Mission OAI Nexus Interviews Research About Us Launch OAI Nexus →
The intelligence layer for rare disease

Building the intelligence infrastructure for rare disease

The Orphan Access Initiative combines human expertise with AI to understand why promising treatments don't reach the patients who need them. Research remains at our core. OAI Nexus turns that research into accessible intelligence.

10,000+
Rare diseases identified globally
95%
Lack an FDA-approved treatment
300M
People affected worldwide
4
Intelligence systems inside OAI Nexus
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Mission

We advance rare disease access through research, AI, clinical intelligence, and healthcare economics, while keeping people at the center.

Why do promising treatments fail to reach the patients who need them? A treatment gets approved, the science works, and patients still can't get it. This question sits at the center of everything OAI does. We started because we kept seeing the same pattern, and the reasons are rarely simple.

Our research draws on real experiences from patients, caregivers, clinicians, and the industry professionals who shape what treatments get developed, approved, and covered. Technology amplifies that work. It never replaces the human judgment at its foundation.

Clinical Lens

Disease biology, therapeutic development, clinical trial design, and the scientific obstacles that slow progress from the lab to the patient.

Financial & Policy Lens

Drug economics, pricing decisions, reimbursement systems, payer behavior, and the commercial realities that determine who gains access.

Integrated Analysis

Most research looks at these sides separately. We think the interesting questions live at the intersection. That is where we focus.

The Treatment Gap

More than 10,000 rare diseases are known. Roughly one in seven has a company or a product behind it.

This is what our own database returns. Nexus Intelligence tracks 11,645 rare diseases along with the companies, products, orphan designations, and trials attached to each one. For most of the list, that query comes back close to empty. The gap is not a shortage of science. It is a shortage of reasons to fund it.

Source: OAI Nexus Intelligence
Built from Orphanet, FDA Orphan Drug Designations, Drugs@FDA, ClinicalTrials.gov

Nexus Intelligence · Field Monitor --:--:--
LEAD II
RESP
62BPM
0Rare diseases tracked
0With a company or product
0FDA orphan designations
0Clinical trials indexed
◆ Flagship Platform

OAI Nexus turns rare disease research into accessible intelligence

One platform, four intelligence systems. Nexus makes the knowledge buried across literature, trials, economics, and lived patient experience searchable, connected, and usable by the people who need it, now more than 10,000 of them.

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Nexus Intelligence

The rare disease research database

The structured foundation of Nexus. Search every rare disease, every company working on one, and every product in development. Sourced from FDA orphan designations, ClinicalTrials.gov, and Orphanet, and built to be usable by anyone making decisions about rare disease treatment.

SearchExploreAsk NexusSourced Data
Launch Nexus Intelligence
11,645
Rare diseases
7,851
Companies
23,883
Products
73,724
Clinical trials

Research remains at our core. OAI Nexus is how that research reaches further, connecting patients, clinicians, and the people who fund and build treatments across one shared intelligence layer.

Explore the platform →
Interview Series

Voices from the field

We talk with patients, caregivers, physicians, researchers, biotech leaders, and investors about what it actually takes to get a rare disease treatment from development to the patient who needs it.

Genomics Leader
August 13, 2026

Interview with Dr. Heidi Rehm

Chief Genomics Officer, Mass General Hospital; Co-Director, Broad Institute

We talk with Dr. Rehm about diagnosing the hardest rare-disease cases, why patients with the same genetic variant can look so different clinically, and how initiatives like the Rare Genomes Project and ClinGen depend on data sharing to move diagnosis forward. We also cover the economics of sequencing ultra-rare conditions and where AI can genuinely improve genomic interpretation.

Genomic MedicineRare Disease DiagnosisClinGen
Biotech Leader
August 6, 2026

Interview with Dr. Viraj Mane

Co-Founder & Chief Scientific Officer, Lactiga

We talk with Dr. Mane about building a therapy from the antibodies in donated human milk, why mucosal immunity has been overlooked next to conventional IgG treatments, and what it takes to finance a clinical-stage biotech through NIH grants, FDA rare disease designations, and venture capital.

