Below are two posters I am presenting at the 2019 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Annual Meeting. The first poster I presented today, the second poster below I will be presenting tomorrow, May 21, between 6-7pm CDT.
HOW MUCH DO INDIVIDUALS VALUE ACCESS TO INNOVATIVE LUNG CANCER THERAPIES? A STATED PREFERENCE SURVEY OF HEALTHY ADULTS IN THE UNITED STATES
OBJECTIVES: To measure the value healthy individuals place on coverage of novel therapies that improve survival in the event they develop lung cancer.
METHODS: A recent web-based survey was administered to US adults without cancer to measure the incremental insurance premium respondents were willing to pay to access lung cancer therapies that improve survival. The survey used a ‘multiple random staircase revealed preference’ method to elicit willingness to pay—in terms of monthly premiums—for coverage of novel therapies associated with survival gains. Survival was calibrated based on a clinical trial comparing nivolumab to standard-of-care chemotherapy among patients with non-small cell lung cancer.
RESULTS: Of 84,937 individuals invited via email, 1,191 responded and 163 completed the survey and met all eligibility criteria. The mean age of respondents was 50.0 (SD: 14.6) years, and 55.2% were female. On average, respondents were willing to pay an additional $57.00 (95% confidence interval (CI): $51.62-$62.37) monthly for coverage of novel therapy providing a 5-year survival of 15%, compared to standard-of-care therapy with a 5-year survival of 4%. Assuming risk neutrality, a lognormal survival distribution and annual lung cancer incidence of 0.07%, the mean survival gain conditional on being diagnosed with lung cancer is 1.95 years and the implied ex-ante value of a life-year under the expected utility framework is $500,066 (95% CI: $452,867-$547,177).
CONCLUSIONS: Individuals place a higher value on insurance coverage for lung cancer treatments than previously estimated. Potential explanations may include: high valuations of life year gains for lung cancer and/or large risk premium individuals place on an insurance reducing future mortality risks.
COMPARATIVE EFFICACY AND SAFETY OF CRIZANLIZUMAB FOR ADULTS WITH SICKLE-CELL DISEASE- A NETWORK META-ANALYSIS
OBJECTIVES : To compare the efficacy and safety of crizanlizumab (5mg/kg) with other treatments for sickle cell disease (SCD) among young adult and adult (≥16 years old) SCD patients not well managed by hydroxyurea via network meta-analysis.
METHODS : A systematic literature review of MEDLINE, Embase, CENTRAL, and clinical trials registries for studies on treatment efficacy in young adult and adult SCD patients identified 65 relevant full-text publications evaluating 49 studies. Networks of randomized controlled trial (RCT) evidence on treatments connected to crizanlizumab 5mg/kg identified for pain crisis, hospitalization, adverse events (AE), and serious adverse events (SAE). Hydroxyurea was not included as the study population was patients that were ineligible, failed or failing on hydroxyurea; transfusions were not included as only study identified was not placebo-controlled. The analysis combined treatment effects relative to placebo estimated by RCTs, thus balancing prognostic factors. Bayesian shared parameter model, using vague priors, combining relative treatment effects was used to analyze all studies simultaneously.
RESULTS : The pain crisis network consisted of 5 RCTs on 10 treatments, hospitalization days network consisted of 4 RCTs on 6 treatments, AE network consisted of 5 RCTs on 7 treatments, and SAE network of 5 RCTs on 8 treatments. Crizanlizumab was more efficacious than L-glutamine on pain crisis prevention (HR= 0.67, 95% credible interval (CrI)=0.50-0.88, Bayesian one-sided p-value 0.002). There was no or limited evidence of a difference between crizanlizumab and L-glutamine on hospitalization day, AE, or SAE rates based on Bayesian one-sided p-values. Results on other treatments (one or multiple doses each of N-acetylcysteine, prasugrel, senicapoc, ticagrelor, and transfusions) were too weak to draw conclusions.
CONCLUSIONS: This NMA provides preliminary data comparing crizanlizumab 5mg/kg and to other treatments—such as L-glutamine—in preventing pain crises in adults with SCD. It is difficult, however, to draw formal conclusions on these comparisons, given the remaining potential biases that could not be accounted for.