Clinicians and patient groups have called on parliament to support a PBS listing for the biologic dupilumab as a breakthrough treatment for atopic dermatitis.
And in response to their testimony at inquiry into allergies and anaphylaxis, MPs said they have written to the federal Minister for Health ahead of the Pharmaceutical Benefits Advisory Committee (PBAC) meeting in March that will re-consider a listing application for the dupilumab after two previous rejections.
Labor MP Dr Mike Freelander said he was moved to write to the minister after hearing the presentation at a hearing by allergist Dr Connie Katelaris, Head of Unit, Immunology and Allergy, Campbelltown Hospital, NSW.
Dr Katelaris told the Standing Committee on Health, Aged Care and Sport that her experience with the drug was that it was effective in about 80% of patients and transformational for young people with severe eczema whose lives were stunted by the huge adverse lifestyle and psychological effects of the condition.
“Having a visible skin disease has a huge impact on a child or an adolescent’s life— particularly for adolescents. These children are covered in scaly sores, they’re itching all the time, they can’t sit still, they’re scratching. This deflects from their ability to learn and concentrate. Most of them, in my experience, end up to be quite introverted. It’s hard to get them to look you in the face,” she said.
Children with eczema would not participate in sport and had difficulty making friendships, she added.
“It’s not a trivial disease. It doesn’t kill them, but it stunts them and it has a huge impact on the whole family. When you have a child or a teenager who is not sleeping and is roaming the house at night because they can’t sleep because of itching, that’s going to disturb the whole family. This is a family condition.”
Dr Katelaris said the trial data for dupilumab is “nothing less than astounding” and it has an good safety profile.
“While we have immunosuppressant agents that I and my colleagues have been dishing out for decades to these patients at this severe end, they come at a huge cost of side effects and potential toxicities. Dupilumab appears to be devoid of that”
While the drug costs around $1600 a month, Dr Katelaris said she had been able to arrange access for a dozen patients.
“It’s one of the happiest things I’ve ever done. These people walk in and say, ‘This is life changing.’ Two or three months down the track, you would never believe the photos that we took at the beginning. I am overwhelmed. I’ve seen the difference it has made.”
But she said drug was facing challenges to meet PBAC cost-effectiveness criteria to get a subsidy, even though the current subsidised treatments for severe eczema such as long-term corticosteroids came with the burden of side effects.
“These people … have been on drugs like cyclosporin, Imuran, methotrexate or mycophenolate. All these immunosuppressant drugs can work but they come at a huge cost—a possibility of cancers down the track, particularly skin cancers,” she said.
“My plea is that we have other models for considering these high-cost drugs for the high-end patient and high-cost drugs for rare diseases, because the health economic arguments that we use in the PBAC for the next cholesterol-lowering drug don’t fit these diseases.
Dr Freelander and the committee also heard from groups such as Eczema Support Australia about how patients and families faced high out of pocket costs they faced for managing severe eczema.
“I think we need to make people aware of how severe eczema can be and how life affecting it can be and what it can also do to families. There are better treatments available. So we need to really put the pressure on now to ramp up the availability of these newer medications,” he said.