ASCO 2017: Have Decisions by the National Institute for Health and Care Excellence Affected Outcomes of British Patients With Metastatic Castration-Resistant Prostate Cancer?

Chicago, IL ( John Graham gave an excellent talk regarding the role of NICE (National Institute for Health and Care Excellence) in the drug therapeutic approval process in the setting of metastatic castration-resistant prostate cancer (mCRPC). His summary of the role of NICE as it relates to the National Health Service (NHS) in England and the UK provides a nice contrast to the drug approval process here in the United States.

He started with a summary of the program itself and reviewed its history, role, and process of drug approval. NICE was set up in 1999 by Tony Blair’s Labour Government to reduce variations in availability and quality of care in the NHS, specifically in England. Other countries within the UK (Scotland, Wales, Northern Ireland) have their own independent systems, though they work with NICE.

While best known for its role in approving funding for new drug therapies, its role extends beyond that. While that is the only role for which it has jurisdiction to approve funding, it does serve in an advisory role for quality standard, clinical guidelines, public health guidelines and interventional procedure guidance. 

What makes NICE unique compared to some other national organizations (such as the FDA) is that they make decisions not just on clinical outcomes data, but also based on cost effectiveness. Specifically, they complete a “comparative analysis of alternative courses of action in terms of both their cost and consequences.” They look at the current treatment and planned new treatment, and assess the value of the health gain for the impacted patient group.

He briefly reviewed some concepts of how to measure health gain or loss. NICE specifically used the EuroQol 5 Dimension (EQ-5D) survey as an assessment of quality of life in response to treatment – while more generic than other surveys, as the assessments of NICE are quite broad, it serves the purpose well. They then utilize “Quality adjusted life years” or QALYs to compare the effectiveness of different therapies for different people. One QALY is defined as “one year of perfectly healthy life for one person.” One QALY can also be two years of 0.5 QoL for one person or one year of 0.5 QoL for two people. 

ICER, or incremental cost effectiveness ratio, then enables cost comparison, as it is a measure of cost for every QALY gained. NICE has previously published evidence that the theoretical max for ICER for a new therapy is 20-30,000 GBP/QALY. However, this is not set in stone. They have approved therapies with much higher ICER and rejected therapies with much lower ICERs. 

The actual technology/drug therapy appraisal involves multiple groups. There is a standing committee of health care professionals and patient/public representation. However, there is also an Evidence Review Board (ERB), made up of an independent group of economists, typically from a UK university. They also get input from expert advisors (patients and charity advisors) and very importantly put an onus on the manufacturer as well. 

He did go on to describe the Cancer Drug Fund (CDF), which was set up in 2010 by the Coalition government. Initially managed independently by 10 different regions in Great Britain, it exceeded its budget by 50% in 2015, and has now been taken over by NICE. It helps cover the costs of certain therapies that have not had enough evidence to get approval from NICE, but there is enough interest that additional data is needed. It helps bridge the gap between evaluation and approval. 

He put up a nice slide summarizing the timeline of submission, funding and approval of the different therapies for mCRPC. He reviewed specifically the timeline for enzalutamide to highlight the interval coverage of the CDF until the medication was approved by NICE. The involvement of the manufacturer in the process, specifically with the back and forth discussion with NICE, helps reduce costs on this taxpayer funded healthcare system. The initial ICER cost of enzalutamide from 40-50,000 GBP/QALY was brought down to <30,000 GBP/QALY during the approval process. 

While the median time to NICE approval from the time of submission is 15 months (range 9-60 months), the median time to CDF coverage is 1 month (median -4 to 15 months). CDF funding helps bridge the gap for coverage.

Prostate cancer mortality has fallen 10% between 2005 and 2014, similar to other countries. At a population level, it seems that this process has not limited availability of these novel therapies to the point that it has caused detriment outcomes to the population. At the same time, they have managed to keep healthcare costs down in this taxpayer funded system.

This system has significant cost benefits on a population level, and the United States can definitely learn from this process. 

Presented By: John Graham, MB ChB, FRCR, FRCP, Musgrove Park Hospital, Taunton, Somerset, United Kingdom

Written By: Thenappan Chandrasekar, MD, Clinical Fellow, University of Toronto, Toronto, Ontario, Canada
Twitter: @tchandra_uromd

at the 2017 ASCO Annual Meeting - June 2 - 6, 2017 - Chicago, Illinois, USA