According to a story from Raconteur, finding sufficient funding for the development of new therapies for rare diseases has always been a major challenge. Rare diseases are defined in the US as any disease that affects less than 200,000 in the country. While some rare diseases have active patient advocacy groups and foundations that are capable of making great strides in obtaining funding for research and drug development, this is most definitely the exception and not the rule.
Why Funding Development is Difficult
In many cases, the patient population is often too small for there to be major mobilization to help fund drug development. Orphan Drug designation and other similar measures that serve to facilitate and incentivize the development of rare disease drugs have gone a long way to help make funding and researching these treatment more appealing, but the situation could still be far better. The vast majority of rare illnesses have no licensed therapies.
Possible Methods
So how can rare drug development be funded? Drug repurposing is one method that could be of great utility in treating rare disease. This simply involves the testing and approval of therapies that are already in use for more common ailments and seeing if they work for a rare disease as well. This approach requires intensive research in order to understand what drugs could work, but if it is successful, the result is major saving of time and money. Repurposing could sharply curtail development costs from about $1 billion to just $50 million.
Another US company, Imara, is drawing funding by reducing the risk inherent to investing in rare drugs. This company is currently testing a possible drug which was shelved by another company as a possible treatment for sickle cell anemia.
One disadvantage of the Orphan Drug laws is that it often results in therapies that are often priced extremely highly. This is a serious barrier to access for many patients. A UK charity may have come with a new method that could help limit this problem called a social impact bond. The charity, called Findacure, wants to issue the bond (funded by investors) in order to fund clinical trials for ten repurposed drugs for rare diseases that have been identified as having an excessive management cost.
If a trial is successful, the projected savings would be used to pay back into the bond to fund more trials and satisfy the investors. While these new methods are not entirely fleshed out, they could be valuable new approaches that could become the future standard for rare drugs.
