Patient view: Getting lung cancer patient voices heard in health technology appraisals
This article was originally published in Scrip
Lung cancer patients should have a louder voice in NICE health technology appraisals, say patient advocates as another new drug –Pfizer's Xalkori (crizotinib) – looks set to be rejected by the HTA.
Lorraine Dallas, director of information & support at the Roy Castle Lung Cancer Foundation isurging Pfizer and NICE to work together to discuss pricing and make the drug available to patients. The foundation described the targeted therapy as "vital" and said it was more effective than chemotherapy, had fewer side-effects, came in a more patient friendly pill format, and was supported by strong clinical evidence.
NICE, the HTA institute for England and Wales, issued final draft guidance on 16 August rejecting the drug looked for previously treated anaplastic-lymphoma-kinase positive advanced non-small-cell lung cancer patients (scripintelligence.com, 16 August 2013). The institute believed that the drug would not be a cost-effective use of NHS resources and had doubts about its overall survival benefits.
Ms Dallas has a different view. "We look at it from a patient's perspective. There is a lack of treatments available for late-stage lung cancer patients, and Xalkori is something that has good clinical evidence behind it," she said. The organization took part in the consultation after the preliminary no from NICE and is of the opinion that if there are patients that would benefit from the drug and the clinical evidence supports it, then those patients should get it. "From a patient perspective, it's about ensuring that people have access to the most appropriate clinical care, including medicines, and recognizing that although decisions have a cost issue and that the community that benefits may be small, they can have a major impact on individuals," she said.
But getting that view across could be easier and there is room for improvement for NICE to involve patients more fully in its decision making and consultations. However, Ms Dallas admits that the voices of lung cancer patients can be particularly difficult to hear. Many are diagnosed when the disease has reached a late stage, and there aren't enough survivors to talk positively about treatment and advocate for other patients, she said. Presented with a traumatic diagnosis and short-term prognosis, patients have to make sense of very complex treatment options. So understandably, becoming an advocate and an audible voice may not be a top priority.
Nevertheless, the tide is turning, and patients are becoming more active and adding their voices to those of patient organizations like the Roy Castle Foundation. Patients are becoming better educated and are asking more questions of their clinicians about why certain treatments might not be available. "It's part of the patient information and education process that patients are becoming more active. They are not just accepting decisions, but understanding why they are being made and participating in them." Use of national and international online forums for patients is also growing, with patients increasingly swapping stories on access to medicines, and this is another way in which they hold decision makers accountable, said Ms Dallas.
Ms Dallas concedes that while companies may have "robust reasons" for charging high prices, NICE also has a difficult job to do in balancing cost and clinical effectiveness, particularly for targeted therapies like Xalkori that are costly for the NHS and don't benefit a lot of patients. However, she says there needs to be a way of presenting the drug so that it is affordable, but it's up to NICE and the company to thrash out the solution.
There are mechanisms in place that have improved the situation, namely, the Cancer Drugs Fund, set up to address poor uptake of oncology medicines in England, and patient-access schemes that make the cost of treatment more affordable. (NICE rejected one such scheme from Pfizer that offered a confidential discount on the price of Xalkori). But what comes next is unclear as the Cancer Drugs Fund is only an interim measure until the UK's new pricing regime is up and running, supposedly from 1 January 2014.There is still no concrete information on what a new value-based pricing regime for new innovative drugs, and a replacement PPRS for drugs that don't come under VBP, might look like.
Excluding discounts, Xalkori costs £4,689 for one 60-count pack of 200mg or 250mg capsules. A course of treatment until disease progression costs between £37,512 and £46,890. After disease progression, a course of treatment would be £51,579. NICE put the incremental cost-effectiveness ratio for Xalkori compared to docetaxel at more than £100,000 per quality adjusted life year gained and £50,200 per QALY gained compared to best supportive care. The drug could benefit 550 patients.