'Preventative' Gene Therapies: Perspectives On Payer Access Challenges And Solutions

Patients with severe genetic diseases for which few disease-modifying therapies are available are experiencing transformational, long-term benefits from gene therapies. According to reports, while gene therapies have been useful in stopping the progression of diseases, they are much less effective in reversing damage already caused. Thus, there has been a push to move the use of gene therapies upstream, namely screening and then treating patients earlier in the disease pathway before they reach a symptomatic stage.

Although this “preventative” approach has the potential to bring benefits for patients and society, it is also associated with an increased time lag between treating patients in the pre-symptomatic stage and seeing either improvement in patient outcomes or reductions in healthcare-related costs.

However, the challenges of evaluating the long-term benefits of these therapies from a payer perspective must also be considered. This white paper by PRECISIONadvisors highlights the shift of gene therapy research into more prevalent diseases and outlines how manufacturers and payers can co-create evidence requirements and models to optimize launch success and sustainability.