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2026 Oxford-Harrington Rare Disease Scholar Award
February 28, 2026
Rare diseases are not actually that rare. Collectively they affect 500 million people around the world. Some, such as Huntington’s disease and sickle cell disease, affect many thousands, others only a handful. My darling son Ivan was one of only a few hundred people in the world with Ohtahara Syndrome, a rare neurological condition.
While they touch every area of medicine, these diseases have one thing in common: for a long time, they have been notoriously difficult to diagnose, let alone treat or cure. When Ivan was born in 2002, the care and help we received was remarkable. However, there was never really a comprehensive explanation for his condition. The “diagnosis” was more just a description of a set of symptoms including developmental delay, cerebral palsy and severe epilepsy.
In the years since, things have changed dramatically. The cost of sequencing one person’s genetic code, or genome, has fallen from $3bn to a few hundred dollars. A process that took a decade now takes one day. Doctors can now read a person’s complete DNA and, in a growing number of cases, can identify the precise genetic change responsible for a disease. Finally, families have been getting the answers they crave.
As prime minister I saw I could play a helpful role in embedding these advances in our national health system. We became one of the first countries to do just that when I launched the world-leading Genomics England in 2012 to sequence the genomes of 100,000 people. The company met that goal in 2018 and is now sequencing the genomes of 100,000 newborns, looking for 200 rare diseases. At the same time, the United States has invested heavily in genetic research and rare disease science.
The transformation of diagnostics has made the pharmaceutical industry take notice, particularly as the study of rare conditions begins to unlock insights into more common illnesses. Previously, the economics of manufacturing treatments for only a handful of people didn’t add up. Now, as experts realise rare disease treatments will accelerate treatments for mainstream conditions like heart disease and cancer, that financial circle is being squared.
David and Samantha Cameron with their children, from left, Elwen, Nancy and Ivan
Credit: Steffan Rousseau/PA
However, a specific challenge remains in getting these medicines to market: early-stage drug development programmes need to demonstrate a path toward commercial viability and use in patients to attract conventional investors. Here, the most promising science still stalls.
That is where the Oxford-Harrington Rare Disease Centre (OHC) comes in. This unique partnership between the University of Oxford and the Harrington Discovery Institute in Cleveland, Ohio brings together, for the first time, patients, academics, philanthropists, business leaders, investors and policymakers from the UK, US and Canada. It provides mentoring and resources at this pivotal, make-or-break stage to de-risk and commercialise potentially life-altering, life-saving work.
Eighteen months ago, when I joined the OHC’s advisory council as chair, I would have described the climate for rare disease breakthroughs as favourable. I believed this was the right cause, at the right time, in the right place. I was convinced by the collective advantages of these three life science superpowers: world-class research institutions; academic freedom; a history of breakthroughs; nimble licensing regimes; a culture of philanthropy; hungry investors; and Britain’s truly national health system.
But as we mark Rare Disease Day, I would argue that this favourable climate is fast becoming an optimal one. We are on the cusp of cracking many of these horrific diseases, and that is thanks to recent progress on several fronts.
The first is regulatory reform. In November, the UK medicines regulator announced its plans to adapt to the swift advances in medicine by changing the way new drugs for rare diseases are approved. Traditional clinical trial models are not always feasible for these conditions due to the small numbers of patients. The gap between diagnosis and treatment can take years. This new approach, also being considered in the US, focuses on approving processes rather than individual drugs, paving the way for a new era of personalised medicine.
The second advance is in commercial incentives, further squaring the financial circle. The US has recently renewed a system where companies developing a paediatric rare disease treatment are rewarded with a voucher to fast-track the development of one other drug. Meanwhile we’ve seen rare-disease companies grow in value and demonstrate commercial viability, particularly when there have been breakthroughs, such as in rare eye conditions. Amicus, which is transforming life for patients with debilitating enzyme deficiencies, was founded by OHC advisory council member John Crowley and recently acquired by BioMarin for nearly $5 billion.
The third advance is the momentum generated by this new transatlantic partnership. For the first time, the OHC has more than 50 drugs in development for conditions including rare brain and heart diseases, as well as rare cancers and childhood epilepsies, making significant headway towards our goal of 40 new drugs for rare diseases by 2034. Ten more of these researchers are joining the programme each year, maximising the chances of meeting that target.
Almost 17 years to the day since we lost our boy, at the age of only six, I feel emotional thinking about how rapidly the outlook is changing for patients with rare diseases like Ohtahara. Instead of hearing words like “management” and “care”, families receiving new diagnoses will hear words like “hope”, “treatment”, and even “long-term future”.
There has always seemed a deep unfairness in the fact that people are denied treatment, simply because their disease affects a few people and not many. Finally, the stars are aligning for that to change completely, with enormous potential to transform the treatment of mainstream diseases too.