Download as PDF

Legacy of Excellence in Real-World Evidence Generation at Arbor Research

Who We Are

Historical Leaders in Real-World Evidence (RWE)
Launched the multinational Dialysis Outcomes and Practice Patterns Study (DOPPS) in 1996

Dynamic and nimble non-profit with a rich track record of data management and using causal inference principles to reduce bias in real-world data analysis

Experts in study design, site identification, and investigator engagement, with contract research organization (CRO) capabilities

What We Offer

Support Throughout the Clinical Development Life Cycle
Support randomized controlled trial (RCT) design and planning

Conceive and lead evidence generation and real-world effectiveness studies

Data Analysis
Proficiency in data curation, linkage, and management as well as biostatistics and epidemiology

Data Solutions
Access to and expertise with large public and private databases with opportunities for logistical/CRO support for custom prospective data collection via our proprietary electronic data capture system or commercial eCRF

A unique global perspective to inform clinical practices, shape evidence-based guidelines and health policies worldwide, and ultimately improve patient outcomes

Data Generation and Access

Data Coordinating Center
  • 25+ years experience coordinating the international DOPPS program, a cohort of > 100K hemodialysis patients across 20 countries, as well as PDOPPS and CKDopps

  • CRO capabilities in diverse international settings

  • PARADIGM – a chronic kidney disease associated pruritus (CKD-aP) treatment registry to evaluate real-world outcomes of Difelikefalin in hemodialysis patients in Europe

Electronic Health Record (EHR) Extraction and Curation

  • Modernized cost-efficient alternative to large cohort studies

  • US dialysis: Long-standing relationships with multiple US dialysis providers and EHR data vendors with rich granular data on labs, medications, and clinical outcomes

  • US CKD: Innovative partnership with NANI, a large nephrology practice with a robust database of >45,000 patients across 75 CKD clinics

Public Data Sources

  • CMS claims: Includes Medicare-eligible in the US with and without CKD

  • United States Renal Data System (USRDS): 300,000+ US dialysis patients with Medicare coverage
  • Patient demographics, ICD-10 diagnoses, prescriptions, labs, hospitalizations, and other dialysis-related variables, and dialysis facility characteristics
  • With years of USRDS leadership, Arbor Research has a unique understanding of this database tailored to dialysis

Transforming data into evidence: RWE across the spectrum of drug development

  • Evidence generation: Create a compelling case for unmet needs
  • RCT planning: Leverage observational data to clarify decision points and optimize design
  • Post Authorization Safety Studies (PASS): Evaluate safety profiles for newly approved medications
  • Real world effectiveness /registry: Understand medication utilization and performance in everyday clinical practice

  • Monitoring trends: Stay up-to-date to understand management strategies and identify gaps in care

  • External control arm: Identify active comparators embedded in a larger cohort study

  • Policy research: Opportunities for natural experiments to understand impact on practices and outcomes

  • Patient-reported outcomes (PRO): Pioneers in developing and capturing PROs to better understand the patient perspective

Research Output

The Arbor Research RWE team has published hundreds of peer-reviewed manuscripts in scientific journals, including over 300 papers utilizing DOPPS, PDOPPS, and CKDopps data – many commissioned by industry partners.

Research deliverables can be defined flexibly and are dependent on the nature of the project, timeline of clinical development, and funder interests. Recent examples of output dissemination include:

  • Manuscript or research letter publications in peer-reviewed journals
  • Abstract publications and presentations at scientific conferences
  • Public dashboards (e.g., DOPPS Practice Monitor) or internal-facing reports with data and recommendations to support strategy
  • Curated analysis-ready databases
  • Document preparation for regulatory review

  • Virtual (webinars, podcasts) and face-to-face (conference symposia) presentations targeted to healthcare professionals, patients, policy makers, and researchers

DOPPs Logo
Arbor Research Logo

WHO WE ARE:

From the creators of DOPPS: A legacy of excellence in Real-World Evidence (RWE) generation.

