Director of Value and Access Policy, ABPI
A better system needs to be in place to get innovative new medicines for rare disease patients quickly.
The science behind bringing new transformative medicines to patients is a challenge. It is a real source of frustration that patients with some of the world’s rarest conditions have no satisfactory treatment.
Our industry works every day to try and improve this situation for patients and their families. Yet patients with the rarest conditions can face challenges at every turn, from receiving a correct and early diagnosis, accessing NHS services and receiving the appropriate treatment option.
New medicines on the way
But there is optimism, nearly 40% of the medicines now in development are for rare, or very rare conditions, including many cell and gene therapies which are personalised to individual patients.
These game changing therapies are often targeted for the youngest and most vulnerable children with debilitating genetic conditions.
We have CAR T-cell therapies in use in the NHS. These treatments allow us to modify the T-cells in patients’ blood to fight cancer cells in cases where all other treatments have been unsuccessful. This is helping us treat and sometimes cure patients with rare cancers.
Nearly 40% of the medicines now in development are for rare, or very rare conditions.
Improving the system
But more needs to be done. There are a number of ways that the government, NHS and pharmaceutical industry can work together to transform the lives of people with rare diseases.
We need the system to be truly joined up and fit for purpose and prevent undue delays in getting a diagnosis. When people are diagnosed and treatment is available, the system must work quickly to make sure patients receive it, regardless of where they live.
Working in collaboration
There are four positive collaborations between the NHS, government and industry that support earlier access to new medicines – the Early Access to Medicines Scheme, the Innovative Medicines Fund, Accelerated Access Collaborative and Innovative Licensing and Access Pathway which should all prioritise looking at medicines for rare and very rare conditions as well as those for more common conditions.
The journey of an approved medicine from scientific breakthrough to treating patients can be long and uncertain. We must not overlook the one in 17 people in the UK who will be impacted by a rare disease.