Costa Enrico, Grimaldi Gianluca, Del Grosso Vittorio, Isgrò Antonella, Genazzani Armando
Agenzia Italiana del Farmaco (AIFA), Roma.
Agenzia Italiana del Farmaco (AIFA), Roma - Dipartimento di scienze del farmaco, Università del Piemonte Orientale, Novara.
Recenti Prog Med. 2022 Jul-Aug;113(7):415-424. doi: 10.1701/3850.38335.
In the last decades, the development of drugs for rare diseases has been supported by regulatory and financial incentives. On the other hand, public health policies have increasingly taken into account the person affected by a rare disease in their strategies. In this perspective, we examined the relation between the regulatory framework on rare diseases and the regulatory framework on drug approval. Technical proposals have been brought forward to protect the needs of individuals.
The legislative framework on rare diseases has developed both at a European and national level with the aim to strengthen the network of centers for diagnosis and care by increasing the degree of social and health protection, as well as to accelerate the assessment, approval and access to new drugs. Since 2000, 210 orphan drugs have been approved by the European Medicines Agency (EMA) (compared to an estimated 7-8,000 rare diseases). Of the 118 orphan drugs active in the community register as of 2020, 97 (82.2%) were available in Italy: 17 (17.5%) in class A; 58 (59.8%) in class H; 12 (12.4%) in class C; 10 (10.3%) in class C-nn. In 2020, expenditure on drugs with an orphan indication accounted for 6% of the total pharmaceutical expenditure (+47% since 2016). These drugs have benefited from incentives at both European and national levels, as well as inclusion in national early access programs. However, the average duration of the assessment process is above the 100-day limit set by law.
The legislation on rare diseases has developed in different directions, and drug legislation has undoubtedly played a major role in terms of the results achieved. However, orphan drugs enter the market with a high price, which increasingly represents a problem of sustainability for health systems but notwithstanding Italy shows a high ratio between the level of social protection and access times. In this perspective, it is necessary to be provided with tools for a better system balance with a view to optimize these timeframes. Introducing in Italy a system for tracking the negotiation process that considers the clock stops as the EMA does, would allow to know at what point the negotiation process is. In addition, once the 100-day period is over (net of any clock stops) and in case of failure to reach a negotiation agreement, a second 60-day negotiation round could be proposed and so on. In this way, all the parts involved the system would have a clear scope of action to conclude the process in a flexible but certain timeframe. In this regard, the joint clinical assessments foreseen by the new HTA Regulations provide an additional opportunity to harmonize central decisions with national requirements.
在过去几十年中，罕见病药物的研发得到了监管和资金激励的支持。另一方面，公共卫生政策在其战略中越来越多地考虑到受罕见病影响的人。从这个角度来看，我们研究了罕见病监管框架与药物批准监管框架之间的关系。已经提出了技术建议以保护个人需求。
欧洲和国家层面都制定了罕见病立法框架，目的是通过提高社会和健康保护程度来加强诊断和护理中心网络，并加速新药的评估、批准和获取。自2000年以来，欧洲药品管理局（EMA）已批准210种孤儿药（相比之下估计有7000 - 8000种罕见病）。截至2020年在社区登记册中有效的118种孤儿药中，97种（82.2%）在意大利有供应：A类17种（17.5%）；H类58种（59.8%）；C类12种（12.4%）；C - nn类10种（10.3%）。2020年，有孤儿适应症药物的支出占药品总支出的6%（自2016年以来增长了47%）。这些药物在欧洲和国家层面都受益于激励措施，以及被纳入国家早期获取计划。然而，评估过程的平均持续时间超过了法律规定的100天期限。
罕见病立法朝着不同方向发展，药物立法在取得的成果方面无疑发挥了重要作用。然而，孤儿药以高价进入市场，这日益成为卫生系统可持续性的一个问题，但尽管如此，意大利在社会保护水平与获取时间之间显示出高比例。从这个角度来看，有必要提供工具以实现更好的系统平衡，从而优化这些时间框架。在意大利引入一个跟踪谈判过程的系统，该系统像EMA那样考虑时钟暂停，将能够知道谈判过程处于什么阶段。此外，一旦100天期限结束（扣除任何时钟暂停时间）且未达成谈判协议，可以提议进行第二轮60天的谈判，依此类推。这样，系统中所有相关方将有明确的行动范围，以便在灵活但确定的时间框架内完成该过程。在这方面，新的卫生技术评估法规所预见的联合临床评估提供了一个额外的机会，使中央决策与国家要求相协调。
