The Value of Public-Private partnerships

There are many contributors on the journey from idea to a therapy and each play a critical role to make the miracle happen, from fundamental research, through medicines discovery and development, to public and private research infrastructures*.  New scientific clues generate private investments. This is a virtuous cycle with different phases occurring in sequence.

There are 6000-7000 rare diseases identified, with some 95 per cent without a cure or treatment option. Rare diseases are some of the most complex, scientifically challenges diseases to tackle.

This challenge is too big for one organisation or even one industry to take on alone. It calls for a paradigm change and breaking the silos and the sequential approach.

No one company, government, academic or civil society group has the research expertise or financial firepower, but by working together as partners, real progress is possible.

Public-private partnerships (PPPs) which bring together knowledge and know-how from all sectors, make it possible to share risks, accelerate the drug development pathway and work in those areas where there is both an important unmet medical need and a societal value.

Through public private collaborations we can understand needs of all stakeholers and design a framework that will decrease the level of risks for all. It is collaboration on the scale of those witnessed during Covid-19– between governments, companies and through public-private partnerships – that is needed if we are to improve the rare disease ecosystem in Europe and meet IRDiRC’s goal to reach 1,000 new therapies in this decade.

There are many models of public private collaboration, ranging from bilateral engagement between one company and a university or a group of players, to large institutional partnerships such as IMI/IHI in Europe (with Screen4Care, ARDAT, STOPFOP amongst others) or the Bespoke Gene Therapy Consortium in the US, that bring together several companies in a precompetitive space to expose and address knowledge gaps and structural weaknesses together with academia, patients, and other key players.

From theory to practice: Examples of public-private collaborations in the field of rare disease


Bespoke Gene Therapy Consortium (BGTC)

Together 4 Rare Diseases

* Translational medicine is an ecosystem, connecting a group of independent but interrelated stakeholders to promote advances in healthcare. It is comprised of patients, academic and industrial research and development professionals, commercialization teams, investment capital, regulatory agencies which enforce government policies, ethics and health insurance payers. These stakeholders often have conflicting goals and objectives and are operating within an evolving ethical framework. In today’s world that is so interconnected by technology, the new ideas and advances in healthcare echo across disciplines to create an extensive and interrelated system.

Mahant, V. Translational medicines “Ecosystem”. J Transl Med 18, 158 (2020).