Rare Disease Research
Rare Disease Research (RDR) is an independent clinical research site network operating four sites across Georgia, North Carolina, Florida, and New Jersey, dedicated exclusively to conducting clinical trials across 20+ rare disease indications for pharmaceutical and biotech sponsors.
- Company typePrivate
- Founded2021
- HeadquartersAtlanta, United States
- Headcount11–50
- GTM typeB2B
- OfferingServices
What Rare Disease Research does
Rare Disease Research (RDR) is an independent clinical research site network founded in 2021 and headquartered in Atlanta, Georgia, that operates clinical trial sites exclusively for rare disease indications. The company is organized as a privately held LLC with no disclosed external funding, and runs four physical sites across Georgia (Atlanta), North Carolina (Hillsborough), Florida (Kissimmee), and New Jersey (Iselin), collectively providing over 20,000 square feet of dedicated clinical research space. RDR positions itself as 100% dedicated to rare disease research with particular depth in neurological rare diseases, currently covering more than 20 indications including Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), Angelman syndrome, Friedreich's ataxia, galactosemia, Fragile X syndrome, metachromatic leukodystrophy (MLD), DEE, and DM1.
The company's core operating technology consists of purpose-built clinical research infrastructure rather than software. Site-level capabilities include a research compounding pharmacy with controlled mixing and refrigerated/room-temperature storage for investigational IV and oral products, a full-service laboratory equipped for cerebrospinal fluid sampling and tissue biopsies, a physical therapy center with a gait and partially-weighted ambulation lab, and diagnostic testing resources (electrocardiography, spirometry, X-ray). RDR claims the ability to initiate studies within 30-60 days. Patient acquisition occurs through direct physician referrals, listings on ClinicalTrials.gov, partnerships with patient advocacy organizations (including PPMD, CureDuchenne, MDA, NORD, FAST, Cure SMA, FARA, and Emily's Entourage), and a structured digital referral pathway via an April 2026 partnership with Netherlands-based myTomorrows.
RDR's business model is professional services for clinical trial conduct. Pharmaceutical and biotech sponsors — including AbbVie, Applied Therapeutics, Astellas, Biogen, Biohaven, BioMarin, Capricor, Dyne Therapeutics, Edgewise Therapeutics, Genentech, Harmony Biosciences, PTC Therapeutics, Sarepta, Solid Biosciences, Takeda, Ultragenyx, Wave Life Sciences, and Zynerba — contract RDR to conduct trial activities across the network. RDR does not bill patients; sponsors cover all study-related costs. The company also maintains channel-partner relationships with major CROs IQVIA and PPD (Thermo Fisher). Go-to-market is sales-led and event-driven, leveraging industry conferences and advocacy organization relationships. Revenue is not publicly disclosed.
Rare Disease Research firmographics
Firmographics- Name
- Rare Disease Research
- Legal name
- Rare Disease Research, LLC
- Website
- https://rarediseaseresearch.com
- Company type
- Private
- Founded year
- 2021
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Short description
- Rare Disease Research (RDR) is an independent clinical research site network operating four sites across Georgia, North Carolina, Florida, and New Jersey, dedicated exclusively to conducting clinical trials across 20+ rare disease indications for pharmaceutical and biotech sponsors.
- Ownership category
- akta.pro rank
Rare Disease Research industry classification
Industry- Product category
- Clinical Research Site Services
- NAICS
- Scientific Research and Development Services (5417), Medical and Diagnostic Laboratories (6215)
- SIC
- Services-Misc Health & Allied Services, Nec (8090)
- akta.pro primary industry
- Clinical Trial Site Operations & Study Coordination (HLAGACAE)
- akta.pro secondary industries
- Neurogenetics & Rare Neurologic Diseases (HLAKAIAO), Molecular & Genetic Testing (PCR/NGS/qPCR) (HLAGADAF), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)
Keywords
Where Rare Disease Research is headquartered
LocationHeadquarters
- HQ city
- Atlanta
- HQ country
- United States
- HQ region
- North America
Offices4 records
Markets served
Rare Disease Research business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Personnel, Operations, Infrastructure, Marketing or Sales, Technology or R&D, Supply Chain
Revenue model
- Clinical Trial Conduct Services: RDR conducts clinical trials for pharmaceutical and biotech sponsors. They receive compensation from study sponsors for conducting research. They do not bill patient participants for study-related assessments or treatment.
