How the patient voice is becoming vital for drug approval
Rebecca Sanders from Lipodystrophy UK tells us how the patient voice helped convince NICE to approve a much-needed drug for this rare disease, and explores how regulators and pharma companies can help make patient involvement in HTA more impactful.
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When England’s drug reimbursement watchdog NICE was on the fence about approving lipodystrophy treatment metreleptin, it was stories from patients that helped it get over the line and into routine use.
Rebecca Sanders, CEO of Lipodystrophy UK, the charity that spearheaded the patient involvement at NICE’s committee meetings for the drug, says that perspectives from people who live with the disease every day were essential for overcoming the shortcomings in the data.
“The numbers are never going to tell the whole story about quality of life,” she says. “There are so many other aspects of the condition that impact not just the patient themselves but their extended family, their carers and their children.
“Without the patient voice it’s difficult for researchers and regulators to understand what that all means. Stakeholders are well-meaning, but you need the personal touch in order to really get across the gravity of the situation.”
This was particularly important for metreleptin because lipodystrophy is a condition with many misunderstood social and mental health impacts on top of its severe medical effects.
Lipodystrophy is a rare disease with two different forms. Generalised lipodystrophy results in a total loss of subcutaneous fat cells. Partial lipodystrophy results in a partial loss, but the fat that remains is redistributed in inappropriate places around the body.
This can lead to metabolic abnormalities including insulin resistance, diabetes, and elevated levels of blood lipids (such as high cholesterol).
In addition, the loss or redistribution of fat can have major effects on people’s appearance.
Fighting for access
As an official charity Lipodystrophy UK has also been able to act as an expert voice in NICE appraisals for the lipodystrophy treatment metreleptin – where patient insights into the misunderstood aspects of the condition proved invaluable.
Before being approved, metreleptin had gone through two previous owners, and had been rejected for funding on the grounds of inadequate evidence in 2019. Eventually it was bought by Amryt who, keen to get the drug licensed, resubmitted it to NICE in 2020.
“One of the difficulties in getting licensing was that early on the drug had been given to patients on a named-patient basis and for compassionate reasons, rather than as a formal clinical trial,” says Sanders, noting that this issue is not uncommon in rare diseases.
“Unfortunately, that resulted in not having the organised, rigorous dataset that is usually required for NICE submission.”
Sanders has been on metreleptin treatment herself for over 10 years, and from the start has been involved in fighting for access to the drug, engaging with the pharma companies, clinical researchers, and NICE.
“NICE is good at encouraging conversations with patient stakeholders in these types of meetings, and we’ve been able to speak as a charity at all of the appraisal meetings for the drug. They’ve also made sure to reach out to patients who are not trustees of the charity,” she says.
NICE gave the patient community several opportunities to submit evidence.
As part of this, Lipodystrophy UK did a survey of the community and presented the data to the committee, while also highlighting individual statements and quotes.
Sanders says that these personal stories gave NICE a chance to understand the real impact of the treatment even when, on the surface, the data didn’t seem to show much efficacy.
“They definitely helped get the human story across. It’s so easy to concentrate on the numbers, the money, and the facts and figures, but that doesn’t tell you anything about the people and how a disease actually impacts them on a day-to-day basis.
“For example, if you say to someone you’re hungry all the time they often don’t understand what that really means because most people, certainly in the western world, have no idea what it’s like to feel truly hungry."
These patient testimonials helped push metreleptin over the line into approval, and the drug was finally approved for routine use on the NHS in January 2021.
There are plenty of rare disease drugs that aren’t so lucky, though, and Sanders says that in order for other treatments to avoid metreleptin’s early reimbursement difficulties more communication between every stakeholder at every stage is vital.
In particular, she says, many of metreleptin’s initial struggles came down to a lack of communication with patients from the earliest stages of development, which led to the wrong kinds of data being collected.
“The earlier HTAs, researchers and pharma companies can start that engagement the better.
“For example, the first committee meeting for metreleptin didn’t touch on how important the hunger aspect was, because nobody had that discussion with us in advance.
“After that meeting, we were able to tell the company how important it is to explain why metreleptin helps with that aspect, and how that impacts quality of life – but of course it would have been best to have that discussion upfront.”
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