Access to gene therapy requires different approach
A large number of new gene therapies will become available in the coming years. These treatments are among the most expensive drugs and could place a large burden on the available healthcare budget. Price negotiations will be only marginally effective in reducing costs. A more differentiated approach is necessary to keep as many gene therapies available to patients as possible. In addition, the government may be faced with making painful choices between the interests of individual patients and collective health care.
Gene therapy offers opportunities for curing seriously ill patients who previously had no treatment options. But gene therapies are also among the most expensive drugs in the world. In the policy report ‘The Value of Gene Therapy’ presented to the Ministers of Health, Welfare and Sport and of Infrastructure and Water Management, COGEM explores, among other things, how many gene therapies (including cell therapies) are in the development pipeline, the costs, and the dilemmas and choices involved. It also discusses the policy instruments the government has to keep gene therapies affordable and available.
At present, 15 gene therapies are authorized on the European market. The number of gene therapies is expected to increase to 100 by 2032. There is no indication that the prices of these treatments will decrease significantly. The growth in the number of gene therapies will place a large burden on the healthcare budget.
COGEM signals that the current approach to price negotiations to reduce drug costs has limited effectiveness. Although the asking prices of some gene therapies include considerable room for manoeuvre, this is not true for all gene therapies. Gene therapies have already been withdrawn from the European market because the manufacturer found reimbursements in EU member states too low. Also, some gene therapies target such small groups of patients that they are not commercially interesting. Moreover, the debate focuses on very expensive gene therapies for very small numbers of patients, while the greatest claim on the healthcare budget will be made by the less expensive and eye-catching gene therapies that are intended for larger numbers of patients, such as CAR-T cell therapies against various cancers.
There is a risk that life-saving therapies may be developed that will not be available to patients due to high prices. The expected increase in the number of gene therapies and the differences between therapies make it necessary to take a more differentiated approach by the government, ranging from price negotiations to market intervention and even public-sector development of treatments. Increasing the budget for expensive medications is also an option. If the budget remains the same, stricter selection of reimbursable gene therapies is inevitable, resulting in some treatments not being available to patients.
Download the policy report for full text: The Value of Gene Therapy. A study of the growth, cost and accessibility of gene therapy products