Pricing tiers
| Model | Billing | Price |
|---|---|---|
| Other | Other | Free participation for clinical trial enrollees |
Go-to-market motion2 records
Distribution channels3 records
Marketing channels5 records
Rare Disease Research product offering
Product offeringCore offering
Rare Disease Research (RDR) operates an independent clinical research site network 100% dedicated to conducting clinical trials for rare diseases, with a focus on neurological indications such as Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Angelman Syndrome, and Friedreich's Ataxia. The company provides sponsors with end-to-end site services — including a research compounding pharmacy, full-service laboratory, physical therapy center, and diagnostic testing — across four U.S. trial sites in Georgia, North Carolina, Florida, and New Jersey. Sponsors contract RDR to run studies, and patients participate at no cost while gaining access to investigational therapies long before potential FDA approval.
Product overview
Rare Disease Research (RDR) is an independent clinical research site network 100% dedicated to conducting clinical research for rare diseases. The company operates four clinical trial sites in Georgia (Atlanta), North Carolina (Hillsborough), Florida (Kissimmee), and New Jersey (Iselin), providing over 20,000 sq ft of dedicated clinical research space including research compounding pharmacy, laboratory facilities, testing resources, and a physical therapy center. RDR offers clinical trial services to pharmaceutical and biotech sponsors, connecting rare disease patients with access to innovative investigational therapies through structured referral pathways. The company does not operate a software product or platform but provides direct clinical research site services, patient intake via Google Forms, and partnerships with patient advocacy organizations to facilitate trial recruitment across more than 20 rare disease indications including Duchenne Muscular Dystrophy, Angelman Syndrome, Friedreich's Ataxia, Spinal Muscular Atrophy, and others.
Differentiator
Problem solved
Functional benefit
Products and services
- Rare Disease Clinical Trial Conduct Services End-to-end clinical trial site services for pharmaceutical and biotech sponsors developing rare disease therapies, conducted across four U.S. trial sites (GA, NC, FL, NJ). RDR recruits and enrolls patients, administers investigational products via its research compounding pharmacy, performs laboratory and diagnostic assessments, conducts physical therapy and gait evaluations, manages biospecimen handling and shipping, and oversees pharmacovigilance — all within a single integrated site network focused on more than 20 rare disease indications, particularly neurological disorders.
Quantifiable outcome
- Can initiate studies in 30-60 days
- +1 more outcomes
Companies that use Rare Disease Research
Customer profileNamed customers18 records
Segments4 records
Ideal customer profiles3 records
Rare Disease Research technology and API
TechnologyTechnology focussed No
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
Rare Disease Research partnerships and signals
Strategic signalPartnerships
Four partnerships are on record, tiered core and minor.
- myTomorrowscorePartnership to improve how rare disease patients are identified and referred into clinical trials through a structured referral pathway. The collaboration initially supports RDR's research sites in Georgia, Florida, and North Carolina, enabling referring physicians to use myTomorrows' platform to submit better-qualified referrals directly to RDR site teams. The partnership aims to reduce administrative burden, improve operational efficiency, and create a smoother experience for patients and families seeking access to rare disease studies.
- Rare Trials SummitminorAffiliate partner - event organizer bringing together key stakeholders in clinical research community
- IQVIAcoreSite partner - IQVIA is a leading global provider of clinical trial services and site network solutions
- PPD (Thermo Fisher)coreSite partner - PPD is a leading contract research organization providing clinical development services
Scale indicators3 records
Recent moves6 records
Expansion highlights4 records
Rare Disease Research competitors and assessment
Company assessmentBroad incumbents
- Medpace: Mid-to-large global CRO with significant rare disease and oncology exposure. Comparable to RDR's end-customers (biopharma sponsors) but operates at full-service CRO scale rather than dedicated site network.
