The "Orphan Drug Act" has been implemented for 38 years, the FDA has granted 5808 orphan drug qualifications and approved 952

544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>

544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'> 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.
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Although the above-mentioned diseases have different pathogenesis and clinical manifestations, they can all be classified as "rare diseases" because of the small number of cases.

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.
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Although the above-mentioned diseases have different pathogenesis and clinical manifestations, they can all be classified as "rare diseases" because of the small number of cases.
544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>According to WHO's definition, diseases with a total population of 0.

65‰ to 1‰ can be defined as " rare diseases .
" At present, there are more than 7,000 rare diseases confirmed internationally, accounting for about 10% of human diseases and affecting about 6% to 8% of the global population.
According to the definition of WHO, diseases with the number of patients taking up 0.

544px;white-space:normal;background-color:#FFFFFF;text-align:center;line-height:normal;box-sizing:border-box ;'> FDA's definition of orphan drugs (source: FDA official website)

FDA's definition of orphan drugs (source: FDA official website)544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>The FDA revised the orphan drug rules in January 1991, December 1992, October 2011, and June 2013.

Several revisions have continued the recommendations to support the development of orphan drugs, and a series of supporting policies such as accelerated approval for drugs that have been identified as orphan drugs have continued to increase the social awareness of orphan drugs, and the enthusiasm of pharmaceutical companies for the development of orphan drugs has also continued.
Warm up.
such as:The FDA revised the orphan drug rules in January 1991, December 1992, October 2011, and June 2013.
Several revisions have continued the recommendations to support the development of orphan drugs, and a series of supporting policies such as accelerated approval for drugs that have been identified as orphan drugs have continued to increase the social awareness of orphan drugs, and the enthusiasm of pharmaceutical companies for the development of orphan drugs has also continued.
Warm up.
such as:

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

• Reduce and waive prescription drug application fees;

• After the drug 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).
  

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

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

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

Reduce and waive prescription drug application fees;

Reduce and waive prescription drug application fees;

Reduce and waive prescription drug application fees;

After the drug is approved, it enjoys a 7-year market exclusivity period;

After the drug is approved, it enjoys a 7-year market exclusivity period;

After the drug is approved, it enjoys a 7-year market exclusivity period;

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

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

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).

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).

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).
544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>Take the market exclusivity period of up to 7 years after the orphan drug is on the market as an example.

This means that during the exclusive period, the FDA will not approve a second application for the same product with the same indication, which is significantly longer than the 5-year exclusive period for the New Chemical Entity (NCE), and the 3-year exclusive period for drug applications submitted due to “changes” The period and the patent challenge period of generic drug ANDA is 180 days.
Take the market exclusivity period of up to 7 years after the orphan drug is on the market as an example.

Note: As of 2021/2/26544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>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.
This plan once again stimulated the industry The enthusiasm for applying for orphan drug qualifications, the number of grants has also soared.
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.
This plan once again stimulated the industry The enthusiasm for applying for orphan drug qualifications, the number of grants has also soared.
544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>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.
As of February 26, the FDA has granted 5808 orphan drug designations and approved 952 orphan drug indications.
Among them, the drugs with the most orphan drug qualifications are Avastin, Lynparza, Imbruvica, Keytruda, Gleevec, Kalydeco, Revlimid, opdivo, Humira, Adcetris (see Table 2).
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.
As of February 26, the FDA has granted 5808 orphan drug designations and approved 952 orphan drug indications.
Among them, the drugs with the most orphan drug qualifications are Avastin, Lynparza, Imbruvica, Keytruda, Gleevec, Kalydeco, Revlimid, opdivo, Humira, Adcetris (see Table 2).

544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>In recent years, Chinese pharmaceutical companies have also increased their applications.
Since 2014, Chinese pharmaceutical companies have obtained more than 70 orphan drug qualifications.
Especially in 2020, a total of 19 Chinese companies have obtained 35 FDA orphan drug qualifications, and Yasheng Pharmaceutical has obtained 8 orphan drug qualifications with 4 drug capsules as the biggest winner.
In recent years, Chinese pharmaceutical companies have also increased their applications.
Since 2014, Chinese pharmaceutical companies have obtained more than 70 orphan drug qualifications.
Especially in 2020, a total of 19 Chinese companies have obtained 35 FDA orphan drug qualifications, and Yasheng Pharmaceutical has obtained 8 orphan drug qualifications with 4 drug capsules as the biggest winner.
544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>It can be said that the US " Orphan Drug Act " is a huge advancement and a driving factor for the development of new drugs.
However, some drug administration agents believe that the continuous blowout of orphan drug applications has caused a substantial increase in the FDA’s workload, a sharp increase in work pressure, and a rapid increase in review efficiency when the FDA’s rare disease product development office staff did not increase accordingly.
It is lowered.
It was previously proposed that the feedback period within 90 days has actually been extended to 120 days or even longer.
It can be said that the US " Orphan Drug Act " is a huge advancement and a driving factor for the development of new drugs.
However, some drug administration agents believe that the continuous blowout of orphan drug applications has caused a substantial increase in the FDA’s workload, a sharp increase in work pressure, and a rapid increase in review efficiency when the FDA’s rare disease product development office staff did not increase accordingly.
It is lowered.
It was previously proposed that the feedback period within 90 days has actually been extended to 120 days or even longer.
The Orphan Drug Act is a huge advancement and driving factor for the development of new drugs.
However, some drug administration agents believe that the Office of Rare Disease Product Development FDA544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>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 .
It believes that some pharmaceutical companies gain more profits by gambling with the system .
Behind their search for orphan drug status, there is a long-term plan to ensure that the drug has a wider range of indications, not just for people with rare diseases.
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 .
It believes that some pharmaceutical companies gain more profits by gambling with the system .
Behind their search for orphan drug status, there is a long-term plan to ensure that the drug has a wider range of indications, not just for people with rare diseases.
The Orphan Drug Act in clinical oncology in the United States .
Some pharmaceutical companies gain more profits by gambling with the system.
544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>ConclusionConcluding remarks544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>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.
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.
"International Rare Disease Day"544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>544px;text-align:justify;white-space:normal;background-color:#FFFFFF;line-height:2em;box-sizing:border-box ;overflow-wrap:break-word ;'>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.
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Science and technology are good, policies are good, and people are good.
They are the source of continuous progress in the pharmaceutical industry.
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.