An article published in Clinical Pharmacology & Therapeutics by the European Medicines Agency (EMA), describes the interactions needed between stakeholders to accelerate patient access to ground-breaking new therapies.
The perspective piece uses the example of Zyntelgo, a recently approved gene therapy for patients with a rare inherited blood condition that causes severe anaemia. This medicine’s swift authorisation was aided by the EMA’s Priority Medicines Scheme (PRIME), which allowed early and enhanced dialogue between developers, the EMA’s scientific advice office and patients’ representatives. These efficient interactions supported a stronger application which demonstrated the benefits and risks, allowing accelerated assessment.
These therapies have presented many regulatory challenges for those assessing benefits and risks for initial authorisation, and those assessing value for pricing and reimbursement decisions.
“Continuous dialogue throughout the development of the medicine, without compromising the impartiality of the assessment at the marketing-authorisation application stage, can help overcome these challenges.”
The authors then go on to discuss some of the specific clinical and manufacturing process problems which were identified and how these were resolved. They also say that accelerated approvals are only possible if a “robust post-approval plan” is illustrated at the marketing authorisation stage.
Early dialogue and frequent interactions between medicine developers, regulators, health technology assessment bodies (HTAs) and patients’ representatives are all key players in effectively delivering data needed to enable accelerated access for patients, to highly innovative therapies that address unmet medical needs.
This article is accessible here.