UK must become leader in rare disease medicine, report says
The UK should look to become a European leader in rare disease medicine, a new report says.
The report, ‘Investing in Rarity: The Economic and Personal Value of Improving Treatment for Rare Disease’, shows that if the UK became the best-performing European country on access to rare disease medicines, benefits would be profound for patients, families, the NHS and the wider economy.
A study commissioned by the Association of the British Pharmaceutical Industry (ABPI) found that improving NHS access to innovative rare disease treatments could deliver up to £4.4 billion (€5 billion) in lifetime health benefits for people living with rare conditions and generate £19 billion (€22 billion) in productivity gains over 10 years.
“Many patients with a rare disease have no treatment options – the pipeline of medicines from pharmaceutical companies has the potential to change this,” said Victoria Jordan, Director of Value and Access Policy at ABPI.
“Improving access to rare disease medicines would transform thousands of lives, ease pressure on families, and deliver significant economic and NHS benefits. Just because these diseases are rare, the case for investing in new treatments must not be overlooked.
“We have an opportunity – and a responsibility – to remove barriers and ensure patients living with rare diseases can benefit from medical advances as soon as they are available.”
Rare disease medicines could reap economic reward
Although individual rare diseases affect fewer than one in 2,000 people, collectively they represent a major public health challenge.
Even where medicines do exist, only 35% of orphan medicines licensed in the EU are fully accessible to patients in England and Scotland, leaving the UK behind comparable European nations.
Rare diseases also carry a heavy economic burden. Figures show one in seven patients and carers has had to stop or reduce professional activity due to a rare disease, costing the UK £2.3 billion (€2.6 billion) each year.
People with rare diseases are almost three times more likely to be unemployed than the general population, and delayed diagnosis and limited treatment options are estimated to cost the UK economy £14.9 billion (€17 billion) annually.
However, if the UK can improve access to orphan medicines for patients while supporting continued innovation in the R&D pipeline, this could bring substantial benefits to patients, the economy and the healthcare system.