Documenting the Full Value of Vaccination: A Systematic Review of Value Frameworks.

OBJECTIVES: Economic evaluations of vaccination may not fully account for nonhealth patient impacts on families, communities, and society (ie, broader value elements). Omission of broader value elements may reflect a lack of established measurement methodology, lack of agreement over which value elements to include in economic evaluations, and a lack of consensus on whether the value elements included should vary by vaccination type or condition. We conducted a systematic review of value frameworks to identify broader value elements and measurement guidance that may be useful for capturing the full value of vaccination. METHODS: We searched Ovid MEDLINE, PubMed, Embase, and the gray literature to identify value frameworks for all health interventions, and we extracted information on each framework's context, value elements, and any available guidance on how these elements should be measured. We used descriptive statistics to analyze and compare the prevalence of broader value elements in vaccination value frameworks and other healthcare-related value frameworks. RESULTS: Our search identified 62 value frameworks that met inclusion criteria, 9 of which were vaccination specific. Although vaccination frameworks included several broader value elements, such as reduced transmissibility and public health benefits, the elements were represented inconsistently across the frameworks. Vaccination frameworks omitted several value elements included in nonvaccination-specific frameworks, including dosing and administration complexity and affordability. In addition, guidance for measuring broader value elements was underdeveloped. CONCLUSIONS: Future efforts should further evaluate inclusion of broader value elements in economic evaluations of vaccination and develop standards for their subsequent measurement.

Barriers to engagement in the care cascade for tuberculosis disease in India: A systematic review of quantitative studies.

BACKGROUND: India accounts for about one-quarter of people contracting tuberculosis (TB) disease annually and nearly one-third of TB deaths globally. Many Indians do not navigate all care cascade stages to receive TB treatment and achieve recurrence-free survival. Guided by a population/exposure/comparison/outcomes (PECO) framework, we report findings of a systematic review to identify factors contributing to unfavorable outcomes across each care cascade gap for TB disease in India. METHODS AND FINDINGS: We defined care cascade gaps as comprising people with confirmed or presumptive TB who did not: start the TB diagnostic workup (Gap 1), complete the workup (Gap 2), start treatment (Gap 3), achieve treatment success (Gap 4), or achieve TB recurrence-free survival (Gap 5). Three systematic searches of PubMed, Embase, and Web of Science from January 1, 2000 to August 14, 2023 were conducted. We identified articles evaluating factors associated with unfavorable outcomes for each gap (reported as adjusted odds, relative risk, or hazard ratios) and, among people experiencing unfavorable outcomes, reasons for these outcomes (reported as proportions), with specific quality or risk of bias criteria for each gap. Findings were organized into person-, family-, and society-, or health system-related factors, using a social-ecological framework. Factors associated with unfavorable outcomes across multiple cascade stages included: male sex, older age, poverty-related factors, lower symptom severity or duration, undernutrition, alcohol use, smoking, and distrust of (or dissatisfaction with) health services. People previously treated for TB were more likely to seek care and engage in the diagnostic workup (Gaps 1 and 2) but more likely to suffer pretreatment loss to follow-up (Gap 3) and unfavorable treatment outcomes (Gap 4), especially those who were lost to follow-up during their prior treatment. For individual care cascade gaps, multiple studies highlighted lack of TB knowledge and structural barriers (e.g., transportation challenges) as contributing to lack of care-seeking for TB symptoms (Gap 1, 14 studies); lack of access to diagnostics (e.g., X-ray), non-identification of eligible people for testing, and failure of providers to communicate concern for TB as contributing to non-completion of the diagnostic workup (Gap 2, 17 studies); stigma, poor recording of patient contact information by providers, and early death from diagnostic delays as contributing to pretreatment loss to follow-up (Gap 3, 15 studies); and lack of TB knowledge, stigma, depression, and medication adverse effects as contributing to unfavorable treatment outcomes (Gap 4, 86 studies). Medication nonadherence contributed to unfavorable treatment outcomes (Gap 4) and TB recurrence (Gap 5, 14 studies). Limitations include lack of meta-analyses due to the heterogeneity of findings and limited generalizability to some Indian regions, given the country's diverse population. CONCLUSIONS: This systematic review illuminates common patterns of risk that shape outcomes for Indians with TB, while highlighting knowledge gaps-particularly regarding TB care for children or in the private sector-to guide future research. Findings may inform targeting of support services to people with TB who have higher risk of poor outcomes and inform multicomponent interventions to close gaps in the care cascade.

