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Our view on rare disease market
Our view on rare disease market
Very few products achieve blockbuster (multi-billion dollar) sales since the number of patients is extremely small for each disease
Regulatory and academic support are always available and can help accelerate development of new drugs to tackle rare diseases. Thus, new competitors tend to arrive to market much sooner than anticipated
Market potential of treatments for rare diseases
Market potential of treatments for rare diseases
Few treatments for rare diseases have sales greater than $1 billion (100 billion yen). The antibody drug Eculizumab (Soliris) is a rare exception. This drug, which is used to treat paroxysmal nocturnal hemoglobinuria, had sales of over 300 billion yen in 2017.
The development of this orphan drug was a fabulous success for Alexion. That said,
as a corporate management strategy, aiming to develop blockbuster treatments for rare diseases is a mistake. This is not merely an ethical issue, it is backed by hard-won marketplace experience.
as a corporate management strategy, aiming to develop blockbuster treatments for rare diseases is a mistake. This is not merely an ethical issue, it is backed by hard-won marketplace experience.
In 2011, Sanofi acquired Genzyme, the developer of drugs that treat rare diseases such as Pompe disease, Fabry disease, and Gaucher disease. The story of Genzyme was made into a Hollywood film. The earnings of Genzyme's rare diseases business have been flat at about $2-3 billion (200 billion yen) for many years.
The thing about rare diseases is, few people suffer from them. As soon as these treatments reach the public, market penetration is 100%. Prices for these drugs are extremely high from the outset, so there is little hope of increasing them. On top of that, government and academic institutions are generous in funding research in new drugs for rare diseases, and that encourages competition.
Shire developed Replagal as a treatment for Fabry disease, and Vpriv to treat Gaucher disease, and managed to win some of Genzyme's market share.
The 7 trillion yen Takeda proposes to pay for a company with 3 trillion yen of goodwill and intangible fixed assets is excessive. The idea that Takeda will be able to recover this investment in the area of rare diseases is unrealistic. There are also ethical issues. We want Takeda to be a company that lives up to its social mission.
The 7 trillion yen Takeda proposes to pay for a company with 3 trillion yen of goodwill and intangible fixed assets is excessive. The idea that Takeda will be able to recover this investment in the area of rare diseases is unrealistic. There are also ethical issues. We want Takeda to be a company that lives up to its social mission.
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