Secretory IgAPrimary ImmunodeficiencyBiotech Financing
Researcher
June 19, 2026

Interview with Dr. Alicia Campbell

Rare Disease Researcher

We talk with Dr. Campbell about rare disease research, access, and the barriers patients face in getting treatment, drawing on her experience researching MED13L syndrome and other ultra-rare conditions.

MED13L SyndromeResearchAccess Barriers
Interview
Coming soon
Interview
Coming soon
Interview
Coming soon

We are always looking for people willing to share their perspective on rare disease access. If you are a patient, caregiver, clinician, researcher, or industry professional, we would love to talk.

Get in Touch →
Research

Access Reports

Each Access Report traces a single rare disease from scientific discovery through to patient access, examining clinical, regulatory, commercial, and policy dimensions in full. Nexus builds on this foundation.

First Report
April 5, 2026

Fibrodysplasia Ossificans Progressiva

Access Barriers in the World's Rarest Disease: FOP and the Road to Treatment

Bone FormationGene TherapyUltra-RarePalovarotene
Second Report
2026

Spinal Muscular Atrophy

From Discovery to $2.1M: The Economics of SMA Treatment Access

Gene TherapyPayer DynamicsNewborn Screening
Third Report
2026

CDKL5 Deficiency Disorder

A Clinical, Economic, and Patient Access Analysis with Emerging Perspectives on Artificial Intelligence

Epileptic EncephalopathyGene TherapyHealth EconomicsArtificial Intelligence
Fourth Report
2026

Sanfilippo Syndrome Type A

The first approved therapy for MPS IIIA, and whether children can reach it in time. A clinical, economic, and patient access analysis of UX111.

Lysosomal StorageGene TherapyUX111 / rebisufligeneAccess Economics
Future Report
In Development

Next Report

Topic currently in development. Follow OAI on LinkedIn for updates on upcoming research and disease selections.

About Us

The team behind OAI

OAI was founded by two students who believe that understanding rare disease access requires both scientific fluency and systems thinking.

Aarush Shah
Co-Founder · Financial Lead · AI Development
Aarush Shah
Financial & Access Analysis · AI Systems

Aarush Shah co-founded the Orphan Access Initiative and shapes its research agenda, working where healthcare meets capital. His focus is the economic machinery that decides which rare disease treatments get built, and who can afford them once they exist. He leads the financial and access track, modeling R&D costs, reimbursement constraints, and the funding structures that determine whether a therapy ever reaches a patient. He also designed and built the AI systems that run OAI Nexus.

He is the founder of ShahTrades, a biotech and equity research platform with over 1 million views, where he runs the Financial Oncology Research Lab. He also serves as CEO and Head of Healthcare Equity Research at Advanced Equities, and has worked in strategic finance across oncology and asset management.

LinkedIn →
Veer Chandwani
Co-Founder · Clinical Lead
Veer Chandwani
Clinical & Scientific Analysis

Veer Chandwani is a student researcher focused on the clinical barriers that limit treatment access in ultra-rare disease. As Co-Founder, he leads OAI's clinical research track, analyzing disease biology, trial design constraints, and the regulatory challenges that slow development from the lab toward patients.

Through physician interviews, literature review, and trial analysis, he examines how therapies actually move from lab to patient. His work identifies where clinical development becomes constrained, contributing the scientific grounding behind OAI's research.

LinkedIn →
The OAI founders presenting Nexus Intelligence
The founders with Nexus IntelligenceOrphan Access Initiative
Student-Led

OAI was founded by students and operates with the rigor, curiosity, and independence that serious academic research demands.

Independent

Our research is not funded by industry, payers, or advocacy organizations. We follow evidence, not interests.

Open Access

All Access Reports, educational content, and interview transcripts are freely available to the rare disease community.