The impact of external reference pricing on pharmaceutical costs and market dynamics.

Growth in the cost of prescription drugs in the US has generated significant interest in the use of external reference pricing (ERP) to tie prices paid for drugs to those in other countries. We used data from the Pricentric ONE™ database, an international drug pricing database, to examine product launch timing, launch price, and price changes from January 2010 - October 2021 in both ERP and non-ERP settings, with a focus on 100 high-priced drugs of interest to Medicare and Medicaid. We found that ERP policies were associated with a 73% reduction in the likelihood of drug launch within 9 months of regulatory approval relative to non-ERP settings. In addition, while ERP was associated with statistically significant reductions in annual drug price changes, such policies did not impact launch price. In addition, no single ERP feature (e.g., number of countries referenced, ERP calculation) was materially associated with the outcomes of interest. We conclude that ERP policies do not appear to impact drug launch price and may delay access to new therapies, raising questions about the utility of such policies in the US and potential consequences abroad.

The Value of New: Consideration of Product Novelty in Health Technology Assessments of Pharmaceuticals.

BACKGROUND: Efforts to understand how treatments affect patients and society have broadened the criteria that health technology assessment (HTA) organizations apply to value assessments. We examined whether HTA agencies in eight countries consider treatment novelty in methods and deliberations. METHODS: We defined a novel pharmaceutical product to be one that offers a new approach to treatment (e.g., new mechanism of action), addresses an unmet need (e.g., targets a rare condition without effective treatments), or has a broader impact beyond what is typically measured in an HTA. We reviewed peer-reviewed publications and technical guidance materials from HTA organizations in Australia, Canada, England, France, The Netherlands, Norway, Sweden, and the United States (US). In addition, we explored how HTA organizations integrated novelty considerations into deliberations and recommendations related to two newer therapies-voretigene neparvovec for an inherited retinal disorder and ocrelizumab for multiple sclerosis. RESULTS: None of the HTA organizations acknowledge treatment novelty as an explicit value criterion in their assessments of pharmaceutical products. However, drugs that have novel characteristics are given special consideration, particularly when they address an unmet need. Several organizations document a willingness to expend more resources and accept greater evidence uncertainty for such treatments. Qualitative deliberations about the additional unquantified potential benefits of treatment may also influence HTA recommendations. CONCLUSION: Major HTA organizations do not recognize novelty as an explicit value criterion, although drugs with novel characteristics may receive special consideration. There is an opportunity for organizations to codify their approach to evaluating novelty in value assessment.

Patient and Caregiver Views on Measures of the Value of Health Interventions.

Purpose: We aimed to investigate patient and caregiver views on the relative importance of traditional and nontraditional domains of value, and to determine if these views differed according to key demographic characteristics. Patients and Methods: We conducted a modified Delphi approach using a web-based survey of adult patients managing a chronic condition or caregivers of a patient with chronic illness who were recruited using purposive sampling focused on demographic and clinical characteristics. The first survey round asked participants to rate the 13 domains of value on a 5-point Likert scale and rank each domain that they rated as important or very important. In the second survey round, participants reconsidered their own and aggregated round 1 results. New questions were added, including "rescuing" domains, challenges faced in taking medication, and a free-text option to add domains not already captured. Results: Initial recruitment resulted in 79 participants. Sixty-three (79.7%) completed the first round, and 58 participants completed both rounds. Overall ratings and rankings were consistent between survey rounds, and respondents ranked most highly domains considered traditional domains of value (for example, survival, costs). Patient activists were about six times more likely to rate each domain as important or very important compared to general disease advocates. Significant factors associated with a higher odds of rating a domain as important or very important were age 35-54 and 55-64 compared to 18-34, while factors associated with a decreased odds were males and patients compared to caregivers. Conclusion: Patients and caregivers place significant emphasis on traditional measures of value compared to nontraditional measures, and participants' prior beliefs impact what aspects of value they deem important.