Gene therapy ante portas

Gene therapies are widely regarded as promising but not yet fully developed therapeutic strategies for the treatment of very rare and otherwise non-treatable diseases. The high expectations were dashed in the early phases of research and development in the 1970s and not even the decoding of the human genome at the end of the nineties could inaugurate the beginning of a new age of gene therapies.

After the first gene therapy, Glybera®, was approved in Europe, it was withdrawn from the European market due to disappointing results, letting many protagonists of the health care system consider the gene therapeutic approach to be questionable and having limited potential. However, current and very impressive study data suggests that several highly potential gene therapies will access the German market in the upcoming years.

Since the paradigm of gene therapy follows a completely different logic than conventional therapies and because gene therapy costs a multiple of common innovative pharmaceuticals, payers are going to face a considerable challenge that cannot be handled with traditional instruments. Here, the authors explain challenges regarding the reimbursement of gene therapies and discuss possible solutions for the health insurance system.