As the number of available gene therapy treatments increases, it becomes unaffordable for society. Healthcare payers, such as insurers and governments, are therefore increasingly sounding the alarm. And the uncertainty in the market is already causing biotech and pharmaceutical companies to withdraw their support from gene therapy for rare diseases. As a result, these promising new treatments no longer even reach the patient.
Wanting to look and think differently.
The classic model is that discoveries are made in the research laboratory and then patented. This can happen in the laboratories of the industry itself, but often also in the labs of public institutions, such as academic hospitals. In the latter case, the ideas are then licensed or sold to the industry, usually before the expensive so-called phase 2 and phase 3 clinical studies begin, with large groups of patients. The industry brings the product to the market and if the discovery comes from another party, that party receives a percentage of the turnover that the industry achieves: royalties.
The industry does this with the aim of making as much profit as possible. This creates a problem when it comes to rare diseases with few patients and therapies that are effective for a very long time. The industry then has to charge a high price per patient to recoup their development costs and also to make enough profit to satisfy their shareholders.
However, the high prices are now increasingly becoming a barrier to receiving reimbursement for therapies, which may result in less and less turnover for companies. This uncertainty about future turnover means that companies no longer want to invest in developing new gene therapies for rare diseases.
Our model is focused on the availability of the treatment for every patient. At transparent costs and socially acceptable margins, only to be able to guarantee the continuity of our company.
How are we going to run our company?
We are a public company: a 100% subsidiary of Erasmus MC. We have a social goal, which we adhere to. Like every academic hospital, Erasmus MC is mainly financed by taxpayers and health insurance premium payers. We also receive money from the Prinses Beatrix Spierfonds; money that donors have given. In short, society pays and we want to handle that money carefully and efficiently.
For that, we also need a system change at the authorities that have to give permission to market the drug. As a society, we need to look differently at these types of new therapies for rare diseases, to make these types of important innovations affordable again. The authorities are already aware of this, but change takes time. With the Erasmus MC, we work together in the DARE-NL and FAST initiatives in the Netherlands.
We are always looking for parties and people who can help us.
So will you help?
Prinses Beatrix Spierfonds
Help mee in de strijd tegen spierziekten en geef voor wetenschappelijk onderzoek. Want alleen met onderzoek kunnen we medicijnen ontwikkelen voor alle mensen met een spierziekte in Nederland.
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