Historical leaders in RWE Support throughout the clinical development lifecycle World-class expertise in applying analytics to kidney diseases and adjacent fields
  • Dating back to launch of the multinational Dialysis Outcomes and Practice Patterns Study (DOPPS) in 1996
  • Rich track record and robust capabilities for RWE in nephrology and adjacent areas
  • Study design, site identification and investigator engagement, logistical and CRO support
  • Complex analytics capabilities
  • Support clinical trial design and planning, evidence generation, post-approval and real-world effectiveness studies
  • Dynamic and nimble non-profit with strong reputation and credibility in nephrology and analytics
  • Solution-focused partnering with primarily industry clients
  • Expertise and reputation in:
    • End stage kidney disease (HD, PD)
    • Advanced NDD-CKD
    • Cardiovascular-reno-metabolic (CVRM)
    • Rare kidney diseases
  • Data curation, linkage, and management
  • Biostatistics and epidemiology
  • Using causal inference principles to address confounding and reduce bias in real-world data

DATA SOLUTIONS:

Access to and expertise with large public and private databases, with opportunities for logistical/CRO support for custom prospective data collection

DOPPS family of cohorts: Rich historical database Electronic health record (EHR) extraction and curation Facile with publicly available data: CMS claims and USRDS
  • Prospective cohort studies administered by Arbor Research, widely regarded as cornerstone research initiatives including over 200,000 patients across 25 countries
  • Offer a unique global perspective to inform nephrology clinical practices, shape evidence-based guidelines and health policies worldwide, and ultimately improve patient outcomes
  • DOPPS: In-center HD
  • PDOPPS: Peritoneal dialysis
  • CKDopps: Non-dialysis dependent stage 3-5 CKD under nephrology care, including a German EHR cohort of >65,000 patients
  • Modernized approach to RWE studies leveraging diverse EHRs
  • Cost efficient solution to Arbor Research and collaborators, with very large sample sizes
  • US dialysis EHR: Long-standing relationships with multiple US dialysis providers and vendors of EHR data with rich granular data on demographics, labs, medications, and clinical outcomes
  • US NDD-CKD EHR: Innovative partnership with NANI, a large nephrology practice with a robust database of >45,000 patients across 75 CKD clinics
  • Rich databases; over 300,000 US dialysis patients per year with Medicare FFS coverage
  • Patient demographics, ICD-10 diagnoses, prescriptions, labs, hospitalizations, and other dialysis-related variables
  • Team understands strengths and limitations, and can help identify the best data source to address the relevant objectives
  • CMS claims: Includes dialysis patients and Medicare-eligible patients with and without CKD
  • USRDS: With years of USRDS leadership, Arbor Research has a unique understanding of this database tailored to dialysis, including 2728 form and detailed information around the time of transition to kidney replacement therapy
DATA COORDINATING CENTER: If for any reason, RWE objectives cannot be achieved using any of the historical, contemporary, or secondary data sources above, Arbor Research is fully capable of designing, launching, and executing studies via primary data collection.

  • Nearly 3 decades of experience as coordinating center for the DOPPS
  • CRO capabilities in diverse international settings
  • Design and launch prospective studies in a cost-effective and efficient manner
  • Example: PARADIGM

TRANSFORMING DATA INTO EVIDENCE:

