uses genes to treat or prevent disease. This allows doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

The current contender for the world's most expensive drug is gene-therapy Zolgensma, developed by Novartis in Switzerland. It’s used to treat spinal muscular atrophy (SMA), and costs over $2 million dollars per treatment.

The authors conclude drug manufacturers should not automatically assume that the combination of a 'gene therapy' label and a small patient population justifies high prices.