Is Real-World Evidence Used in P&amp;T Monographs and Therapeutic Class Reviews?

Hurwitz, Jason T.; Brown, Mary Louise; Graff, Jennifer; Peters, Loretta; Malone, Daniel C.

doi:10.18553/jmcp.2017.16368

Cited by 19 publications

(21 citation statements)

References 35 publications

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“…While RCTs remain the gold standard for making coverage and reimbursement decisions, payers recognize the role high-quality RWE can play [7][8][9]81]. Payers are interested in real-world, long-term, comparative effectiveness and safety data from head-to-head comparisons with current standard of care, as well as RWD about prescription patterns and medication adherence, HCRU, costs and economic endpoints, PROs, and the identification of distinct patient subpopulations for personalized medicine [6][7][8][9]11].…”

Section: Discussionmentioning

confidence: 99%

Real-World Evidence: Bridging Gaps in Evidence to Guide Payer Decisions

Roberts

Ferguson

2020

PharmacoEconomics Open

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Randomized controlled trials (RCTs) are preferred by payers for health technology assessments and coverage decisions. However, the inclusion of a highly selective patient population and the rigorously controlled conditions in RCTs may not be reflective of real-world clinical practice. Real-world evidence (RWE) obtained from an analysis of real-world data (RWD) from observational studies can bridge gaps in evidence not addressed by RCTs and is thus valuable to public and private payers for decision-making. Through a broad literature search to obtain insights into payers' experience, we found that payers have concerns about real-world studies with respect to data quality, poor internal validity, potential bias, and lack of meaningful endpoints. However, they valued RWE to fill evidence gaps not addressed by RCTs, such as high-quality, real-world, long-term effectiveness and safety data; head-to-head drug comparisons; cost analyses for tiering formulary placement; medication use and adherence patterns; identification of relevant responder and non-responder patient subpopulations; and patient-reported outcomes (PROs). RWE can be used to assess clinically meaningful endpoints and gauge the impact of interventions on the quality of healthcare. Here, we review how payers use or can use RWD on the comparative effectiveness and safety of treatments, PROs, medication adherence and persistence, prescribing patterns, healthcare resource utilization, and patient characteristics and/or biomarkers associated with treatment response when making health technology assessments and payer coverage decisions across therapeutic areas.

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Section: Discussionmentioning

confidence: 99%

Real-World Evidence: Bridging Gaps in Evidence to Guide Payer Decisions

Roberts

Ferguson

2020

PharmacoEconomics Open

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“…Registries provide real-world information beyond what is captured in a randomized clinical trial setting [20, 21]. A total of 12,090 patients with psoriasis (with 48,870 patient-years of follow-up) have been enrolled in the PSOLAR registry [15] as of August 2015, making it the largest psoriasis registry to date.…”

Section: Discussionmentioning

confidence: 99%

Demography, baseline disease characteristics, and treatment history of psoriasis patients with self-reported psoriatic arthritis enrolled in the PSOLAR registry

et al. 2018

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Background To evaluate demographics, family history, and previous medication use at enrollment in a subset of psoriasis patients with self-reported psoriatic arthritis (PsA) enrolled in Psoriasis Longitudinal Assessment and Registry (PSOLAR). Methods PSOLAR is an international, prospective, longitudinal, disease-based registry that collects data in patients receiving, or are eligible to receive, systemic or biologic treatments for psoriasis. Baseline demographic, disease characteristics, medical history, and prior medication use at enrollment were evaluated in PSOLAR psoriasis patients self-reporting PsA ( n = 4315); a subset of which had their diagnosis of PsA established by a healthcare provider (HCP; n = 1719); patients with psoriasis only ( n = 7775); and the overall PSOLAR population ( n = 12,090). Results At enrollment, demographic characteristics were distinct between psoriasis patients self-reporting PsA and psoriasis only patients. Of the patients with psoriasis self-reporting PsA, 44.4% had cardiovascular disease (CVD), 26.3% had psychiatric illness, and 3.2% had inflammatory bowel disease (IBD), with each more prevalent than among patients with psoriasis only ( p < 0.001). Overall, 17.5% of psoriasis patients self-reporting PsA had a family history of PsA, 29.8% had used systemic steroids, 39.5% had used nonsteroidal anti-inflammatory drugs, and 83.5% had used biologics. Conclusions Demographics, family history, and previous medication use were generally comparable between “PsA established by a HCP” patients and psoriasis patients self-reporting PsA in the PSOLAR registry, but there were statistical differences compared with the psoriasis only group regarding the prevalence of certain comorbidities (CVD, psychiatric illness, and IBD). These analyses provide important data regarding characteristics of psoriasis patients with self-reported PsA in PSOLAR. Trial registration NCT00508547 . Electronic supplementary material The online version of this article (10.1186/s41927-018-0034-7) contains supplementary material, which is available to authorized users.

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“…Earlier work has revealed inconsistencies in how US payers cover medical technology, for example [10][11][12]. Further research to elucidate coverage criteria would be helpful [13][14][15][16].…”

Section: Discussionmentioning

confidence: 99%