What has changed in the treatment of rare diseases in a decade?

More than 5000 orphan drug qualificationsonce, rare diseases are synonymous with "no cure." In the 1983 Orphan Drug Act, only 10 rare disease drugs were on the market in a full 10 yearsBetween January 1, 1983 and October 31, 2019, its official database showed that the FDA issued 5,219 "orphan drug eligibility" (note: the same drug/therapy for different indications, can obtain multiple orphan drug eligibility), the final number of drugs approved 843, can be said to be a world wide differenceit's worth noting that the industry's enthusiasm for rare diseases is holding high amid the research and development boom in orphan drugsOver the past 10 years, the number of "orphan drug eligibility" issued by the FDA has increased year by 7% year-on-year, reaching an all-time high of 477 in 2017, a 2.5-fold increase over 2010, reflecting the increasing concentration of innovative drug development in the field of rare diseasesThe number of drugs approved for "orphan drug eligibility" has risen rapidly at a CAGR of 16%, with a total of 94 models approved for different indications in 2018 (Note: For different indications, a drug can be approved multiple times)In 2019, that number also reached 75nearly half of the orphan drug ratio
so far in 2010, the FDA has approved 380 new drugs to market, a 60 percent increase over the previous decade (new drugs listed here include CDER-approved NME, as well as CBER-approved gene therapy, cell therapy, and recombinant vaccines)Among them, the number of new drugs on the market for orphan drug qualification reached 165, accounting for 43% of the total number of new drugs approved by the FDA in the past decadein the past 10 years, the number of FDA new drug approvals at a compound annual growth rate of 7% year by year, at the same time, the number of new drugs eligible for orphan drug with a compound annual rate of 14% rapid increase, the annual proportion of orphan drugs is constantly rising, 2018 reached an all-time high, the proportion of orphan drugs reached 58%a major breakthrough in rare diseases
in the past 10 years, 54 new FDA drugs that have been eligible for both breakthrough therapies and orphan drugs, covering 42 diseases, of which we have found many breakthroughs in the treatment of rare diseases, such as 12 in China's First Rare Diseases Catalog Of the 11 rare diseases, 11 have been approved by the FDA, bringing the total to 15, and 70% of the time on the market is focused on 2017 and 2018, which has seen significant results in accelerating the policy of accelerating the listing of new drugs for orphans or rare diseases"China's first list of rare diseases", there are 11 rare diseases with FDA-approved new drugs (data source: public information, pharmaceutical Ming-Kang de's content team mapping)conclusionthe conclusion, in the past 10 years, the field of rare disease treatment has been a welcome developmentBoth the number of approved drugs and the enthusiasm of pharmaceutical companies for research and development remain high But we also need to realize that the number of rare diseases is huge, and there are a lot of gaps that need to be innovated and broken It has also left a huge opportunity for many traditional pharmaceutical companies that develop small-molecule drugs and biological products, or biotech start-ups specializing in gene therapy We look forward to the emergence of more rare disease treatment drugs in the future for the benefit of rare patients around the world Note: This paper aims to introduce the progress of medical health research, not the recommendation of treatment programs For treatment plan guidance, visit a regular hospital data sources: Instant Drug Counts, FDA Website References: , Glynn, Wei Cuijie, History, et al (2018), China Rare Disease Sage Research, Beijing Medicine, DOI:CNKI: SUN: BJYX.0.2018-05-016 Original Title: What have changed in the treatment of rare diseases in the past decade?