Problem: What problem is this project trying to address?
There are 7,000 existing rare diseases in the world, affecting around 250 million people. However, in Europe for example, only five percent of patients have approved therapeutic solutions. Under the current system, patients and their families undergo a great deal of stress, anxiety and pain while waiting for treatments to be developed and approved, and they place blame on pharmaceutical companies and regulatory bodies, which in their eyes only lengthen the process and cause barriers to treatment.
Repositioning of existing drugs offers opportunities to accelerate treatment discovery, since repositioned drugs have already passed a significant number of tests and can bypass much of the early cost and time needed to bring a drug to the market. However, many challenges exist which prevent the mainstream use of drug repositioning for rare diseases.
The first is a structural lack of coordination among the actors in the repositioning process. Actors involved in developing drugs work in silos and do not have clear incentives to coordinate; for example, it is considered a conflict of interest for pharmaceutical companies to engage with patients. In addition, regulatory bodies maintain cautious relationships with the private sector and patient groups to remain nonaligned. The result can be mistrust and fear, and a lack of collaborative spirit, which makes repositioning difficult.
Several well-intended efforts do try to work on drug repositioning for rare diseases, but they face challenges. Patient groups engage in drug repositioning, but tend to focus on one particular rare disease instead of working across rare diseases, which can result in competition for resources. Biotech companies engage in drug repositioning, but it is uncommon for them to focus on rare diseases because of low profit margins. Furthermore, their engagement with patient groups, a critical element for successful repositioning efforts, is limited. Finally, several non-profits have emerged on the scene to fundraise and drive research for rare diseases; however, none of them are implicating pharmaceutical companies directly in the process. It is extremely challenging to cover the full cost of the entire repositioning process – from drug development, to clinical trials, to commercialization – without engagement of the private sector.
The current pharmaceutical industry business model focuses mainly on developing blockbuster drugs that impact millions of people. It is thus challenging for pharmaceutical companies to see the market in rare diseases, which is made up of approximately 7,000 rare diseases that impact many smaller populations (the threshold is 750 patients for 1 million individuals). Although repositioning opportunities can be of high therapeutic value, they cannot be commercialized at premium price. Furthermore, the internal process that a pharmaceutical company must undergo to reposition a drug is often considered too resource-intensive, requiring agile project management and stakeholder engagement, which are not the specialties of pharmaceutical companies. To date, there is currently no externally facing operational and financial model for drug repositioning that enhances the opportunity for pharmaceutical companies to invest in the clinical testing and commercialization phases.
These challenges come with a counter opportunity. The pharmaceutical industry needs to think about new business models for long-term survival. The industry is increasingly challenged by identifying and selling blockbuster drugs to the masses. With the shift toward personalized medicine (notably based in scientific discoveries in genome mapping), the blockbuster model is no longer the only viable model. However, pharmaceutical companies cannot make the shift to new models alone, and more than ever needs access to agile and neutral partners that are able to bridge the efforts of patient groups, public biomedical institutes and regulatory bodies to adapt to more individualized ways of conducting drug development and healthcare.
Solution: What is the proposed solution? Please be specific!
In a health system that lacks incentives to coordination, EspeRare serves as a neutral and trusted broker to coordinate and drive the complicated process of drug repositioning among the major actors: patient groups, regulatory bodies, clinical research partners and pharmaceutical companies. Through this neutral positioning, Esperare applies all the pieces of a comprehensive solution – R&D and project management expertise, patient-centricity, and hybrid financing mechanisms – to advance the discovery of new treatments for underserved patients.
An important piece of this solution is the engagement of the pharmaceutical industry. Under a system in which pharmaceutical companies rarely engage in drug repositioning for rare diseases – as it is considered resource intensive with low returns – Caroline provides them with an attractive and viable alternative. EspeRare incentivizes investing in drug repositioning by pooling additional financial resources from patient groups, reducing transaction costs and offering pharmaceutical companies various win-win exit strategies depending on whether they want to keep or concede the commercialization and intellectual property. Furthermore, this repositioning process opens up future business opportunities for pharmaceutical companies by preparing them for the evolution toward personalized medicine servicing smaller niche groups.
Caroline’s approach to drug repositioning extends beyond its benefits to rare disease populations. Her repositioning approach can be replicated to other mainstream diseases as health systems more generally move toward more personalized care approaches. She is also considering replicating her approach to other health domains, such as prevention, which faces similar challenges to inventing sustainable economic models and avoiding two-tiered medicine.
EspeRare is currently undergoing two repositioning programs and has 5-7 more in the pipeline, set to launch within the coming years. Not only will these programs accelerate the birth of new treatments, but they will also improve patients’ quality of life by providing guidance, support and experimental protocols. At a more systematic scale, EspeRare’s model - with investment pooling and multi partnerships - is pushing the health sector to improve and cheapen access to health more broadly.