Patient centricity starts with communication and collaboration and these are the only ways to ensure people living with rare diseases like scleroderma will be able to access potentially transformative new therapies.
As part of their Listening to the Patient Voice series, Sue Farrington, PIF Chair and Chief Executive of Scleroderma and Raynaud’s UK (SRUK), told pharmaphorum how pharma and advocacy groups must work together if new hopes are to be realised.
After years of unmet need, new treatments that could transform the lives of people living with scleroderma are finally on the horizon. But securing access to these therapies for the 19,000 scleroderma patients in the UK is not expected to be plain sailing, said Sue.
The small patient population not only makes clinical trials difficult to design but means the finished drug can lack a large target market. “We need to think about what might encourage pharma to look at this, even though it’s probably not commercially viable. In the US and the EU, orphan drug legislation has been introduced to provide incentives. As a result, we’ve seen a growing interest in rare orphan diseases, but not as much in the UK. It is frustrating,” said Sue.
SRUK is working with the Specialised Healthcare Alliance and Genetic Alliance UK to develop a more “fit for purpose” process for appraising orphan drugs.
Part of that is a proposed Cancer Drugs Fund-style pot for new rare disease treatments which have been through clinical trials but do not have enough evidence for cost effectiveness.
Collaboration is key to achieving so many aims in the road to better quality of life for people with rare diseases. Sue believes that involving patients from the very start of the development pathway is essential to progress.
“If the therapy works, and taking it is easy, it will reduce the costs of health delivery which is a win/win. It’s about delivering with patients rather that delivering for patients.”
While there has been a trend towards pharma working more closely with individual patients, there was “real value” in enlisting patient organisations as well, said Sue.
She added: “Companies are finding patients by working with clinicians, and that’s great, but there are huge benefits to working with patient organisations.
Asked what the industry could be doing to help groups like SRUK address unmet patient needs, Sue said clearer paths of communication would help immeasurably.
“Companies are starting to listen to patients and are slowly starting to work with patient organisations, but we find it really challenging to find the right person to talk to,” she said, adding that while some companies did employ dedicated advocacy liaison executives, they had different job titles in each organisation making them hard to track down.
If you work for an advocacy group and would like to take part in their Listening to the Patient Voice series, contact email@example.com
Read the full article here: https://pharmaphorum.com/views-and-analysis/patient-centricity-delivering/