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 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

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.

Real-world evidence (RWE) Capabilities at Arbor Research

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

Who We Are

What We Offer

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