Evaluation of orphan maintained biological medicinal products in the European Union between 2018 to 2023: a regulatory perspective.

IF 3.6 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Paolo Caferra, Thomas Fraisse, Maria Letizia Trincavelli, Laura Marchetti, Anna Maria Piras
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引用次数: 0

Abstract

Objectives: Orphan medicinal products (OMPs) authorized in the European Union (EU) benefit from market exclusivity, fee waivers, and national incentives. Maintaining orphan status during the marketing authorization application (MAA) requires meeting eligibility criteria, especially demonstrating significant benefit (SB), which is challenging. This study identifies key features linked to successful orphan status maintenance for biological OMPs approved in the EU between 2018 and 2023.

Methods: Data from European public assessment reports and orphan maintenance assessment reports were analyzed.

Results: Among the 50 biological OMPs granted orphan designation, 68.0% had to demonstrate SB over existing treatments, with 91.2% leveraging the clinically relevant advantage area, utilizing better clinical efficacy (83.8%) and subpopulation (38.7%) sub-domains. However, 32% did not need to demonstrate SB due to a lack of alternative treatments, most of which were ultra-orphan drugs. Advanced therapy medicinal products and monoclonal antibodies were the most numerous OMP categories, whereas oncology and immunomodulation were the preferred therapeutic areas.

Conclusion: The Orphan Regulation plays a critical role in advancing treatments for rare diseases, fostering innovation while addressing unmet medical needs. Nonetheless, the insufficient return on investment criterion remains underused, whereas refining major contribution to patient care guidelines and incorporating real-world evidence may enhance regulatory evaluations.

2018 年至 2023 年欧盟孤儿维护生物药品评估:监管视角。
目标:在欧盟(EU)获得授权的孤儿药(OMP)可享受市场独占、费用减免和国家激励措施。在申请上市许可(MAA)期间保持孤儿地位需要满足资格标准,尤其是证明显著获益(SB),这具有挑战性。本研究确定了 2018 年至 2023 年欧盟批准的生物 OMP 成功维持孤儿地位的关键特征:方法:分析欧洲公开评估报告和孤儿地位维持评估报告中的数据:在获得孤儿地位的50种生物OMP中,68.0%必须证明其优于现有疗法,其中91.2%利用了临床相关优势领域,利用了更好的临床疗效(83.8%)和亚人群(38.7%)子领域。然而,有 32% 的公司由于缺乏替代治疗方法而无需证明 SB,其中大部分是超非专利药物。先进治疗药物产品和单克隆抗体是数量最多的 OMP 类别,而肿瘤学和免疫调节则是首选治疗领域:孤儿条例》在推动罕见病治疗、促进创新、满足未得到满足的医疗需求方面发挥了关键作用。然而,投资回报不足的标准仍未得到充分利用,而完善对患者护理指南的主要贡献并纳入真实世界的证据可能会加强监管评估。
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来源期刊
Expert Opinion on Biological Therapy
Expert Opinion on Biological Therapy 医学-生物工程与应用微生物
CiteScore
8.60
自引率
0.00%
发文量
96
审稿时长
3-8 weeks
期刊介绍: Expert Opinion on Biological Therapy (1471-2598; 1744-7682) is a MEDLINE-indexed, international journal publishing peer-reviewed research across all aspects of biological therapy. Each article is structured to incorporate the author’s own expert opinion on the impact of the topic on research and clinical practice and the scope for future development. The audience consists of scientists and managers in the healthcare and biopharmaceutical industries and others closely involved in the development and application of biological therapies for the treatment of human disease. The journal welcomes: Reviews covering therapeutic antibodies and vaccines, peptides and proteins, gene therapies and gene transfer technologies, cell-based therapies and regenerative medicine Drug evaluations reviewing the clinical data on a particular biological agent Original research papers reporting the results of clinical investigations on biological agents and biotherapeutic-based studies with a strong link to clinical practice Comprehensive coverage in each review is complemented by the unique Expert Collection format and includes the following sections: Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results; Article Highlights – an executive summary of the author’s most critical points.
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