Fantastic Returns on Rare Disease Drug Development

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

Data source: EvaluatePharma; sales unit: USD 100 million; pharmaceutical products exclude oncology drugs and include only the life-threatening rare disease drugs

In addition, Alexion’s Soliris ranked 27^th in the sales rankings, but its sales are forecast by EvaluatePharma to grow to USD 5.1 billion to rank 9^th in the rankings in 2022 after its indication of generalized myasthenia gravis (GMG) is approved.

Rapidly changing competition

Positions of the top rare diseases change significantly compared to one similar analysis last year (Industry work on rare diseases continues to bear fruit, March 3, 2016).

For example, Roche’s Esbriet (pirfenidone) for idiopathic pulmonary fibrosis (PF) and Novo Nordisk’s NovoSeven (recombinant coagulation Factor VIIa) for hemophilia would have prominent places in 2020 according to data last year, but it seems that they will disappear in 2022 in the report this year, which is clearly due to the competitive products in the therapeutic fields of their indications.

Biogen’s Spinraza (nusinersen) for the spinal muscular atrophy (SMA) of children and adults is expected to join Top 10 rare disease drugs by 2020; Sarepta’s Exondys 51 (eteplirsen) for Duchenne muscular dystrophy will enter Top 30.

By Xu Guanyu, Li Minhua

From PHIIC