Obstacles to patients gaining access to advanced therapies

The Innovative Medicines Initiative (IMI) is the world’s biggest public-private partnership in the life sciences, part funded by the EU’s Horizon 2020 programme and part by the European pharmaceutical industry (through EFPIA). Genetic Alliance UK has previously been involved in a number of IMI-supported projects, most recently EUPATI (European Patients Academy on Therapeutic Innovation).

IMI recently published a concept paper looking at the key obstacles to European patients gaining access to effective advanced therapies. Advanced Therapy Medicinal Products (ATMPs) such as stem cell therapies, tissue therapies, gene therapies and combined therapies are regarded as some of the most promising types of innovative therapy, from which much is expected by patients with genetic conditions. Unfortunately, despite the hopes of patients, following the ATMP regulation (1394/2007), equitable and timely access to ATMPs has not been forthcoming.

IMI identified a number of issues in all stages of the development pathway: preclinical and clinical development, manufacturing, pricing, development and access. We responded to the consultation IMI concept paper, drawing on our ongoing work in this area, and in particular discussions at a multi-stakeholder workshop we held in November 2015 on how to improve patient access to advanced therapies.

We suggest that the most important challenges to the translation of advanced therapies, and those that are best suited to the public-private partnership model, are:

  • development of platform technologies such as a generic virus/vector system and universal allogeneic cell therapies
  • provision of a precompetitive platform encouraging early interaction between researchers and regulators
  • assessment of the value of clinical data gained though the hospital exemption/”specials” scheme and its use for regulatory purposes
  • development of innovative reimbursement mechanisms suitable to ATMPs

IMI will continue exploring this topic with stakeholders, and their conclusions will inform the development of future projects from 2017 onwards. We will be engaging with these discussions, and the field of advanced therapies more generally, to ensure that the voice of patients is clearly heard in these discussions.

Full response