Implications and Use Cases for HTA Decision Predictive Models

Predictive modeling can be a powerful tool for understanding how multiple factors contribute to an event. As outlined in previous posts, Context Matters created a model that used variables from oncology assessments by the Scottish Medicines Consortium (SMC) to identify the most influential variables for a positive reimbursement decision by that agency and used the model to demonstrate the impact that economics (i.e., patient access schemes and ICERs) have in predicting positive SMC decisions for oncology drugs. However, as discussed in our previous post, Understanding Predictive Modeling, not all models are created equal. One measure of a predictive model’s quality is its ability to deliver actionable results and insights. If a model is not able to inform the initial hypothesis and provide a path forward, it is just an academic exercise. But what does it mean for a predictive model to be “useful” and to deliver “actionable results”? What are some of the implications and use cases for predictive models of Health Technology Assessment (HTA) agency decisions?

Key Drivers of HTA Decisions for "Life-Changing" Drugs

There are mechanisms in place to reward innovative and “life-changing” drugs. Most of these mechanisms exist in the regulatory process, but what about the reimbursement process? Are “life-changing” drugs rewarded by health technology assessment (HTA) agencies with positive reimbursement decisions? This original research applies a data-driven approach to evaluate the key drivers of HTA agency decisions for drugs that provided significant improvement in benefits over existing therapies.

Update on Recent Cancer Drugs Fund Changes

On March 16, 2016, the National Institute for Health and Care Excellence (NICE) approved some major changes to the UK’s Cancer Drugs Fund (CDF). The most significant of these changes is that NICE will now be evaluating all oncology drugs approved by the European Medicines Agency (EMA), including those previously funded through the CDF. Prior to this change, the CDF had independently conducted its own reviews. Now, the CDF will only serve as a funding source to be used at NICE’s discretion. This reorganization was precipitated by years of budget difficulties and numerous disagreements about the respective roles of NICE and the CDF.

NICE has started to release oncology reviews under its revised technology appraisal process, with many additional reviews in development and expected to be released in the next few months. In this post, we will discuss those reviews and what they may indicate about the future of oncology drug funding in the UK. 

Immunotherapies and the Race to Become the Standard of Care

A hot and promising new area of oncology treatment is immunotherapy, or IO drugs (which we discussed in a recent webinar). These therapies unlock immune system cells and help the immune system recognize and fight cancer tumors. To date, four IO drugs have been approved by the FDA: Yervoy (ipilimumab), Keytruda (pembrolizumab), Opdivo (nivolumab), and Tecentriq (atezolizumab).

Discussion on Drug Value Heats Up in US Election

Healthcare costs, specifically those related to prescription drugs, are a major election issue for many Americans. According to the August 2016 Kaiser Health Tracking Poll, two-thirds of Americans say Medicare access and healthcare affordability are top election issues, and 53 percent of voters say that prescription drug costs in particular are top priorities. Many of the efforts to address these issues concern the use of comparative efficacy and cost-effectiveness research. These forms of research, widely used integrated in the healthcare systems of many countries in Europe and elsewhere, are only recently gaining traction in the United States. Policy efforts and election outcomes could have a major impact on how these forms of research are used in the future.

Predictive Modeling: Understanding the Outliers

In predictive modeling, it is essential to evaluate how good the model fits the underlying data. The closer the model is to fitting the underlying data, the stronger the model. We recently built a model to predict Scottish Medicines Consortium (SMC) reimbursement decisions for oncology health technology assessments (HTAs). Our binary model, that is the model that attempted to predict a positive versus negative SMC reimbursement decision, was able to correctly predict 75% of the oncology decisions in the data set. By statistical standards, 75% is considered a “good” level of prediction. But what happened in the other 25% of assessments? Examining where the model “got it wrong” is an important tool in understanding the data and knowing how to apply the learnings and insights.

OPDIVO: The High Stakes of Comparative Efficacy

The Wall Street Journal recently reported that Bristol-Myers Squibb stock dropped dramatically on August 5th, 2016. This occurred after clinical trial results indicated that BMS’s immunotherapy drug OPDIVO® (nivolumab) failed to demonstrate a clinical improvement compared to chemotherapy in patients with newly-diagnosed lung cancer.

The financial market reaction to the clinical trial results highlights the growing importance of comparative efficacy research, both for obtaining approval and market access and, increasingly, for remaining competitive and driving profits. The impact of this announcement shows that the stakes are higher than just obtaining regulatory approval. Clinical trial results can have immediate and significant consequences before any regulatory decisions are even made. Here we discuss the importance of comparative efficacy research on market access, pricing, profitability, and competition.