Wens Io, Claessens Zilke, Vanneste Alice, Barbier Liese, Janssens Rosanne, Huys Isabelle
Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
Front Med (Lausanne). 2025 Jan 7;11:1506243. doi: 10.3389/fmed.2024.1506243. eCollection 2024.
The 2020 pharmaceutical strategy for Europe stressed that rethinking regulatory policies to foster innovation in disease areas with unmet medical needs (UMN) is one of the European Commission's (EC) priority areas. To understand stakeholders' views regarding appropriate UMN criteria and incentives, the EC developed a survey and launched it for public consultation between September and December 2021. This study aims to assess stakeholders' views on the policy revisions proposed by the EC, particularly those regarding the definition of UMN, its criteria and incentives and evaluate how stakeholders' views are reflected in the proposed reform of the EU pharmaceutical legislation of 2023.
The public consultation survey comprised 14 questions including multiple-choice and open answer questions about the reform of the pharmaceutical legislation. A mixed-method analysis was conducted on publicly available data of stakeholders' responses, including descriptive and quantitative statistics for multiple-choice questions and a qualitative thematic framework analysis for open answer questions. A subgroup analysis was performed to assess differences and similarities in stakeholders' views, and results were compared with the proposed reform of the EU pharmaceutical legislation.
A total of 478 participants completed the survey consisting of 36% industry, 19% end-users, 17% healthcare providers, 7.5% researchers and 7.5% public bodies. All stakeholder groups favored including "absence of satisfactory authorized treatment" and "disease seriousness" as defining criteria for UMN. However, stakeholders disagreed on including the criterion "lack of access for patients," with public bodies and industry being less in favour. Industry favored maintaining or having additional incentives like transferable exclusivity vouchers on top of current intellectual property rights to foster innovation. In contrast, other stakeholders supported alternative proposals, namely enhancing the use of scientific advice and implementing expediting measures for regulatory evaluation of medicines targeting UMN.
Stakeholders agreed on including availability of alternatives and disease seriousness in the UMN definition but highlighted its ambiguity. Industry participants supported additional incentives like transferable exclusivity vouchers, whereas others preferred scientific and regulatory support. These findings underscore the need for further discussion on UMN criteria and incentives to stimulate innovation while ensuring patient-centric outcomes and equitable access to medicines across Europe.
2020年欧洲制药战略强调，重新思考监管政策以促进在未满足医疗需求（UMN）疾病领域的创新是欧盟委员会（EC）的优先领域之一。为了解利益相关者对适当的UMN标准和激励措施的看法，欧盟委员会开展了一项调查，并于2021年9月至12月期间进行公开征求意见。本研究旨在评估利益相关者对欧盟委员会提议的政策修订的看法，特别是关于UMN的定义、其标准和激励措施，并评估利益相关者的观点如何在2023年欧盟制药立法的提议改革中得到体现。
公开征求意见调查包括14个问题，涵盖关于制药立法改革的多项选择题和开放式回答问题。对利益相关者回复的公开可用数据进行了混合方法分析，包括对多项选择题的描述性和定量统计以及对开放式回答问题的定性主题框架分析。进行了亚组分析以评估利益相关者观点的差异和相似性，并将结果与欧盟制药立法的提议改革进行比较。
共有478名参与者完成了调查，其中行业占36%，终端用户占19%，医疗保健提供者占17%，研究人员占7.5%，公共机构占7.5%。所有利益相关者群体都赞成将“缺乏令人满意的获批治疗方法”和“疾病严重性”作为UMN的定义标准。然而，利益相关者在是否纳入“患者难以获得治疗”这一标准上存在分歧，公共机构和行业不太支持。行业倾向于维持或增加激励措施，如在现有知识产权之外提供可转让的排他性凭证以促进创新。相比之下，其他利益相关者支持替代提议，即加强科学建议的使用，并对针对UMN的药物监管评估实施加速措施。
利益相关者同意在UMN定义中纳入替代方案的可用性和疾病严重性，但强调其模糊性。行业参与者支持可转让的排他性凭证等额外激励措施，而其他利益相关者则倾向于科学和监管支持。这些发现强调需要就UMN标准和激励措施进行进一步讨论，以刺激创新，同时确保以患者为中心的结果以及欧洲各地公平获得药品。
