Access to Innovative Neurological Drugs in Europe: Alignment of Health Technology Assessments Among Three European Countries

Abstract

Even for products centrally approved, each European country is responsible for national market access after European Medicines Agency (EMA) approval. This step can result in inequalities in terms of access, due to different opinions about the therapeutic value assessed by Health Technology Assessment (HTA) bodies. This study aims to provide a comparative analysis of HTA recommendations issued by EU countries (France, Germany, and Italy) for new neurological drugs following EMA approval. In the reference period, we identified 11 innovative medicines authorized in Europe for five neurological diseases (cerebral adrenoleukodystrophy, spinal muscular atrophy, metachromatic leukodystrophy, migraine, and polyneuropathy in patients with hereditary transthyretin amyloidosis), including eight drugs for genetic rare diseases. We found no agreement on the therapeutic value (in particular the “added value” compared to the standard of care) of the selected drugs. Despite the differences in terms of assessment, the access has been usually guaranteed even if with various types of limitations. The heterogeneity of the HTA assessment of clinical data among countries is probably related to the uncertainties about clinical value at the time of EMA approval and the lack of long-term data and of direct comparison with available alternatives. Given the importance of new medicines especially for rare diseases, it is crucial to understand and act on the causes of inconsistency among the HTA assessments, in order to ensure rapid and uniform access to innovation for patients who can benefit.

Editorial: Innovative treatments for neuro-psychiatric diseases

“…Finally, Gozzo et al analyzed the assessment of therapeutic value issued by European countries for innovative drugs approved for multiple sclerosis (MS). In Europe, all new drugs to treat neurodegenerative diseases as well as autoimmune and other immune dysfunctions must be authorized by the European Medicines Agency (EMA) via the Centralized Procedure (EMA, 2020 ; Gozzo et al, 2021a , b ). Then, each Country is responsible for pricing and reimbursement decisions made according to the assessments performed by their own health technology assessment (HTA) bodies (van Nooten et al, 2012 ).…”

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Editorial on the Research Topic Innovative treatments for neuro-psychiatric diseasesNeuropharmacology represents one of the therapeutic areas with the greatest number of molecules in development, reflecting research advances in the understanding of the basis of the diseases useful for promoting potentially innovative interventions (Pankevich et al, 2014;Gozzo et al, 2021a). However, neuropsychiatric conditions historically have been among the most difficult for which developing effective and safe new therapies, due to the complexity of pathophysiology and clinical presentation; therefore, curative treatments for important diseases, such as neurodegenerative diseases, are still lacking (O'Donnell et al, 2019;EC, 2020).

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