Clinical trials are the culmination of many years of research and development. The amount of years and the amount of capital it takes to test any experimental therapies in humans is often prohibitive.
Only for-profit companies can finance the entire clinical development of innovative technologies. Because it costs millions of dollars in fees and developmental costs and because the risk of failure is high, successfully tested experimental technologies are made available to patients at a price based on 3 factors: the cost of development, the cost of manufacture, and the size of the “disease population”. For the same developmental costs, the bigger the disease population, the smaller the price of the therapy to the patients.
In rare diseases like LGMD2I, the disease population is so small that companies are not sure they can recoup the cost of development, and often shy away from developing innovative treatments for these diseases.
Strategies, like the ones below, are being put to bear to help resolve these challenges
- To decrease the cost of development
- One way to do that is to decrease the number of participants required to complete clinical studies.
- To create appropriate research tools
- Established and easily available research tools are very appealing to R&D organizations. With these tools (animal models, antibodies,…), companies can easily test the feasibility of a project and measure the efficacy of a technology right out of the bench.
- To simplify requirement, outcome, design of clinical trials and adapt them better to the need and abilities of the patients
- In many muscular dystrophy clinical studies, the main outcomes (measures of muscular function) are the 10-meter walk and the 4-stair climb. What about the people that cannot do that?
- Active patient communities
- When a company initiates a clinical trial, it has already put a lot of resources in play, and plans to continue doing that for many years. The more willing patients are to participate in the study, the more willing the company is to continue the clinical development of a technology with these patients
- Active and pro-active patient communities are more likely to help companies define (and test) what matters to them. Companies are more likely to develop a treatment that aim to resolve an actual need.
- Take the development into own hands
- If companies do not want to get involved early, one solution for patient community in rare diseases is to fund the early development of a promising technology. While it is challenging because it costs enormous amount of capital, it is not impossible.