Health Technology Assessment (HTA) agencies limit patient access to drugs in two primary ways: by issuing “do not recommend” decisions and by recommending a drug for reimbursement in only a subset of the population approved in the market authorization. To determine market access strategy, manufacturers need to understand how agencies narrow the populations eligible for their drugs and also how to design trials to ensure evidence generation for each patient subgroup. Policy makers need to be aware of decision trends to determine if current HTA models are flexible enough to recognize the way values emerge over time and efficient enough to ensure all the “right” patients have access to the best treatments.
UK’s NICE and Restrictiveness
To better understand the challenges facing both manufactures and policy makers, we initiated a research initiative to examine trends in restrictiveness and see what the data shows. We focused on the UK’s National Institute for Healthcare and Excellence (NICE) because of their high level of activity and their influential presence in global HTA. Our research compared NICE’s reimbursement decisions to the European Medicines Agency (EMA) market authorizations, both overall and for oncology and non-oncology drugs. We had the privilege of presenting this research at ISPOR’s 17th Annual European Congress in Amsterdam.
Overall, our analysis showed that:
- NICE issued negative (“do not recommend”) decision in 32% of reviews (2007 to 2013)
- In that same period, NICE issued positive decisions in 68% of reviews
- Of the positive reviews, 52% included population restrictions on top of those included in the market authorizations (“recommend with restrictions”), effectively narrowing the population of patients eligible for reimbursement for the reviewed drugs
- Across all reviews with “recommend with restrictions” decisions, an average of 1.7 restrictions were added to the market authorizations
The most common added restrictions were for “contraindication or intolerance.”
Comparing Oncology vs. Non-oncology
We also separately evaluated oncology and non-oncology drugs. Oncology drugs were much more likely than non-oncology drugs to receive “do not recommend” decisions (56% vs. 16%). For drugs with a positive decision, there was no difference in the rates of “recommend” or “recommend with restrictions” decisions between oncology and non-oncology reviews. Over time, the rates of “do not recommend” decisions appear to increase for oncology reviews, but there does not appear to be any trends in “do not recommend” decisions for non-oncology. Interestingly, there was a significant decrease in the rates of “recommend with restrictions” decisions for oncology reviews, but no trend in non-oncology reviews.
Overall, NICE was more restrictive than the market authorization in 52% of positive reviews. NICE is limiting the population eligible for reimbursement in more than half of its positive decisions. NICE is even stricter with oncology reviews. Oncology reviews are more likely than non-oncology reviews to be issued a “do not recommend” decision. Even though there is no difference among the positive decisions in rates of “recommend with restrictions” decisions between oncology and non-oncology, there has been a decrease over time in the number of “recommend with restrictions” decisions for oncology drugs. Over time, it seems like NICE is replacing “recommend with restriction” decisions with “do not recommend” decisions for oncology reviews.
The large difference between the NICE decisions for oncology and non-oncology and the apparent replacement of “recommend with restriction” decisions with “do not recommend” decisions is interesting, especially in the context of the Cancer Drug Fund (CDF).
NICE appears to be substituting negative decisions for restricted recommendations. This conclusion receives support from independent research on a different sample of NICE oncology reviews conducted by researchers at the Health Economists’ Study Group. Their research found that restricted recommendations fell, and negative recommendations rose, following the establishment of the CDF in England. If you are not familiar with the CDF, we recently wrote, Curious About the Cancer Drug Fund?, explaining the CDF and how they fit into the larger oncology picture in England.
NICE and CDF: Things to Consider
Obviously NICE and oncology are hot topics that we will continue to follow, but in looking at the data at this point in time, there are a few questions we think are worth asking:
- Is NICE passing the buck on oncology drugs to the CDF?
- Is NICE’s current HTA model not the best method to evaluate oncology drugs?
- How will the new value-based assessment affect NICE decisions? Will NICE be more restrictive or less?
Click here to see the full presentation.