The BIA Rare Disease Industry Group (RDIG) represents a group of innovative biopharmaceutical companies that specialise in treatments for rare and ultra-rare diseases.
RDIG is committed to developing recommendations that can pragmatically inform and improve the challenge of ensuring patient access to treatments for rare and ultra-rare conditions, sometimes referred to as orphan and ultra-orphan medicines.
What is a rare disease?
A rare disease is defined as one that affects less than 5 in 10,000 of the general population and an ultra-rare disease affects less than one patient per 50,000 of the European population. In the UK, it is estimated that 1 in 17 people – approximately 3.5 million people – may be affected by a rare disease at some point in their lives. The impact that rare diseases have on patients, their families, and society is profound, as many are severe, chronic and progressive, with high mortality rates.
Rare insights: examining the social values of treating rare diseases
Rare insights presents the findings of primary research into public opinion on how treatments for rare diseases should be evaluated and funded within the NHS. The findings signal the need for fresh research to determine the social values that should underpin the methods and processes used by NICE to make decisions about the NHS funding of treatments for rare diseases.
Evaluating patient access to rare disease treatments
This report, with research provided by global consultancy firm PwC, provides a comprehensive assessment of the progress that has been made in improving access to rare disease treatments in the UK, and the challenges that remain.
The report is based on extensive research and conversations with key stakeholders in the UK rare disease space, including NHS England, NICE, patient groups, clinicians, and industry.
There are around 7,000 known rare diseases. Despite this, only 5% of rare diseases have a single licensed treatment. As a result, many people living with rare diseases die prematurely or live with debilitating symptoms. This places huge burdens on caregivers, families, and society.
Rapid scientific advancements are enabling the life sciences sector to develop new life-changing and potentially curative treatments for rare and ultra-rare diseases (otherwise known as orphan and ultra-orphan medicines). Developing a drug for any condition is a long and expensive process, but for rare diseases, the situation is even more difficult.
Watch this animation to find out about the challenges of developing treatments for rare diseases.