The FDA granted 5808 orphan drug approvals for 38 years after the implementation of the "Orphan Drug Act"

WenYu Jian

Huntington's disease, neurofibromatosis, Duchenne muscular dystrophy, osteogenesis imperfecta, Marfan's disease, Bruton's agammaglobulinemia, congenital adrenal hypoplasia, premature aging, inability to fall asleep, whitehead overnight, pica , Foreign accent syndrome.

According to the definition of WHO, diseases with the number of patients taking up 0.

The diagnostic, therapeutic and preventive drugs developed for rare diseases are collectively referred to as "Ophan Drugs".

The United States is the first country in the world to pay attention to the development of orphan drugs.

Before 1983, there were only more than 30 drugs on the market in the United States involved in the treatment of rare diseases.

The "Orphan Drug Act" has sorted out the definition of "rare diseases", including: (1) The number of patients in the United States is less than 200,000 per year; (2) The number of patients is more than 200,000, but there is no treatment for the disease in the United States.

FDA's definition of orphan drugs (source: FDA official website)

The FDA revised the orphan drug rules in January 1991, December 1992, October 2011, and June 2013.

·Clinical research and clinical trial expenses enjoy a 50% tax credit preferential policy;

·Reduction of prescription drug application fees;

·After thedrug is approved, it enjoys a 7-year market exclusivity period;

·Open up a "green channel" for orphan drug approval;

·Appropriate 30 million US dollars each year to support phase I to phase III clinical trials of products that have obtained orphan drug qualifications (2013-2017 fiscal years).

Take the market exclusivity period of up to 7 years after the orphan drug is on the market as an example.

Since then, many multinational pharmaceutical giants have joined the ranks of orphan drug development and have reaped great returns.

Up to now, Roche's Avastin (bevacizumab) is the product that has received the most orphan drug qualifications, and 11 sub-indications have been extended by the exclusive period due to orphan drug qualifications (see Table 1).

Table 1 List of bevacizumab orphan drug qualifications

Note: As of 2021/2/26

In June 2017, the FDA issued the "Orphan Drug Modernization Plan", which proposed to process all orphan drug eligibility applications submitted for more than 120 days within 90 days, and all new applications must be responded to within 90 days.

According to the FDA official website, since the implementation of the "Orphan Drug Act" in 1983, the number of orphan drug qualifications granted by the FDA and the number of approved orphan drug indications have increased year by year.

Table 2 Drugs with the most qualifications for orphan drugs

In recent years, Chinese pharmaceutical companies have also increased their applications.

It can be said that the US "Orphan Drug Act" is a huge advancement and a driving factor for the development of new drugs.

A study published in the American Journal of Clinical Oncology stated that pharmaceutical companies are taking advantage of policy loopholes to substantially declare orphan drugs, which seems to run counter to the original intention of the Legislative Orphan Drug Act.

Conclusion

The introduction of policies is like the emergence of new technologies.
It is a double-edged sword.
How to make better use of policy dividends for patients is also constantly challenging the original aspirations of practitioners.
The theme of this year’s "International Day of Rare Diseases" is "Rare is many.
Rare is strong.
Rare is proud.
" Conspiracy.

Undoubtedly, the concerted efforts of public welfare, business, and policies can encourage more people to pay attention to patients with rare diseases, more pharmaceutical companies to increase the development of orphan drugs, and more policies to support the research and payment of drugs for rare diseases.
.
Science and technology are good, policies are good, and people are good.
They are the source of continuous progress in the pharmaceutical industry.