Department of Science, Technology and Society, 307 Bethune College, York University, 4700 Keele Street, Toronto, ON, M3J 1P3, Canada.
University of São Paulo Public Health School, Health Law Research Center of the University of São Paulo, Av. Dr. Arnaldo, 715, São Paulo, Brazil.
Orphanet J Rare Dis. 2022 Sep 5;17(1):344. doi: 10.1186/s13023-022-02476-6.
Rare diseases are associated with difficulties in addressing unmet medical needs, lack of access to treatment, high prices, evidentiary mismatch, equity, etc. While challenges facing the development of drugs for rare diseases are experienced differently globally (i.e., higher vs. lower and middle income countries), many are also expressed transnationally, which suggests systemic issues. Pharmaceutical innovation is highly regulated and institutionalized, leading to firmly established innovation pathways. While deviating from these innovation pathways is difficult, we take the position that doing so is of critical importance. The reason is that the current model of pharmaceutical innovation alone will not deliver the quantity of products needed to address the unmet needs faced by rare disease patients, nor at a price point that is sustainable for healthcare systems. In light of the problems in rare diseases, we hold that re-thinking innovation is crucial and more room should be provided for alternative innovation pathways. We already observe a significant number and variety of new types of initiatives in the rare diseases field that propose or use alternative pharmaceutical innovation pathways which have in common that they involve a diverse set of societal stakeholders, explicitly address a higher societal goal, or both. Our position is that principles of social innovation can be drawn on in the framing and articulation of such alternative pathways, which we term here social pharmaceutical innovation (SPIN), and that it should be given more room for development. As an interdisciplinary research team in the social sciences, public health and law, the cases of SPIN we investigate are spread transnationally, and include higher income as well as middle income countries. We do this to develop a better understanding of the social pharmaceutical innovation field's breadth and to advance changes ranging from the bedside to system levels. We seek collaborations with those working in such projects (e.g., patients and patient organisations, researchers in rare diseases, industry, and policy makers). We aim to add comparative and evaluative value to social pharmaceutical innovation, and we seek to ignite further interest in these initiatives, thereby actively contributing to them as a part of our work.
罕见病的治疗存在医疗需求未得到满足、药物可及性差、药价高、证据不匹配、公平性等问题。虽然全球各国（高收入和中低收入国家）在面临罕见病药物研发挑战方面存在差异，但也存在跨国表达的共性，这表明存在系统性问题。药物创新受到高度监管和制度化，导致创新途径非常固定。虽然偏离这些创新途径具有挑战性，但我们认为这一点至关重要。原因是，仅靠当前的药物创新模式无法提供满足罕见病患者未满足需求所需的产品数量，也无法以对医疗体系可持续的价格点提供这些产品。鉴于罕见病领域存在的问题，我们认为重新思考创新至关重要，应提供更多的空间来探索替代创新途径。我们已经在罕见病领域观察到大量新类型的倡议，这些倡议提出或采用替代药物创新途径，这些倡议的共同点是涉及多样化的社会利益相关者，明确解决更高的社会目标，或者两者兼而有之。我们的立场是，可以借鉴社会创新原则来构建和阐明这些替代途径，我们将其称为社会制药创新（SPIN），并应给予其更多的发展空间。作为一个跨学科的社会科学、公共卫生和法律研究团队，我们研究的 SPIN 案例分布在跨国范围内，包括高收入和中等收入国家。我们这样做是为了更好地了解社会制药创新领域的广度，并推动从床边到系统层面的变革。我们寻求与那些从事此类项目的人（例如患者和患者组织、罕见病研究人员、行业和政策制定者）合作。我们旨在为社会制药创新增加比较和评估价值，并寻求激发对这些倡议的进一步兴趣，从而积极参与其中，作为我们工作的一部分。
