The challenges of access to innovative medicines with limited evidence in the European Union

Frontiers in Pharmacology

Pharmacology

Antònia Agustí¹  Caridad Pontes²  Antonio Vallano³ 

[1]Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, Barcelona, Spain

[2]Clinical Pharmacology Service, Vall d’Hebron University Hospital, Barcelona, Spain

[3]Medicines Department, Catalan Healthcare Service, Barcelona, Spain

[4]Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, Barcelona, Spain

[5]Digitalization for the Sustainability of the Healthcare System DS3-IDIBEL, L’Hospitalet de Llobregat, Spain

[6]Medicines Department, Catalan Healthcare Service, Barcelona, Spain

[7]Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, Barcelona, Spain

[8]Healthcare Management of Hospitals, Catalan Institute of Health, Barcelona, Spain

关键词: drug approval;    drug costs;    orphan drug;    antineoplastic agents;    European Union;   

DOI  :  10.3389/fphar.2023.1215431

 received in 2023-05-01, accepted in 2023-08-21,  发布年份 2023

来源: Frontiers

【 摘 要 】

The European Medicines Agency (EMA) fosters access to innovative medicines through accelerated procedures and flexibility in the authorization requirements for diseases with unmet medical needs, such as many rare diseases as well as oncological diseases. However, the resulting increase of medicines being marketed with conditional authorizations and in exceptional circumstances has lead to higher clinical uncertainty about their efficacy and safety than when the standard authorizations are applied. This uncertainty has significant implications for clinical practice and the negotiation of pricing and reimbursement, particularly as high prices are based on assumptions of high value, supported by regulatory prioritization. The burden of clinical development is often shifted towards public healthcare systems, resulting in increased spending budgets and opportunity costs. Effective management of uncertainty, through appropriate testing and evaluation, and fair reflection of costs and risks in prices, is crucial. However, it is important not to sacrifice essential elements of evidence-based healthcare for the sake of access to new treatments. Balancing sensitive and rational access to new treatments, ensuring their safety, efficacy, and affordability to healthcare systems requires thoughtful decision-making. Ultimately, a responsible approach to timely access to innovative medicines that balances the needs of patients with healthcare systems’ concerns is necessary. This approach emphasizes the importance of evidence-based decision-making and fair pricing and reimbursement.

【 授权许可】

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Copyright © 2023 Vallano, Pontes and Agustí.