There is something cynical about the fact that although orphan drugs (OD) are now available for some rare diseases, they are so expensive that the treating physician hardly knows how to treat patients with them. Solutions are therefore urgently needed to improve the supply situation. One possibility is to distribute the costs as they arise, and thus also the responsibility, among different partners.
The dilemma of these expensive innovations is obvious: While no drugs for rare diseases were developed for decades, the market has changed in the last eight years due to new laws, greater public pressure and expiring patents. On the one hand, this is very gratifying, as around 300 million people worldwide suffer from one of the known 7,000 rare diseases – on the other hand, however, patients are wondering why the urgently longed-for drug is now not covered by health insurance.
Expensive health care system
Health insurance funds are economically managed administrative bodies for social, i.e. broad, health care. They can only spend at most as much money as the insured pay in and must consider a cost-benefit ratio. The total expenditure for our modern healthcare system in Switzerland is already 65 billion per year (source BAG). If the expenses for orphan drugs were added to this, the bill would be overstretched: With estimated annual costs of CHF 100,000 per patient for a new OD, a good 50 billion francs would be added to the bill for just under half a million people affected (estimate, Federal Court of Lausanne 2011). The modern Swiss healthcare system cannot cope with that by a long shot, especially since we already afford one of the most expensive in the world.
Solutions
Every medical professional would like to help his patients – especially when it comes to a disease that in the majority affects children and, in addition to a great restriction of the quality of life, also results in a short lifespan.
The fact that health insurers refuse to cover costs in most cases and that the FOPH imposes restrictions on certain patient groups – as in the case of hepatitis C therapy – calls for urgent solutions. Fortunately, however, new ideas and answers are emerging for this.
One is in the way we innovate. If different partners share the responsibility and costs of developing a new drug and it ends up costing less, it can be offered at a lower price. Orphanbiotec AG is one example of this. Here, partnerships and innovation form the backbone for shared success in responsible entrepreneurship. Spend less money and still achieve the goal – actually a truism, but one that some pharmaceutical companies and investors have not yet achieved. An investor who invests in a new drug expects not only the promised effect, but also that it can be sold in sufficient numbers to generate a sustainable profit.
Innovation through investors
There are now signs of a change in thinking on the part of investors, as more and more people are looking for a “responsible investment” where entrepreneurial funds are used to address a hitherto unsolved social or medical problem. The so-called “crowd-investing”, which has been very successful in recent years, reduces the investment risk of the individual, as the amount invested is small and the risk is thus shared with many others. The model has already been used successfully in various countries.
A math problem
Our simulation involves an investment in a new orphan drug. The risk is minimized by relying on repositioning, i.e. the use of (in our case natural-based) active ingredients with a known efficacy and side effect profile. Since the market for a new OD does not have to be fought for – patients are already waiting for the therapeutic agent – the normally very high marketing costs are low here. Drug X should later be affordable for insurance companies and cost no more than CHF 4000 per year per patient. A total of 15 million Swiss francs will be needed for the development. The new OD targets a market of 100,000 patients in Europe. The investment is divided among various sources. Through crowd-investing, 5 million francs are to be raised – a moderate interest rate of approx. 3% p.a. can be expected.
We would like to know from you how much money you would be willing to invest via crowd-investing in such an orphan drug project? We welcome your input on this on the Orphanbiotec Foundation’s online blog: http://blog.orphanbiotec-foundation.com.
HAUSARZT PRAXIS 2015; 10(6): 9
