Rajueni Khujith, Chakraborty Choudhury Mohua
Department of Clinical Pharmacy, Poona College of Pharmacy, Bharati Vidyapeeth University, Pune, Maharashtra, India.
Indian Institute of Science, DST Center for Policy Research, Bengaluru, India.
PLOS Glob Public Health. 2023 Sep 27;3(9):e0001498. doi: 10.1371/journal.pgph.0001498. eCollection 2023.
India has a massive burden of rare diseases (RDs), with an estimated 96 million people living with RDs but limited options for treatment. Repurposing drugs used for other common conditions are essential alternative for RDs due to their cost-effectiveness and reduced timeline. India's patent regime prevents the evergreening of drugs, and a large generic manufacturing industry provides ample opportunity to explore the potential of repurposed drugs for treating RDs, known as repurposed orphan drugs (ROD). However, there is no portal or other source for information on orphan drugs in India. This study assesses the availability of RODs in India through quantitative analysis. In the absence of a separate orphan drug designation in India, we consider USFDA-approved orphan-designated products as the reference. We searched USFDA-approved RODs in recognized sources in India, such as CDSCO, AYUSH gazette, FSSAI, and Indian Pharmacopeia, which provides a list of drugs approved for marketing in India. We classified the drugs into separate groups based on their record from different sources and explored the regulatory implications of the differential representations. We found that almost 76% of the USFDA-approved RODs are listed in one of the Indian regulatory bodies' records either in the same form (61%) or in a different fixed-dose combination. For 67 drugs no information was found, these drugs have to be imported for use. Only 17 of the 43 RDs mentioned in the National Policy for Rare Diseases, matched the indications listed for approval of one of the 279 RODs identified in the study. This underscores the scarcity of RD treatment and the pressing need for accessibility in India. This information on RODs will help relevant stakeholders to efficiently manage RDs in India. The study also highlights existing gaps in Indian regulatory databases that limits access to accurate information about the availability of drugs.
印度罕见病负担沉重，估计有9600万人患有罕见病，但治疗选择有限。由于成本效益高且研发周期短，将用于其他常见病症的药物重新用于罕见病治疗是至关重要的替代方案。印度的专利制度防止药物专利延期，庞大的仿制药制造业为探索重新利用药物治疗罕见病（即重新利用的孤儿药）的潜力提供了充足机会。然而，印度没有关于孤儿药的信息门户或其他信息来源。本研究通过定量分析评估印度重新利用的孤儿药的可及性。由于印度没有单独的孤儿药指定，我们将美国食品药品监督管理局（USFDA）批准的孤儿指定产品作为参考。我们在印度的认可来源（如中央药品标准控制组织（CDSCO）、阿育吠陀公报、食品安全与标准管理局（FSSAI）和印度药典）中搜索USFDA批准的重新利用的孤儿药，这些来源提供了在印度批准上市的药物清单。我们根据药物在不同来源的记录将其分为不同组，并探讨了不同呈现方式的监管影响。我们发现，几乎76%的USFDA批准的重新利用的孤儿药以相同形式（61%）或不同的固定剂量组合列在印度监管机构的记录中。有67种药物未找到相关信息，这些药物必须进口使用。《国家罕见病政策》中提到的43种罕见病中，只有17种与本研究确定的279种重新利用的孤儿药之一批准的适应症相匹配。这凸显了印度罕见病治疗的稀缺性以及获取治疗手段的迫切需求。这些关于重新利用孤儿药的信息将有助于相关利益攸关方在印度有效管理罕见病。该研究还强调了印度监管数据库中存在的现有差距，这些差距限制了获取有关药物可及性的准确信息。
