Patient centricity is about delivering with, not for, patients

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.

Sue Farrington talks about patient centricityAs part of our Listening to the Patient Voice series, Sue Farrington, chief executive of Scleroderma and Raynaud’s UK (SRUK), told us 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.

About 12 new therapies for the rare condition, characterised by the body making too much collagen, are in the pipeline.

But securing access to these therapies for the 19,000 scleroderma patients in the UK is not expected to be plain sailing, said Sue.

Unmet need

“There is a real lack of effective, non-toxic treatments. It is an autoimmune condition, and a lot of the current treatments are pulled in from the cancer area, meaning they can be quite toxic,” she said, explaining that the condition caused a tightening of the skin that people described as “like turning into stone”.

The reason for this gap is one that’s familiar in many rare diseases – 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.

Barriers to access

Of course, work going on in other countries does hold the potential to help people in the UK, but a small patient population poses other barriers to access.

“It doesn’t mean these therapies don’t translate, but it does mean it will be a bit of a tortuous process when there is the possibility of them being made available,” said Sue.

“Because there are so few patients, there’s often not the volume of evidence the NHS wants, so it stalls there. We recognise that as the number of new treatments has grown, this can often create additional pressures on NHS finances.

“But this is a challenge that needs to be tackled working collaboratively across sectors, if we are to fully realise the ambitions of accelerated access to treatments for patients where there is no cure and the current treatments have damaging side effects.  We think the current appraisal process needs streamlining to enable timely, patient-focused and transparent commissioning of rare disease medicines.”

SRUK is not the only organisation to think this and 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.

“The wider picture is that 1 in 17 people will be affected by a rare disease at some point in their lives,” said Sue.

“People with rare diseases should not have a different experience of care or have less access to treatments, simply because their disease is rare. Patients in England aren’t afforded the same access to new rare disease treatments as other European countries.

“This fund would enable people to get access to these drugs and give a greater length of time to assess efficacy.”

Joint working

Collaboration is key to achieving so many aims in the road to better quality of life for people with rare diseases.

“If we are going to make real progress, we need to take a more collaborative approach across sectors and disciplines.

“That must include working with patient organisations, biotech and governments.”

Sue believes that involving patients from the very start of the development pathway is essential to progress.

She explained: “We hear about the challenges of getting patients involved in clinical trials and even more so getting them to adhere to medication.

“Asking what outcomes are important to them, what was it like to be diagnosed, how many tests, they had to have and how many times they have to go to the hospital: all these questions have to be asked, and, more importantly, listened to so that more patient centric therapies can be developed.

“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.

Wider perspectives

She added: “Companies are finding patients by working with clinicians, and that’s great, but there are huge benefits to working with patient organisations.

“Individual patients will have their own perspectives, but we have that real breadth of consumer voice.”

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.

Simply reaching out to patient groups such as SRUK could make a huge difference, she said.

If you work for an advocacy group and would like to take part in our Listening to the Patient Voice series, contact