Implementation of a collaborative drug development strategy for unaddressed rare diseases

There are over 7000 rare diseases known, and 95% of them have no treatment option. Many of these diseases are devastating and life-limiting, and many affect children. 3.2 million people in the UK are affected by a rare disease, and those affected globally are estimated at 400 million. At the same time, the small market size of each individual rare disease, combined with limited knowledge and low awareness, together create a formidable barrier hindering the development of treatments. As a result, in reality pharmaceutical companies cannot commercially justify allocating resources toward rare-disease drug development using the traditional pharma model. This creates an unacceptable gap between unmet needs and unharnessed resources.

Aspire biosciences, a newly formed company, was founded based on a novel approach to rare disease drug development. We aim to find new treatments for unaddressed rare diseases by creating and nurturing working partnerships between rare disease patient organisations and drug development companies. Having analysed a number of anecdotal success stories in rare disease drug development, all sharing a component of serendipity, our founder designed a methodology which bridges the gap by shifting the cost-benefit function of rare-disease drug development. The approach involves systematically identifying relevant assets within drug development companies, and partnering patient organisations with the companies around these opportunities. It effectively eliminates the companies' search costs, streamlines key steps in drug development, and makes the process more cost-effective and potentially viable.

To test the concept, our founder ran a pilot event based on highly structured speed-dating between patient groups and companies. Pharma teams (each consisting of a decision maker and a scientist) met with several patient-group teams (each consisting of a patient representative and a medical expert) for focused one-to-one discussions of potential treatments. This proof-of-concept event received excellent feedback from participants, led to several initial collaborations, and drew interest from investors. It forms the basis for our _Systematic Serendipity_ framework. Subsequently, we founded Aspire Biosciences, which currently implements this solution via specialised partnering events, and is looking to further implement it through a searchable database and AI platform. We are currently organising our next two partnering events, and expanding our team. Aspire Biosciences will harness both human and machine intelligence to identify drug development opportunities, and pursue them through nurturing working partnerships between rare disease patient groups and drug development companies.