Database Study Design

Evidence need:

Recommended initial maintenance therapy for COPD include long-acting agents, such as LAMA/LABA or ICS/LABA dual therapy

Real-world evidence comparing the effectiveness of different single inhaler dual therapies, which would help inform treatment decisions, is limited in the UK with existing evidence restricted to randomised controlled trials

Solution:

Three comparative effectiveness research COPD studies using linked UK primary and secondary care data (CPRD-HES)

• Feasibility of comparative effectiveness research studies determined via a published 3-stage pre-study feasibility framework¹
• New-user, active comparator, retrospective cohort designs employing IPTW to adjust for measured confounders

Treatment comparisons and primary endpoints of:

• • UMEC/VI vs. TIO/OLO – Rescue medication use (prescriptions) over 12 months
  • UMEC/VI vs. IND/GLY – Rate of moderate-to-severe acute exacerbations of COPD (AECOPD) over 12 months
  • UMEC/VI vs. twice daily ICS/LABA – Medication adherence (as measured via the proportion of days covered [PDC]) over 12 months

Superiority and non-inferiority margins were determined based on clinical relevance and existing literature

Key findings:

UMEC/VI demonstrated superiority over TIO/OLO regarding 12-month rescue medication use

Non-inferiority of UMEC/VI vs. IND/GLY was demonstrated on moderate-to-severe AECOPD rate at 6, 12 and 18 months

UMEC/VI was shown to be superior to twice-daily ICS/LABA in medication adherence

Czira A, Requena G, Banks V et al. Comparative Effectiveness of Umeclidinium/Vilanterol versus Inhaled Corticosteroid/Long-Acting β2-Agonist in Patients with Chronic Obstructive Pulmonary Disease in a Primary Care Setting in England. nt J Chron Obstruct Pulmon Dis. 2023 Apr;18:643-659.

3x posters (# 2118, 2119, 3619) presented at: ERS; 2022 Sep 4-6; Milan, Italy

1 Requena G et al. Poster #159 presented at: ICPE; 2022 Aug 24-28; Copenhagen, Denmark

COPD – Chronic obstructive pulmonary disease; LAMA/LABA – Long-acting muscarinic antagonist/long-acting β2-agonist; ICS/LABA – Inhaled corticosteroid/long-acting β2-agonist; CPRD – Clinical Practice Research Datalink; HES – Hospital Episode Statistics; IPTW – Inverse probability of treatment weighting; UMEC/VI – Umeclidinium/vilanterol; TIO/OLO – Tiotropium/olodaterol; IND/GLY – Indacaterol/glycopyrronium

Evidence need:

There is a paucity of data on the economic burden of HF by LVEF; namely, for HFrEF and HFpEF patients

Real-world evidence on the HCRU and medical costs of HF management is critical to support the economic value propositions of the client’s asset to US payers

Solution:

A retrospective, longitudinal cohort study of a prevalent HF population in the US via IBM® Watson Health’s Limited Claims-EHR Dataset (MarketScan Commercial and Medicare Supplemental linked to Explorys)

Treatment group at index (HFrEF, HFpEF or HFuEF) was determined by most recently observed LVEF-specific diagnosis:

• Sensitivity analyses were conducted on treatment group assignment to account for conflicting LVEF-specific diagnoses

All-cause and HF-related HCRU and expenditures were reported by service type (inpatient, outpatient, and pharmaceutical), including for HF hospitalisations (hHFs) and urgent HF visits:

• • Urgent HF visits were defined as emergency department visits with HF as the primary diagnosis, but not constituting an hHF.
  • Definitions as per the CDISC were not feasible based on availability/ granularity of data within the study data

Key findings:

HFrEF and HFpEF present sizeable burden on the US healthcare system across a broad age range

Urgent HF visits are important clinical events representing a target for quality improvement

Accurate coding and LVEF-specific diagnosis of patients may also represent an opportunity for improvement in care quality

Lam CSP, Wood R, Vaduganathan M et al. Contemporary economic burden in a real-world heart failure population with Commercial and Medicare supplemental plans. Clin Cardiol. 2021; 44(5): 646-655

LVEF – Left ventricular ejection fraction; HFrEF – HF with reduced EF; HFpEF – HF with preserved EF; HCRU – Healthcare resource use; HFuEF – HF with unknown/ambiguous EF; hHF – HF hospitalisation; CDISC – Clinical Data Interchange Standards Consortium

Evidence need:

Novel pharmacological treatments have been developed for mUC which has a very poor prognosis

Current epidemiological trends and real-world treatment pathways in UC are largely unknown

Solution:

A retrospective, longitudinal cohort study using the HCEI/RWD Co. database (since acquired by JMDC) between Jan-2010 and Mar-2020

• Covers approx. 20 million patients from over 180 private and public medical institutions

Newly diagnosed la/mUC patients were identified via complex algorithmic classification, due to the lack of diagnostic coding for UC, and divided by index tumour site (bladder, ureter, renal pelvis or urethra):

• • Local coding systems (MEDIS-DC and YJ) were used to identify the patient population and treatments; often mapped to internationally recognized standard coding system codes (e.g. ICD-10 and ATC) to improve accuracy.

Endpoints included prevalence and incidence, treatment pathways and healthcare resource use

Key findings:

Age-adjusted incidence and period prevalence of la/mUC increased from 2015 to 2019, and estimates were high for bladder la/mUC compared with other la/mUC.

Use of GEM+CIT combination therapy decreased with age and advancing lines of therapy compared with GEM+CARB.

Yamamoto O A et al. Oral presentation at: JSCO; 2021 Oct 21-23; Yokohama, Japan
Manuscript in development

HCEI/RWD Co. – Health, Clinic, and Education Information Evaluation Institute/Real World Data Company; DPC – Diagnostic procedure combination; ICD-10 – International Classification of Diseases, Tenth Revision; ATC – Anatomical Therapeutic Chemical; HCRU – Healthcare resource use; GEM – Gemcitabine; CIS – Cisplatin; CARB – Carboplatin  

Evidence need:

Despite the importance of effective long-term treatment in UC, treatment changes are observed in substantial numbers of patients

Also a lack of comparative HCRU and cost data regarding the implications of incomplete response to available biologic treatment options – improved understanding of this relationship may help inform UC disease management strategies

Solution:

A retrospective, longitudinal cohort analysis of German statutory health insurance (SHI; Gesetzliche Krankenversicherung) claims data covering approx. 5 million patients and over 50% of German SHIs:

• Data analysis conducted in Germany to confirm with SHI regulations; regular/ close communication and quality control/ review of outputs required to ensure correct interpretation and execution of study protocol, respectively

New users of biologic therapy for UC divided by index treatment: adalimumab, golimumab, infliximab or vedolizumab

Endpoints included biologic therapy dose escalations, concomitant steroid and/or immunosuppressant use, and all-cause and UC-related HCRU and direct medical costs:

• Dose escalations defined as a daily dose increase of ≥50% compared with recommended maintenance dosing

Key findings:

Control with current biologics is suboptimal despite a number of available treatment options; with substantial dose escalations, concomitant medication use and HCRU observed

Novel treatments, or better utilisation of existing treatments, that provide sustained steroid-free remission without the need for dose escalations or concomitant therapies may be warranted

Dignass A, Waller J, Cappelleri JC et al. Living with ulcerative colitis in Germany: a retrospective analysis of dose escalation, concomitant treatment use and healthcare costs. J Med Econ. 2020 Apr;23(4):415-427.

HCRU – Healthcare resource use