International Rare Disease Day|The success rate of clinical development of rare disease drugs in the past 10 years is 17%

February 28 is the 14th "International Rare Disease Day".

According to the latest report on the success rate of drug clinical development: From 2011 to 2020, the average success rate of rare disease (excluding tumor indications) drugs from phase 1 clinical trials to the approval of the US FDA for marketing is 17.

The conversion success rate of rare disease drugs in the four development stages is higher than the industry average

According to the report, the researchers analyzed the success rate of 12,728 transitional projects (involving 1779 companies) that occurred in 9704 drug clinical development projects in the past ten years (2011-2020), of which 1256 were Rare disease drug development project.

The analysis found that the success rate of rare disease drugs from phase 1 clinical to phase 2 clinical is 67.

Picture source: reference [1]

It is worth mentioning that the success rates of rare disease drugs in these four stages are higher than the average development success rate of drugs in all disease fields in these four stages.

The success rate of rare disease drugs from phase 1 clinical to approval is 17.

In addition, researchers also evaluated the success rate (LOA) of rare disease drugs from phase 1 clinical trials to FDA approval.

According to the report, the average success rate of rare disease drugs (excluding tumor indications) from phase 1 clinical to FDA approval is 17.

At the same time, researchers also evaluated the success rate of drug development for tumor indications in rare diseases.

In 2020, the number of orphan drugs approved by the FDA accounted for a record high

It is worth mentioning that in the 2006-2015 drug clinical development success rate report previously released by researchers, there were only 521 drug development projects in the field of rare diseases.

This has also been verified in the new drugs approved by the US FDA.

▲The proportion of orphan drugs approved by the FDA in 2020 is the highest in the past 10 years (graphic by WuXi AppTec's content team)

I believe that with the efforts of all sectors of society, more and more rare disease drugs will be approved for the market, providing patients with new treatment options.

Note: This article aims to introduce the progress of medical and health research, not to recommend treatment options.

Reference materials:

[1]"Clinical Development Success Rates and Contributing Factors 2011–2020".