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Payers' Views on the Heterogeneity of Treatment Effect in Oncology

When making coverage decisions in oncology, payers find difficulties in translating evidence on treatment effect heterogeneity into coverage policies.    
Published Online: Jun 22,2017
Abdalla Aly, PhD; David Yoder, PharmD, MBA; Fran├žoise Pradel, PhD; and C. Daniel Mullins, PhD
Objectives: The heterogeneity of treatment effect (HTE) refers to the nonrandom variability in response to treatment. This study describes how US payers use HTE evidence when formulating coverage policies for oncology drugs.
Study Design: We employed a qualitative approach using semistructured, in-depth interviews with 15 payers.
Methods: An interview guide was developed based on theory and pilot interviews. Themes that emerged from this content analysis were summarized along with verbatim quotes from payers.
Results: All payers agreed that HTE is important to incorporate into coverage policies of oncology treatments. The FDA label is the most overwhelming determinant of whether HTE evidence gets incorporated into a coverage policy. If not in the FDA label, payers find it difficult to use HTE evidence due to the inability to precisely differentiate responders from nonresponders and the logistical difficulty to operationalize HTE. All payers reported that randomized controlled trials are the most trusted source to establish HTE evidence. In addition to the FDA label, payers also consider treatment guidelines, the quality/magnitude of HTE evidence, the availability of effective alternative substitutes, treatment line, cancer aggressiveness, and politics. When a biomarker and a companion diagnostic are involved, the degree to which HTE evidence is incorporated into coverage policies will also depend on the clinical and analytic validity of the test and the ability to accurately and pragmatically distinguish responders from nonresponders.
Conclusions: Payers’ oncology coverage decisions are affected by a myriad of factors. Payers require more definitive HTE evidence to make more efficient coverage decisions.
Am J Pharm Benefits. 2017;9(3):-0

The FDA approval of costly anticancer medications that yield a modest incremental survival benefit has become more prevalent in the past few years, with the price range reaching $10,000 per month in 2013 compared with $4500 per month in 2003.1 Between October 2012 and October 2013, the FDA approved 9 anticancer drugs.

Although this significant number of new drug approvals in a 12-month period indicates increased drug development activity and improvements in regulatory processes (eg, accelerated approvals), most approved drugs were associated with high price tags and a modest survival benefit.2

Healthcare payers are facing great challenges when making coverage decisions, with uncertainty surrounding the value of oncology treatments specific to their plan members.3 Therefore, it is important that payers appropriately assess patient heterogeneity in response to treatment and use that information to inform coverage policies for oncology drugs.
Heterogeneity of treatment effect (HTE) refers to the nonrandom variability in the response to a treatment among different individuals given their characteristics. Kravitz et al define HTE as the “magnitude of the variation of individual treatment effects in the population”4; they further explain that, when HTE is present, the modest average effects realized in clinical trials often reflect a mixture of patients, where a group experiences the average effect, others experience less than or more than the average effect, and some may be harmed by treatment.4

Failing to account for HTE could mean that healthcare payers may initially be paying for treatments that are ineffective, and for additional downstream costs attributed to initially ineffective or harmful therapies.