Browsing by Author "Butani, Dimple"

Filter results by typing the first few letters
Now showing 1 - 2 of 2
  • Thumbnail Image
    ItemOpen Access
    Evaluating the Value for Money of Precision Medicine from Early Cycle to Market Access: a comprehensive review of approaches and challenges.
    (Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research, 2023-05) Chen, Wenjia; Wang, Yi; Zemlyanska, Yaroslava; Butani, Dimple; Wong, Nigel Chong Boon; Virabhak, Suchin; Matchar, David Bruce; Teerawattananon, Yot

    Objectives

    This study aimed to perform a comprehensive review of modelling approaches, methodological and policy challenges in the economic evaluation (EE) of precision medicine (PM) across clinical stages.

    Methods

    First, a systematic review was performed to assess the approaches of EEs in the past 10 years. Next, a targeted review of methodological papers was conducted for methodological and policy challenges in performing EEs of PM. All findings were synthesized into a structured framework that focused on Patient population, Intervention, Comparator, Outcome, Time, Equity and ethics, Adaptability and Modelling aspects, named the "PICOTEAM" framework. Finally, a stakeholder consultation was conducted to understand the major determinants of decision making in PM investment.

    Results

    In 39 methodological papers, we identified major challenges to the EE of PM, including that PM applications involve complex and evolving clinical decision space, clinical evidence is sparse due to small subgroups and complex pathways in PM settings, a one-time PM application may have lifetime or intergenerational impacts but long-term evidence is often unavailable, equity and ethics concerns are exceptional. In 275 EEs of PM, current approaches did not sufficiently capture the value of PM but that of targeted therapies, nor differentiate Early EEs from Conventional EEs. Finally, policy makers perceived the budget impact, cost-savings and cost-effectiveness of PM as the most important determinants in decision-making.

    Conclusions

    There is an urgent need to modify existing guidelines or develop new reference case that fits into the new healthcare paradigm of PM to guide decision making in R&D and market access.
  • Thumbnail Image
    ItemOpen Access
    Mapping the value for money of precision medicine: a systematic literature review and meta-analysis.
    (Frontiers in public health, 2023-01) Chen, Wenjia; Wong, Nigel Chong Boon; Wang, Yi; Zemlyanska, Yaroslava; Butani, Dimple; Virabhak, Suchin; Matchar, David Bruce; Prapinvanich, Thittaya; Teerawattananon, Yot

    Objective

    This study aimed to quantify heterogeneity in the value for money of precision medicine (PM) by application types across contexts and conditions and to quantify sources of heterogeneity to areas of particular promises or concerns as the field of PM moves forward.

    Methods

    A systemic search was performed in Embase, Medline, EconLit, and CRD databases for studies published between 2011 and 2021 on cost-effectiveness analysis (CEA) of PM interventions. Based on a willingness-to-pay threshold of one-time GDP per capita of each study country, the net monetary benefit (NMB) of PM was pooled using random-effects meta-analyses. Sources of heterogeneity and study biases were examined using random-effects meta-regressions, jackknife sensitivity analysis, and the biases in economic studies checklist.

    Results

    Among the 275 unique CEAs of PM, publicly sponsored studies found neither genetic testing nor gene therapy cost-effective in general, which was contradictory to studies funded by commercial entities and early stage evaluations. Evidence of PM being cost-effective was concentrated in a genetic test for screening, diagnosis, or as companion diagnostics (pooled NMBs, $48,152, $8,869, $5,693, p < 0.001), in the form of multigene panel testing (pooled NMBs = $31,026, p < 0.001), which only applied to a few disease areas such as cancer and high-income countries. Incremental effectiveness was an essential value driver for varied genetic tests but not gene therapy.

    Conclusion

    Precision medicine's value for money across application types and contexts was difficult to conclude from published studies, which might be subject to systematic bias. The conducting and reporting of CEA of PM should be locally based and standardized for meaningful comparisons.