It is estimated that there are 5,000-8,000 rare diseases in the world. Although each rare disease is only a few hundred or thousands of people, it is rare in the United States and the European Union. The total number of sick people adds up to at least 55 million. In the United States, 33 million people suffer from more than 7,000 rare diseases. In Europe, about 6% to 8% of people suffer from rare diseases. If all rare diseases are classified as one type, they will suffer in reality. People with rare diseases are no longer “rare”. However, before the law was introduced, pharmaceutical companies did not pay attention to the development of orphan drugs because it was unprofitable. Faced with such an embarrassing situation, the United States began to legislate in 1983 and passed the world’s first Orphan Drug Act (ODA).
According to the Act, the US Congress provides policy incentives for the development of orphan drugs, including: (1) after the orphan drug is listed, it has exclusive rights within 7 years, and the FDA does not approve other new drug listings (NDA); (2) have priority in clinical trials; (3) provide development grants and research funds to orphan drug companies; (4) provide rapid approval channels for new drug applications for orphan drugs; (5) FDA can assist companies in completing clinical trials of orphan drugs Test; (6) The application fee for new drugs for orphan drugs is exempted. The European Union, Japan, Australia, Singapore, South Korea and other places have also promulgated the orphan drug law. The emergence of these bills has greatly promoted the development and listing of orphan drugs.
The current development of orphan drugs
Orphan drugs involve fewer patients, but they often have poor performance due to less competition and favorable policies. Good, R & D companies have made huge profits. At present, due to the difficulty in research and development of new drugs, pharmaceutical patents expired, and the impact of many generic drugs, profits are gradually decreasing. At the same time, the drug regulatory authorities are increasingly strict with drug application requirements, the cost of new drug research and development is rising, and the field of rare drug drugs is A new choice for large pharmaceutical companies. In 2016, the development of orphan drugs was excellent. The FDA-approved orphan drug growth rate was 6%, the EU orphan drug growth rate was 9%, and the Japanese orphan drug growth rate was 15%. Global orphan drugs have developed rapidly since 2003.
The development of orphan drugs in the United States, the European Union, and Japan
As the indications for orphan drugs continue to increase, the weight There are also more and more pound-bomb drugs. The leading orphan drug, Relilimid, has more than $7 billion in global sales in 2016 and more than $4 billion in sales in the US, making it the world’s best-selling orphan drug.
2016 US orphan drug sales TOP10
Orphan drug development prospects in the next five years
The orphan drug market has broad prospects. In 2016, sales of orphan drugs were US$114 billion, an increase of 12.2% over 2015. In the next five years, the total sales of orphan drugs will reach 209 billion US dollars, with a compound annual growth rate of 11% (2017-2022), which is twice the growth rate of the prescription drug market. By 2022, orphan drugs will account for 21.4% of global drug sales.
Global Orphan Drug Sales and Growth Rate (2000-2022)
Orphaned Pharmaceutical Company for the Next Five Years TOP 10
It is expected that by 2022, Celgene will rank first in the orphan drug sales list, becoming the world’s number one orphan drug manufacturer, and Bristol-Myers Squibb (BMS) and Novartis ranks second and third in turn. The rankings of Abbvie and Johnson & Johnson will continue to move forward. The top 4 orphan drug manufacturers have sales gaps of $400 million, and the sales of three of them are mainly dependent on one product, namely Celgene (product Revlimid) and Bristol-Myers Squibb (product). Opdivo) and Roche (product Rituxan). Among them, Revlimid accounted for 80% of Celgene’s sales, and Opdivo accounted for 68% of BMS. By 2022, 7 of the top 10 orphan drug manufacturers are expected to become global orphan drug companies, and the top 5 orphan drug manufacturers will account for nearly one-third (30.6%) of the orphan drug market.
Top 10 orphan drug companies in the next five years
Top 10 orphan drug sales in the next 5 years
It is predicted that in 2022, Revlimid is still the world’s best-selling orphan drug, which was approved by the FDA in December 2005 for the treatment of rare myelodysplastic syndrome, followed by Approved for rare disease indications – Non-Hodgkin lymphoma and multiple myeloma, and it is currently being developed for several other rare disease indications. Compared to 2016, Revlimid’s compound annual growth rate is 12%, with sales reaching $13.5 billion. Bristol-Myers Squibb’s Opdivo ranked second with sales of $9.1 billion, with an annual growth rate of 16%. In the top ten sales of drugs, Johnson & Johnson’s Darzalex, Imbruvica and Merck’s Keytruda grew. The trend was the strongest, with sales increasing from 570 million, 630 million, and 1.4 billion in 2016 to 5.8 billion, 3.4 billion, and 7.9 billion. The compound annual growth rate was 47%, 32%, and 33%, respectively. Of the ten products, only Roche’s Rituxan performed poorly and will experience negative growth.
Top 10 orphan drug sales in the next 5 years
Summary and Outlook
Since the United States enacted the Orphan Drug Act and the Hatch-Waxman Act, in just 30 years, orphan drugs have been blown out, and there are hundreds of drugs on the market. At the same time, the legal guarantee accelerates the innovation and investment of small and medium-sized biotechnology enterprises in the research and development of orphan drugs. Under the dual role of government guidance and technological development, the research and development of orphan drugs in American biological products has an absolute proportion. For China, there are a large number of rare patients in China, and the demand for orphan drugs is huge. The orphan drugs have low investment, high return, short development cycle and huge market space. However, as China has not yet introduced relevant policies and regulations, it still has to make a lot of efforts to improve the level of drug innovation in China.
Reference data and literature:
2. Incentives for the development of orphan drugs in Europe and America and their enlightenment to China
3. Orphan drugs and orphan drug legislation
This article was written by the author of the drug headline submission. The point of view only represents the author himself, does not represent the head of the drug, and welcomes exchanges and supplements.