Clinigen launches scheme to support rare disease patients

Clinigen's Jerome Charton
Jérôme Charton/LinkedIn

Jérôme Charton, Clinigen's chief executive

Clinigen has launched a new programme in the UK that aims to help organisations representing patients with rare diseases participate in R&D for new medicines and support their members in accessing them once they become available.

The NaviGATE initiative is an education programme aimed at rare disease patient advocates and organisations that will provide tools to help them engage with healthcare providers, industry, and regulatory bodies more effectively, according to the pharma services specialist.

The programme is starting with a survey directed at the rare disease community that is trying to get a handle on their needs and the gaps that need to be addressed. It will be used to develop educational materials.

Patient-representing organisations often serve as the only source of medical information for rare disease patients and their families, but in some cases lack the expertise needed to navigate the complex pharmaceutical landscape, according to Clinigen.

Individual rare diseases may affect only a few people in the population, but collectively they represent a major public health issue, with a prevalence estimated at somewhere between 3.5% and 5.9%, according to IQVIA estimates.

The pharma industry is increasingly developing therapies for rare disorders – in part because they can command high prices and be in line for tax credits and other perks, as well as reduced development and regulator review times – but 95% of them still have no approved therapies.

Even when new treatments become available, there is no guarantee that they will be accessible to patients - particularly, for example, highly-priced cell and gene therapies, although, manufacturers and health systems are exploring outcome-based pricing and other models to improve affordability.

Clinigen says it has deep experience running early access programmes for patients, including for rare diseases, which makes it well-placed to help organisations advocate on behalf of themselves and their patients to make sure hard-to-reach medicines are available to those who need them.

The company recently backed away from its previous mixed business providing licensed and unlicensed pharmaceutical products, as well as services to focus entirely on the latter, after going into private hands when it was acquired for £1.3 billion by investment group Triton last year.

"I am thrilled to see the launch of the NaviGATE educational programme," commented Jérôme Charton, the company's chief executive. "Providing patients, families, and patient organisations with the tools to better engage with healthcare organisations represents a significant step forwards in our commitment to the millions of people affected by rare diseases," he added.