Advanced therapy medicinal products (ATMPs) comprising cell therapy, gene therapy, and tissue-engineered products, offer a multitude of novel therapeutic approaches to a wide range of severe and debilitating diseases. To date, several advanced therapies have received marketing authorization for a variety of indications. However, some products showed disappointing market performance, leading to their withdrawal. The available evidence for quality, safety, and efficacy at product launch can play a crucial rule in their market success. To evaluate the sufficiency of evidence in submissions of advanced therapies for marketing authorization and to benchmark them against more established biological products, we conducted a matched comparison of the regulatory submissions between ATMPs and other biologicals. We applied a quantitative assessment of the regulatory objections and divergence from the expected data requirements as indicators of sufficiency of evidence and regulatory flexibilty, respectively. Our results demonstrated that product manufacturing was challenging regardless of the product type. Advanced therapies displayed critical deficiencies in the submitted clinical data. The submitted non-clinical data packages benefited the most from regulatory flexibility. Additionally, ATMP developers need to comply with more commitments in the post-approval phase, which might add pressure on market performance. Mitigating such observed deficiencies in future product development, may leverage their potential for market success.

包括细胞疗法，基因疗法和组织工程产品在内的先进疗法药物（ATMP），为多种严重和令人衰弱的疾病提供了多种新颖的治疗方法。迄今为止，几种先进疗法已经获得了针对各种适应症的上市许可。但是，某些产品的市场表现令人失望，导致其退出市场。产品上市时可获得的质量，安全性和有效性的证据可以在其市场成功中发挥至关重要的作用。为了评估提交高级药品用于上市授权的证据的充分性，并将其与更成熟的生物产品进行比较，我们对ATMP和其他生物制剂之间的法规提交进行了匹配比较。我们对监管异议进行了定量评估，并将其与预期数据要求的差异分别作为证据充足和监管灵活性的指标。我们的结果表明，无论产品类型如何，产品制造都具有挑战性。先进疗法在提交的临床数据中显示出严重的缺陷。提交的非临床数据包受益于法规灵活性。此外，ATMP开发人员需要在批准后阶段遵守更多承诺，这可能会给市场绩效带来压力。减轻这种在未来产品开发中发现的缺陷，可以利用其在市场上取得成功的潜力。