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    Home > Active Ingredient News > Drugs Articles > Global R & D pipeline accounts for more than 30% of rare diseases, and later R & D meets challenges

    Global R & D pipeline accounts for more than 30% of rare diseases, and later R & D meets challenges

    • Last Update: 2019-07-26
    • Source: Internet
    • Author: User
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    In recent years, research and development of drugs for rare diseases has become one of the most active and fastest-growing fields, accounting for more than 30% of global research and development pipelines The continuous investment in research and development of drugs for rare diseases has promoted the rapid growth of clinical trials in the later stage Recently, the tufts Center for the study of drug development (CSDD), a well-known University of tufts, collected 4562 FDA regulated clinical trial data from the website of clinical trials.gov The analysis of these data shows that there are challenges in the research and development of drugs for rare diseases in science and operation, and new clinical research and development strategies, operation specifications and solutions are needed Since 2010, the proportion of drugs for rare diseases in new drug approval has doubled In 2018, more than half of drugs and biological products approved in the world are for rare diseases Of the 59 new drugs approved by the FDA last year, 34 (58%) were approved for rare diseases, up from just a third a few years ago In 2018, there were 3500 small and large molecules targeting rare diseases in the R & D pipeline, accounting for more than 30%, more than double the 1530 in 2010 Significant growth in orphan drug eligibility and orphan drug approval in the past 25 years, FDA granted orphan drug status has increased significantly During 1994-98, only 301 drugs obtained orphan drug qualification The number soared to 1800 in the same period of 2014-18, nearly six times that of 1994-98! At the same time, we also saw a significant increase in the total number of approved orphan drugs - from 66 in 2003-08 to 316 in 2014-18 Since 2000, the proportion of approved orphan drugs in all qualifications has remained relatively stable, at present 18%, slightly higher than 16% since the enactment of the orphan drug act of 1983 (ODA) During 2004-2018, the continuous investment in research and development of drugs for rare diseases achieved results Initially, there were about 4100 clinical trials for treatment of rare diseases worldwide In the initial new clinical trials for rare diseases, the vast majority (84%) are currently in phase 1 and phase 2 clinical development The growth rate of phase 2 and phase 3 clinical trials for rare diseases is twice that of phase 1 clinical trials From the clinical stage to the approval stage, the clinical trials of rare disease drugs took 131 months on average, 68% longer than that of non rare disease drugs and 41% longer than that of other cancer-related diseases Compared with the evaluation time of non rare disease drugs, the overall evaluation time of rare disease drugs is 4 months faster On the whole, the development time of drugs for rare diseases is 4 years longer than that of all other drugs The scale and scope of clinical trials vary significantly Clinical trials of drugs for rare diseases have the characteristics of small sample size, large heterogeneity and difficult recruitment, which makes the determination and enrollment of patients face unprecedented challenges at any stage of clinical trials In terms of numbers, compared with non rare disease drugs, the number of research sites for phase I clinical trials of rare disease drugs is 6 times that of non rare disease clinical trials, and the number of patients recruited is one quarter of the latter Phase 2 and phase 3 clinical trials of rare diseases accounted for half to 60% of the total average number of research sites, and the number of enrolled patients was as low as 15% of the total average number of clinical trial volunteers Compared with the recruitment of non rare disease patients, the failure rate of screening and random enrollment of rare disease patients in clinical trials is significantly higher, which shows the challenge of recruitment and maintenance of rare disease patients Among the patients screened in the clinical trials of rare diseases, 81% of them were not qualified for the group, 56% of them could not be randomly enrolled in the group The failure rate of screening in the clinical trials of non rare diseases was 57%, and the failure rate of random enrollment was 36% Compared with non rare diseases, patients with rare diseases participating in randomized clinical trials had a lower rate of out of group According to the summary of the failure rate of clinical trial patient screening and random enrollment in 2014-2018 (picture source: tufts CSDD), the research and development heat of drugs for rare diseases is not reduced, which is expected to change the dilemma that many patients with rare diseases have little or no drugs to choose However, we also need to note that from the clinical to the approval, the use of drugs for rare diseases takes an average of 4 years longer than that for non rare diseases Moreover, the clinical trials of rare disease drugs generally involve more research sites, fewer patients are recruited, and it is difficult for patients to confirm and enter the group In order to benefit the patients with rare diseases all over the world, how to deal with these challenges is worth thinking and solving jointly by the industry and regulators Reference [1] growth in rare release R & D in chaining development strategy and execution Vol 21 No 4 Retrieved Jul 18, 2019 from https://static1.squarespace.com/static/5a9eb0c8e2ccd1158288d8dc/t/5d2490ae0072ee0001a198 / 15626774223360 / summary julyaugust_.pdf
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