Thomas Bols, Head of Government Affairs and Patient Advocacy, EMEA and APAC at PTC, recently shared his perspective with Open Access Government on how preparing Europe for a new generation of innovative therapies has the potential to transform the lives of patients.
Gene therapies are bringing hope to patients living with ultra-rare diseases. In the article Thomas explores solutions in three key areas to ensure patients can benefit from novel treatments like gene therapies and to ensure an environment where R&D continues to evolve and flourish. Ultimately, to achieve the below outlined opportunities, Thomas notes, all stakeholders including biopharmaceutical companies, regulators, payers, HTA bodies and policymakers need to work together. Read below a brief synopsis:
Speed up the diagnosis of rare diseases through innovative therapies
The inclusion of more diseases in newborn screening panels is a potential solution and an important component of any national rare disease action plan, as disease outcomes are often better if patients are treated early.
Recalibrate market access processes
Regulatory and HTA frameworks were set up for the well-known therapy model, where treatment is given and paid for across a lifetime and often, to large numbers of patients. Gene therapies don’t fit this model. An evaluation must reflect these small patient numbers to encourage innovation.
Harmonize and simplify the collection of real-world evidence
The collaboration of biopharmaceutical companies, regulators, payers, HTA bodies and policymakers is important to design clear and uniform guidance so that real-world data is inter-changeable, interoperable and consistently accepted across Europe.
Working together with biopharmaceutical companies, regulators, payers, HTA bodies and policymakers, we have an opportunity to forge new ground so patients, caregivers and health systems can realize the full benefit of medical innovation. We are at the frontier of an exciting therapy pipeline.