- Syneos Health: Global biopharmaceutical solutions organization offering both CRO and site network (formerly Clinipace Worldwide). Comparable as a broad incumbent in clinical development services.
- IQVIA: World's largest CRO with integrated site network (including Site Lab, Q² Solutions). An RDR channel partner and the dominant incumbent in clinical trial services broadly.
- PPD (Thermo Fisher Scientific): Global CRO owned by Thermo Fisher Scientific with site network and clinical development services. An RDR site partner and a broad incumbent across all therapeutic areas including rare disease.
Direct peers
- Accel Research Sites: Multi-therapeutic clinical research site network operating across the US Southeast and beyond. Directly comparable to RDR in dedicated-site-network model and patient-recruitment focus.
- Velocity Clinical Research: Large multi-site clinical research site network operating across the US in Phase II-IV trials. Highly comparable to RDR in operating model (independent site network serving pharma/biotech sponsors) though broader in therapeutic scope and significantly larger scale.
- IMA Clinical Research: Independent clinical research site network with multi-state US operations. Comparable to RDR in dedicated-site-network business model and sponsor-contracted trial services.
- Cook Research: Rare disease-focused clinical research affiliate of Cook Children's Health Care System, with dedicated pediatric rare disease trial capabilities. Closely comparable to RDR's dedicated rare disease focus and pediatric neuromuscular specialty.
Emerging players
- Antidote Technologies: Digital patient-recruitment and clinical trial matching platform. Comparable to RDR's myTomorrows channel as an emerging patient-acquisition enabler for clinical trial sites and sponsors in rare disease.
- myTomorrows: Netherlands-based healthtech platform that connects patients and physicians to clinical trials, including in rare diseases. Directly comparable to RDR's myTomorrows partnership and serves as both a partner and adjacent player in patient-trial matching.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks7 records
Key highlights7 records
Customer concentration
Rare Disease Research social profiles
Digital presenceRare Disease Research financial estimates
Financial estimateRevenue estimate
Valuation estimate
Rare Disease Research leadership team
Management profileNumber of profiles
Profiles1 record
Rare Disease Research funding detail
Funding detailFunding overview
Funding rounds
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Rare Disease Research M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Rare Disease Research
What does Rare Disease Research do?
Rare Disease Research (RDR) operates an independent clinical research site network 100% dedicated to conducting clinical trials for rare diseases, with a focus on neurological indications such as Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Angelman Syndrome, and Friedreich's Ataxia. The company provides sponsors with end-to-end site services — including a research compounding pharmacy, full-service laboratory, physical therapy center, and diagnostic testing — across four U.S. trial sites in Georgia, North Carolina, Florida, and New Jersey. Sponsors contract RDR to run studies, and patients participate at no cost while gaining access to investigational therapies long before potential FDA approval.
Is Rare Disease Research a public or private company?
Rare Disease Research is a private company. It is classified as founder individual operated bootstrapped and is currently operating.
When was Rare Disease Research founded?
Rare Disease Research was founded in 2021. It employs 11 to 50 people.
Where is Rare Disease Research based?
Rare Disease Research is headquartered in Atlanta, United States, in the North America region.
How does Rare Disease Research make money?
One revenue line is on record: clinical Trial Conduct Services.
Who are Rare Disease Research's main competitors?
Broad incumbents on record are Medpace, Syneos Health, IQVIA and PPD (Thermo Fisher Scientific). Direct peers are Accel Research Sites, Velocity Clinical Research, IMA Clinical Research and Cook Research. Emerging players are Antidote Technologies and myTomorrows.
Does Rare Disease Research have an API?
No public API is recorded for Rare Disease Research.
What industry is Rare Disease Research in?
Rare Disease Research's product category is Clinical Research Site Services. Its primary akta.pro industry code is HLAGACAE, Clinical Trial Site Operations & Study Coordination, with a secondary code of HLAKAIAO, Neurogenetics & Rare Neurologic Diseases. Its NAICS code is 5417 and its SIC code is 8090.