To mark Rare Disease Day we recently spoke with Jonathan Wadsley (Weston Park Hospital, Sheffield, UK) about recent advancements in care for medullary thyroid cancer.
Could you tell us what the current licensed treatment options for medullary thyroid cancer are?
One of the things that’s important to understand with medullary thyroid cancer is it can be a very indolent condition. Meaning that the cancer could have spread to the bones and the liver for example but it can still be quite a stable disease. Therefore, patients can live with this for many years without requiring any treatment. On the other hand, there is a population of patients who have more aggressive disease that is changing more rapidly and causing them more problems. This is the group that we now have new treatments for. There are two licensed drugs available for this group, they are called cabozantinib (Cometriq®) and vandetanib and they are specifically licensed for patients with advanced and progressive medullary thyroid cancer.
What events led up to Cometriq being recommended for reimbursement by the NHS?
A number of years ago an international trial was run, testing Cometriq in a population of patients with medullary thyroid cancer that was progressing. A number of centers in the UK, including Sheffield, took part in the trial. This trial demonstrated that taking the drug slowed down the progression of the medullary thyroid cancer when compared with patients who were taking placebo, so patients undoubtedly derived benefit from the trial treatment. Cometriq then went through the process of being approved by the regulatory agencies. It was approved by the European Medicines Agency a few of years ago.
The next step for Cometriq in the UK was for it to go through the NHS systems to gain approval to use the drug. So the drug has actually previously been available through the Cancer Drugs Fund, a government fund set up to fund very expensive anti-cancer medicines. However, the overall aim was always for it to be temporarily funded while NICE conducted an appraisal to decide whether it was a cost effective treatment that the NHS should be using. The recent development is that NICE have completed their appraisal and they have decided that this drug gives clinical benefit to patients and is cost effective so they’ve announced that it can be available for routine care for patients with this condition through the NHS.
Could you tell us about the key implication of this recommendation?
It means now patients with progressive medullary thyroid cancer will be able to, without question, access this drug and that will mean that they can access the benefits of the treatment in terms of slowing down progression of the disease, improving symptoms and we would hope – extend their life expectancy. So although there has been available there has been some anxiety over how long that will continue for, now we have the security of knowing that it is going to continue to be available.
Looking forward, what advancements would you like to see in treatment options for rare cancers?
We’re conscious we can always do better, so whilst this drug is very welcome it’s not a cure and unfortunately it is associated with some side effects. Additionally, whilst the treatment can improve the patient’s quality of life by shrinking disease and helping relieve pain, unfortunately some of the side effects can have an adverse effect on quality of life. Patients can feel unwell because of the side effects of the drug and as this drug is something that the patients continue indefinitely for as long as it’s working, patients can be taking it for numbers of months or years and then can be burdened with side effects. So I think the focus on further research really is looking for treatment that is a; more effective and b; more tolerable in the way of side effects.
Are there any other trials in the process at the moment?
Unfortunately we don’t have any clinical trials in medullary thyroid cancer that I’m aware of running in the UK at the moment, which is a disappointment, so we need to engage with the drug companies to look for compounds that may have efficacy and look to conduct more trials.
Dr Jonathan Wadsley is a Consultant Clinical Oncologist based at Weston Park Hospital, Sheffield. He has a specialist interest in the management of thyroid cancer and is a member of the NCRI Thyroid Cancer Clinical Studies Group.