Caroline, a mother to a child with a rare disease who spent seven years working in the pharmaceutical industry, recognized that a novel financial and operational model was needed to develop repositioning opportunities that often “sits on shelves” of pharmaceutical companies, thus give a chance to much-needed unexplored treatments to reach patients underserved by medicine.
Coming from the pharmaceutical sector, Caroline was able to identify the biggest barriers to biopharmaceutical companies investing in drug repositioning for rare diseases. The first was the high R&D cost and the second was the complex stakeholder process. EspeRare overcomes both by offering an attractive and viable commercial model for these companies to engage. She reduces the overall costs of the repositioning process by pooling additional investment resources (mainly public grants, patient group funds and pro-bono partnerships with the best experts possible). In most situations, pharmaceutical companies contribute about one quarter of the cost of what they would invest if they were working alone. EspeRare also makes the process of repositioning drug more efficient by diving stakeholder engagement and coordination of collaboration-intensive activities that requires agility and are complexes to manage for large pharmaceutical companies, such as working with patient organizations, academic center, key opinion leaders and regulatory authorities.
Caroline maintains patient-centricity above all in order to work across silos and include the patient voice at each step of the process. With patient groups, EspeRare identifies critical medical unmet needs and prioritize rare diseases of focus. She then uses a data mining process that integrates data on existing drugs and rare diseases pathophysiological processes to uncover unexplored repositioning opportunities. This data driven approach allows her to more systematically identify and evaluate repositioning opportunities with high therapeutic potential across a range of rare diseases.
Once repositioning opportunities are identified, EspeRare negotiates, with asset owners, rights to develop the existing drugs in the rare disease of interest. To further incentivize the deal making, EspeRare offers flexible partnership models, depending on whether the asset owner is looking to keep the exclusivity on the commercialization or concede the intellectual property to EspeRare. However independently of the business model that is favored, social and commercial win-win “guiding principles” are established between EspeRare and its biopharmaceutical partner to secure ethical development and affordable patient access to treatments.
The outcome of Caroline’s repositioning approach is to give a chance, accelerated and de-risked drug repositioning, which is not achievable with traditional approaches. It applies its drug development know-how to conduct preclinical and early clinical assessments of the therapeutic opportunities. EspeRare sits at the interface of the major actors (patient groups, regulatory bodies, clinical research partners) and use its in-house pharmaceutical R&D expertise, to drive all rang of development activities required to conduct a robust evaluation of the therapeutic potential of its programs. The key here is strategic coordination and constant engagement of patients and other key partners throughout the process. For example, EspeRare saves significant time and gains access to a trusted interaction with patient organization by tapping into Ashoka Fellow Sharon Terry’s network of 1,500 patient groups, which is a huge lever to integrate the patient voice and access to data and patient samples at the very onset of drug development activities. A second outcome is guiding and accompanying patients as trusted allies during these often complicated and difficult phases. For example in those diseases where the lack of biomedical knowledge if often a critical barrier to therapeutic development, Caroline is supporting the development of key components of this “drug development infrastructure” such as clinical end-points, natural history data, prevalence information, ect…
EspeRare is currently working on its first two ongoing programs, the first being the development of Rimeporide, a drug (developed by Merck for heart failure and abandoned in Phase I trials), which EspeRare is currently repositioning in Duchenne Muscular dystrophy, a deadly pediatric myopathy for which no appropriate treatment currently exists. EspeRare has also initiated a second repositioning program in a rare renal disease; with a UK biomedical center expert in that disease, together they have initiate research activities to demonstrate that a drug, originally developed in a brain cancer until Phase III trials, has a potential to treat patient affected by his debilitating renal disease.
Caroline’s strategy to engage future companies is to look for those internal champions that see the longer-term vision and the opportunity in drug repositioning for rare diseases. She is engaging those business leaders that recognize the opportunity to evolve R&D and drug development for personalized medicine. Already, Caroline has found these champions in Merck Serono and she is currently exploring other pharmaceutical partnerships as well.
Finally, Caroline is thinking beyond rare disease treatment. She sees rare diseases as the best case study to explore new models for health personalization challenges. By developing innovative strategies for rare diseases, EspeRare aims to open a new path to affordable drugs and better healthcare as a whole. Her approach involves: a) implication in the of policies that will prevent two-tiered medicine, for instance within working groups in Brussels; and b) broadening her scope of research to non-rare disease repositioning opportunities, for instance to treat a specific subpopulation of autistic children.