The growing interest of the pharmaceutical industry in rare disease drugs has not showed any sign of wane. According to a new report of EvaluatePharma, the increase in R&D investment of relevant indications has converted into the overall growth of pharmaceutical product sales in recent years. There is no lack of pharmaceutical giants in this field, such as Roche, Boehringer Ingelheim, J & J (Actelion), Biogen and Sanofi.
The rare disease drugs in the report of EvaluatePharma include drugs first approved for one rare disease or approved for many indications and generating more than 25% sales from their rare disease indications.
Drugs for cancer treatment can almost meet the standard for rare disease drugs, while oncology has different commercial characteristics from rare diseases in the traditional sense, despite their attraction to the pharmaceutical industry due to many same reasons. To put it simply, the rare disease drugs stated here refer to drugs for long-term treatment of life-threatening rare diseases excluding oncology drugs.
Rare disease drugs are more profitable than non-rare disease drugs owing to their market exclusivity period and pricing advantage. The total sales of rare disease drugs reached USD 114 billion in 2016, accounting for 16.4% of the global non-generic drug market, and these figures are forecast to grow to USD 209 billion and 21.4% in 2022, doubled over the last decade. Their CAGR (Compound Annual Growth Rate) will be 11.1% in the next 6 years, twice that of non-rare disease drugs.
The sales of the top 25 rare disease drugs excluding oncology will be USD 37.4 billion in 2022, accounting for about 3.8% of global pharmaceutical product sales; their CAGR will be 18% in the next 6 years, almost three times that of market average (see the following table).
Global sales of Top 25 rare disease drugs in 2016/2022
|
Pharmaceutical product |
Company |
Indication |
2016 |
2022 |
|
Soliris (eculizumab Solution for Intravenous Infusion) |
Alexion |
Paroxysmal Nocturnal Hemoglobinuria (PNH); Atypical Hemolytic Uremic Syndrome (aHUS) |
28.43 |
51.39 |
|
Orkambi (lumacaftor/ivacaftor) |
Vertex |
Cystic fibrosis |
9.80 |
36.34 |
|
Ofev (nintedanib capsules) |
Boehringer Ingelheim |
Idiopathic pulmonary fibrosis |
7.63 |
18.27 |
|
Advate (Recombinant Human Coagulation Factor VIII) |
Shire |
Hemophilia A |
12.58 |
17.40 |
|
HyQvia (Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase) |
Shire |
Primary Immunodeficiency (PI) |
...
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