Our recent collection of thought leadership features scientific publications from Precision authors Joanna MacEwan, Jacki Chou, Shannon Cope, and others!
Publication in Medical Decision Making Policy & Practice on how clinical trial endpoints in cancer are dominated by OS and PFS, with a widespread demand for OS. This study assessed patient and provider preferences for such endpoints in the context of breast cancer. Patients and providers alike valued reductions in adverse event risk and evidence from high quality randomized controlled clinical trials. In addition, whether a treatment offered continuous periods of stable disease holding OS constant significantly affected nurses’ treatment choices.
Citation: MacEwan JP, Doctor J, Mulligan K, May SG, Batt K, Zacker C, Lakdawalla D, Goldman D. The Value of Progression-Free Survival in Metastatic Breast Cancer: Results From a Survey of Patients and Providers. MDM Policy Pract. 2019 Jun 21;4(1):2381468319855386. doi: 10.1177/2381468319855386. eCollection 2019 Jan-Jun
Publication in BMC Health Services Research that quantified the short-term budget affordability of Hepatitis C treatments for three State Medicaid programs (in NC, OR and WI).
Publication in BMC Health Services Research that provides an example of how expert opinion can be elicited and synthesized with observed survival data using a transparent and formal procedure (using SHELF framework), capturing expert uncertainty, and ensuring projected long-term survival is clinically plausible. This will be increasingly important given early approvals of new oncology treatments and subsequent HTA evaluations in the absence of long-term follow-up.
Citation: Cope S, Ayers D, Zhang J, Batt K, Jansen JP. Integrating expert opinion with clinical trial data to extrapolate long-term survival: a case study of CAR-T therapy for children and young adults with relapsed or refractory acute lymphoblastic leukemia. BMC Med Res Methodol. 2019 Sep 2;19(1):182. doi: 10.1186/s12874-019-0823-8.
Publication in ScienceDirect that retrospectively assessed the predictive accuracy of six ICER-US budget impact analyses noting large differences between what was predicted vs. actual budget impact. ICER’s market uptake estimates exceeded the actual, real-world update by between 7-54 times yielding “large deviations between estimates and real-world consumption.”
Citation: Snider JT, Sussell J, Tebeka MG, Gonzalez A, Cohen JT, Neumann P. Challenges with Forecasting Budget Impact: A Case Study of Six ICER Reports. Value Health. 2019 Mar;22(3):332-339. doi: 10.1016/j.jval.2018.10.005. Epub 2018 Dec 14.