The traditional model
Under the traditional model, discoveries are made in research laboratories and then patented. This can happen in industry laboratories, but often also in public institutions such as academic hospitals. In the latter case, discoveries are subsequently licensed or sold to industry, usually before so-called Phase 3 clinical trials begin—the expensive studies involving large groups of patients. The industry then brings the product to market, and if the discovery originated from another party, that party receives a percentage of the revenue generated by the industry: royalties.
Why rare diseases are under pressure
The industry operates with the aim of maximizing profit. This creates a problem for rare diseases with small patient populations and therapies that are effective for a very long time. To recover both development costs and investment costs (including risk premiums), and still generate sufficient profit to satisfy shareholders, companies must charge a very high price per patient.
The impact of high prices
These high prices are increasingly becoming an obstacle to reimbursement, which can lead to declining revenues for companies. This uncertainty about future revenue discourages companies from investing in the development of new gene therapies for rare diseases.
Our model: accessible to everyone
Our model focuses on making treatment available to every patient, at transparent costs and socially acceptable margins—solely to ensure the continuity of our company and to enable future developments. That means no profit distribution to shareholders.
System change is necessary
This also requires a system change among the authorities that grant approval to bring medicines to market. As a society, we need to look differently at new therapies for rare diseases in order to make these important innovations affordable again. The authorities are already aware of this, but change takes time. Together with Erasmus MC, we collaborate in the Netherlands within the DARE-NL and FAST initiatives.