RWE across the spectrum of drug development

RCT planning
  • RCTs are gold standard for evidence, but can require enormous investment
  • Arbor Research can leverage observational data to help understand decision points and optimize design by supporting a projection of event rates in different scenarios, estimating screening failure rates and supporting retention strategies
  • Example: https://pubmed.ncbi.nlm.nih.gov/39719420/
Evidence generation
  • Real-world data can create a compelling case for unmet need, including disease burden, limitations of standard-of-care treatments, and associations with outcomes
  • Evidence can strengthen regulatory submissions, payer negotiations, and physician adoption, increasing access to innovative therapies and ultimate improving patient outcomes
  • Example: https://pubmed.ncbi.nlm.nih.gov/34221383/
Post-approval safety
  • Post Authorization Safety Studies (PASS) are crucial to comprehensively evaluate safety profiles for newly approved medications
  • Example: Arbor Research maintains ongoing work to carry out important PASS work, with readouts to follow [wait for Akebia approval to modify and share this bullet]
Real-world effectiveness
  • Real-world studies can complement RCT data to understand how treatment patterns and adherence may differ outside of a controlled trial setting
  • Understanding how medications are used in everyday clinical practice can offer insights to patients, healthcare providers, regulators, and policymakers
  • Example:https://pubmed.ncbi.nlm.nih.gov/36726438/
Data unification and alignment
  • In the case of rare disease, it can be particularly beneficially to unify international RCT and real-world clinical practice data into a single, consistently-coded coherent resource large enough to support investigations
  • Example: Arbor Research supports the Advancing Patient Evidence in X-Linked Hypophosphatemia (APEX) study, with data from North America, Europe, Israel, Japan, and Korea, and has submitted manuscripts for peer-review
Monitoring trends
  • With medical care continuously evolving, tracking contemporary trends in patient treatments and outcomes has many benefits, including highlighting gaps in care, identifying unmet need, and understanding management strategies abreast
  • Remaining abreast of these trends can help proactively adjust positioning and messaging strategies, inform potential areas for differentiation, and build a stronger value proposition for treatments
  • Example: <ahre=”https://www.dopps.org/DPM-HD/” target=”_blank”>https://www.dopps.org/DPM-HD/
Registry
  • Treatment registries are crucial to investigate a number of aspects of medication utilization that are often not possible in RCTs
  • These data can advance medical knowledge by revealing utilization patterns, safety, and effectiveness of newly approved medications, and can provide evidence for label changes and regulatory decision-making
  • Example: PARADIGM
External control arm
  • Findings from single-arm studies can be efficiently extended by identifying an appropriate control group embedded in a larger cohort study
  • Active comparators can help mitigate biases, especially when utilizing complex statistical methods such as inverse probability weighting or matching
  • Example: https://pubmed.ncbi.nlm.nih.gov/36411021/
Policy research
  • Since 1972, when Congress granted comprehensive coverage under Medicare to any patient diagnosed with kidney failure, dialysis has been covered for all patients with kidney failure in the US
  • Changes to CMS policy can thus have far-reaching consequences for dialysis patients, often providing opportunities for natural experiments to understand the impact on clinical practices and patient outcomes
  • Example: https://pubmed.ncbi.nlm.nih.gov/39378354/
Patient reported outcomes
  • PROs play a crucial role in understanding the real-world impact of diseases and treatments from the patient’s perspective, including subjective symptoms such as pain, fatigue, and itch
  • With an increasing emphasis on PROs by regulatory agencies, PRO-focused RWE can support drug approvals, label expansions, and reimbursement decisions
  • Example: https://pubmed.ncbi.nlm.nih.gov/33604539/
RESEARCH OUTPUT: The Arbor Research RWE team has published hundreds of peer-reviewed manuscripts in scientific journals, including over 300 papers utilizing data from the DOPPS. Deliverables can be defined flexibly, and will be dependent on the nature of the project, timeline of clinical development, and interests of the funder. Recent examples of output dissemination include:

  • Publication of manuscript or research letter in peer-reviewed journal
  • Abstract publication and presentation at scientific conference
  • Internal-facing report or dashboard, with data and recommendations to support strategy
  • Curated analysis-ready database
  • Preparation of documents for regulatory review
  • Virtual (web-based) symposia targeting nephrologists or other medical professionals

Contact us [link to a mailto: to SGT mailbox] to learn more about how Arbor Research can serve your RWE needs.