Lifetime Economic Burden of Crohn’s Disease and Ulcerative Colitis by Age at Diagnosis
Clinical Gastroenterology and Hepatology
Gary R Lichtenstein, Ahva Shahabi, Seth A Seabury, Darius N Lakdawalla, Oliver Díaz Espinosa, Sarah Green, Michelle Brauer, Robert N Baldassano
2019
Understanding the burden of Crohn’s disease (CD) and ulcerative colitis (UC) is important for measuring treatment value. We estimated lifetime healthcare costs incurred by patients with CD or UC by age at diagnosis.
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Evaluation of Medical Technologies with Uncertain Benefits
NBER Working Paper No. 26058
Darius N. Lakdawalla, Charles E. Phelps
2019
Cost-effectiveness analysis (CEA) remains the de-facto method of choice to evaluate and compare medical interventions. Standard approaches to CEA use the average (mean) outcomes from clinical effectiveness studies such as randomized controlled trials. This paper generalizes standard methods to include uncertainty in clinical outcomes and proposes a generalized version of the quality-adjusted life-year (QALY), referred to as a quality- and risk-adjusted life-year (QRALY). Our approach requires new information from clinical studies – not only means and variances of health outcomes, but also skewness. With that added parameter, this paper shows how Taylor Series expansions of expected utility can account for two distinct effects of uncertainty: the “insurance value” of reducing overall risks to health, and the “value of hope” produced by the presence of positively skewed outcomes. Simulations demonstrate that stochastic terms are particularly important when baseline disease severity is high, and mean treatment effects are low. They also demonstrate that the variance-based term has the greatest importance among the stochastic terms, although skewness- and kurtosis-based terms can be significant in some situations.
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The Value of Progression-Free Survival in Metastatic Breast Cancer: Results From a Survey of Patients and Providers
MDM Policy & Practice
Joanna P. MacEwan, Jason Doctor, Karen Mulligan, Suepattra G. May, Katharine Batt, Christopher Zacker, Darius Lakdawalla, Dana Goldman
2019
Value assessments and treatment decision making typically focus on clinical endpoints, especially overall survival (OS). However, OS data are not always available, and surrogate markers may also have some value to patients. This study sought to estimate preferences for progression-free survival (PFS) relative to OS in metastatic breast cancer (mBC) among a diverse set of stakeholders—patients, oncologists, and oncology nurses—and estimate the value patients and providers place on other attributes of treatment. Methods. Utilizing a combined conjoint analysis and discrete choice experiment approach, we conducted an online prospective survey of mBC patients and oncology care providers who treat mBC patients across the United States. Results. A total of 299 mBC patients, 100 oncologists, and 99 oncology nurses completed the survey. Virtually all patients preferred health state sequences with contiguous periods of PFS, compared with approximately 85% and 75% of nurses and oncologists, respectively. On average, longer OS was significantly (P < 0.01) preferred by the majority (75%) patients, but only 15% of nurses preferred longer OS, and OS did not significantly affect oncologists’ preferred health state. However, in the context of a treatment decision, whether a treatment offered continuous periods of stable disease holding OS constant significantly affected nurses’ treatment choices. Patients and providers alike valued reductions in adverse event risk and evidence from high-quality randomized controlled clinical trials. Conclusions. The strong preference for observed PFS suggests more research is warranted to better understand the reasons for PFS having positive value to patients. The results also suggest a range of endpoints in clinical trials may have importance to patients.
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Understanding price growth in the market for targeted oncology therapies.
The American Journal of Managed Care
Sussell J, Vanderpuye-Orgle J, Vania D, Goertz HP, Lakdawalla D
2019
The causes of oncology drug price growth remain unclear. Analyzing corresponding trends in revenue can help understand these causes. This study seeks to assess changes over time in prices, patient counts, and drug-level revenues in the US market for oncology therapies and to investigate whether price growth is driven by an increased ability by pharmaceutical firms to capture profits.
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How Does Treating Chronic Hepatitis C Affect Individuals in Need of Organ Transplants in the United Kingdom?
Value in Health
Anupam Bapu Jena, Julia Thornton Snider, Oliver Diaz Espinosa, Andy Ingram, Yuri Sanchez Gonzalez, Darius Lakdawalla
2019
To estimate the impact of cures for chronic hepatitis C (CHC) infection on organ donation in the United Kingdom. Curing CHC infection reduces the need for liver transplants and enables cured individuals to donate organs of all types.
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