Market Access · Early Access
Access Before
Approval.
Compassionate use, expanded access, and named patient programmes designed, submitted, and managed to full regulatory compliance.
FullCompliance
Overview
Patient Reach, Regulatory Integrity
Early access programs pharma teams design must achieve two things simultaneously: getting therapy to patients who cannot wait, and maintaining the regulatory integrity of the formal approval programme running in parallel. A named patient program that generates adverse event data inconsistent with the clinical trial database, or a compassionate use programme that creates pricing precedents in unapproved markets, creates problems that outlast the programme itself. We design early access programmes that protect the sponsor's regulatory and commercial position while maximising patient reach. Every early access programme is a regulatory submission process, an operational logistics challenge, and a compliance monitoring commitment — all running concurrently.
- Compassionate use and expanded access
- Country-specific regulatory pathways
- Compliant patient tracking infrastructure
- Programme exit and pricing risk planning
What We Deliver
Capabilities Across Every Stage
Three integrated disciplines, delivered by senior specialists as one accountable programme.
Compassionate & Expanded Access
Compassionate use pharma programmes operate under distinct regulatory frameworks in each country. In Germany, named patient supply can proceed under existing regulatory provisions with minimal pre-authorisation; in France, the ATU/AAP framework requires ANSM submission and formal programme approval before supply begins. Expanded access programs in the USA are managed through FDA's IND framework, with distinct application pathways for individual patient, intermediate-size, and widespread access scenarios. Pre approval access drugs in the EU require navigation of national competent authority requirements that differ not just in process but in the evidence standards applied to the benefit-risk assessment. Access before approval pharma programmes in the UK operate under MHRA's unlicensed medicines framework with specific prescriber documentation requirements.
Regulatory Pathways
Compassionate use regulations across major markets share common principles — unmet medical need, no alternative treatment, acceptable benefit-risk — but differ significantly in how those principles are operationalised into submission requirements. Expanded access strategy must account for these differences before a programme is designed. Regulatory access programs are managed through our dedicated regulatory affairs team, which maintains current knowledge of compassionate use and expanded access requirements in all major markets. Global patient access programs require patient tracking infrastructure, prescriber management workflows, and adverse event reporting integrations that meet the compliance requirements of every country in which supply is made.
Patient Access Strategy
Patient access strategy pharma planning begins with a clear definition of the patient population, the countries in which early access is sought, and the timeline between current programme status and anticipated marketing approval. Early drug availability decisions must be made with full visibility of the regulatory, operational, and commercial implications — including the potential impact on pricing in markets where early access supply price can influence formal reimbursement negotiations. Our programmes are designed with programme exit in mind — transitioning patients to commercial supply at approval, maintaining regulatory compliance throughout. For sponsors requiring global early access programs USA EU UK across multiple markets simultaneously — compassionate use programs worldwide.
FDA·EMAMHRA · TGA
4Global Regions
ExitPlanning Built In
ISO 90012015 Quality System
Key Deliverables
What You Receive
Tangible, submission-ready outputs — not status updates.
Programme Design Document
Complete framework covering regulatory strategy, eligibility criteria, operational model, and governance.
Regulatory Correspondence
All submissions and agency communications required to establish and maintain programme — authorisation.
Patient Tracking Database
Compliant, audit-ready database of programme participants, prescriber records, and supply documentation.
Compliance Reports
Periodic regulatory reports documenting programme activity, adverse events, and patient outcomes.
Why Vigilare
Why Sponsors Choose Vigilare
What sets this engagement apart from a generic vendor.
Multi-Market Frameworks
Regulatory expertise in compassionate use and expanded access frameworks across the USA (FDA), EU member states, UK (MHRA), and Australia (TGA) — managed by a team with direct agency interaction experience in each jurisdiction.
AE Tracking Infrastructure
Patient tracking and adverse event reporting infrastructure established before first patient supply — not assembled reactively when a regulatory audit requests programme records.
Programme Exit Planning
Programme exit planning integrated from design stage — every early access programme is designed with the formal approval and commercial launch transition already mapped.
Pricing Risk Assessment
Pricing and reference pricing risk assessment before programme launch — identifying markets where early access supply price could create adverse precedents for formal reimbursement negotiations.
Who We Work With
Configured to Your Model
We engage differently depending on the function you already have in place.
Rare Disease Sponsors
Challenge
Small patient populations with urgent unmet need and no available treatment before approval
How We Help
Programmes reaching eligible patients through rare disease networks with full regulatory compliance maintained throughout.
Oncology Programme Teams
Challenge
Therapies showing strong early signal in populations with limited therapeutic alternatives
How We Help
Expanded access frameworks providing access without compromising the pivotal study timeline or data integrity.
Late-Stage Development Companies
Challenge
Submission timelines of 12–18 months with patients unable to wait for formal approval
How We Help
Bridging early access programme closing the gap between last study visit and first commercial supply.
FAQs
Early Access Programs, Answered.
01
What is a named patient program?
A named patient program allows a physician to request an unapproved medicine for a specific identified patient who has a serious or life-threatening condition for which no adequate authorised treatment is available. Supply is made outside of a clinical trial, under the sponsor's responsibility, and is subject to regulatory notification or approval requirements that vary by country. Named patient programmes require their own adverse event reporting, pharmacovigilance, and regulatory compliance infrastructure separate from the clinical trial programme.
02
How do early access programs work?
Early access programmes operate under country-specific regulatory frameworks that permit pre-approval supply of unapproved therapies for patients with serious unmet medical need. In the USA this is managed through FDA's expanded access IND framework; in the EU through national compassionate use and named patient supply provisions; in the UK through MHRA's unlicensed medicines framework. Sponsors must demonstrate an acceptable benefit-risk profile, maintain safety monitoring, and submit periodic programme reports to the relevant regulatory authority throughout the programme's operation.
Related Services
Complete the Access Strategy
Bundle this with the wider evidence and access programme — coordinated from one team.
Ready to enable early access?
Speak to our team about your programme requirements. We respond within one business day.