The Rare Responsibility
This space explores how the pharmaceutical industry could take on rare diseases as a collective.
There are thousands of rare diseases, but most are forgotten by traditional drug development.
- Estimated rare diseases: ~7,000 globally
- Without approved therapies: ~95%
- People affected globally: ~300 million
The Rare Responsibility
Dear Impossible Readers,
Many ideas crossed my mind before I started this blog. Ultimately, this idea is the one that sparked. After all, what could a single person in an 8+ billion world population achieve?
The Rare Responsibility Consortium is a visionary, non-profit initiative that calls on the pharmaceutical industry to unite in a shared commitment. Each company adopts one rare disease and dedicates resources toward developing a meaningful treatment. Rather than competing for the same targets, this consortium encourages collaboration and knowledge-sharing. Each participant contributes according to their unique scientific strengths and infrastructure.
Pharmaceutical companies are uniquely positioned to lead this effort. They already possess the capital, expertise, clinical networks, and regulatory experience that complement the work of small startups, academic labs, and NGOs. By repurposing existing resources, they can make a transformative difference with minimal commercial risk and enormous human impact.
Welcome to my blog,
Yours Possibly
Further Reading
Baynam, G., Groza, T., McMurry, J.A. and Dawkins, H.J.S., 2020. How many rare diseases are there? Nature Reviews Drug Discovery, 19(2), pp.77–78.
Bowen, A.E. and Nargundkar, S., 2025. Drug development for rare diseases: a case for patient-centricity, equity, and access to clinical trials. Journal of Rare Diseases, 4(1), p.15.
Denton, N., Mulberg, A.E., Molloy, M., Williams, M.J., Urquhart, L. and Haffner, M.E., 2022. Sharing is caring: a call for a new era of rare disease research and development. Orphanet Journal of Rare Diseases, 17, p.389.
Douglas, C.M.W., Aith, F.M., Boon, W., Rea, C. and Arapostathis, S., 2022. Social pharmaceutical innovation and alternative forms of research, development and deployment for drugs for rare diseases. Orphanet Journal of Rare Diseases, 17, p.344.
Fermaglich, L.J. and Miller, K.L., 2023. A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act. Orphanet Journal of Rare Diseases, 18, article 163.
Haendel, M., Vasilevsky, N., Unni, D., Bologa, C., Harris, N.L., Rehm, H.L., Hamosh, A.,
U.S. Food and Drug Administration (FDA), n.d. Rare diseases at FDA. [Accessed 4 Aug. 2025].
Vavassori, S., Russell, S., Scotti, C. and Benvenuti, S., 2024. Unlocking the full potential of rare disease drug development: exploring the not-for-profit sector’s contributions to drug development and access. Frontiers in Pharmacology, 15, p.1441807.
Worldwide Clinical Trials, Rare Disease Clinical Development. [Accessed 6 Aug. 2025].
impossibly possible 