Assess whether a rare disease programme meets orphan drug designation criteria in the relevant jurisdiction.
Healthcare & Pharmaceutical Management
Orphan Drug Development and Rare Disease Market Access
Guide rare disease programmes from orphan drug designation and small-population trial design through to health technology assessment and managed entry agreements that secure patient access.
🗓️Duration5 training days
📚Content4 modules · 8 sessions
🎓On completionAccredited attendance certificate
About the programme
Course Overview
Rare disease programmes carry a distinctive set of problems: too few patients for a conventional trial, natural history that is barely documented, and payers who must judge value with limited comparative evidence. This course addresses each problem in sequence. Participants start with orphan drug designation criteria and rare disease epidemiology, then design small-population clinical trials that use crossover, adaptive or single-arm designs where a traditional randomised trial is not feasible. The programme covers natural history studies and surrogate endpoints as tools for building an evidence base before pivotal data exists, and the accelerated approval and conditional marketing authorisation routes that let a promising therapy reach patients earlier under continued evidence commitments. Later sessions turn to patient registries and real-world evidence, partnership with patient advocacy groups in trial design and evidence generation, and the health technology assessment and managed entry agreements that determine whether an ultra-orphan product reaches formulary. Participants leave with a development and access plan built around a single rare disease case.
Expected Learning Outcomes
Design a small-population clinical trial using crossover, adaptive or single-arm methods where appropriate.
Build a natural history study that documents disease progression ahead of pivotal trial data.
Select surrogate endpoints capable of supporting an accelerated or conditional approval submission.
Establish a patient registry that generates real-world evidence to support post-approval commitments.
Plan collaboration with patient advocacy groups on trial design and outcome selection.
Structure a managed entry agreement that addresses payer uncertainty about an ultra-orphan product's value.
Who Should Attend
Clinical development leads planning trials for therapies in rare and ultra-rare diseases.
Regulatory affairs professionals pursuing orphan drug designation and accelerated approval.
Market access managers preparing health technology assessment submissions for orphan products.
Medical affairs staff building patient registries and real-world evidence programmes.
Patient advocacy liaisons coordinating input into rare disease development plans.
Health economists structuring managed entry and outcomes-based agreements for payers.
Course Modules
Select any module to see its sessions and points.
01Orphan Drug Designation and Development Strategy
2 sessions · 8 points
Session 1Rare Disease Epidemiology and Designation Criteria
- Assess prevalence and unmet-need data against the designation criteria used by major regulators.
- Map the natural history and disease subtypes relevant to a specific rare disease programme.
- Identify orphan drug exclusivity and incentive provisions relevant to a development timeline.
- Build a target product profile that reflects realistic rare disease trial feasibility.
Session 2Small-Population Clinical Trial Design
- Select a trial design, such as crossover, adaptive or single-arm, suited to a small patient population.
- Justify a statistical approach that accounts for limited sample size and patient heterogeneity.
- Plan patient recruitment across multiple countries to reach a viable trial population.
- Design a protocol that minimises patient burden while preserving scientific rigour.
02Regulatory Pathways for Accelerated and Conditional Approval
2 sessions · 8 points
Session 1Natural History Studies and Surrogate Endpoints
- Design a natural history study protocol that captures clinically meaningful disease milestones.
- Select surrogate endpoints with a plausible link to long-term clinical outcomes.
- Use natural history data as an external comparator where a concurrent control arm is not feasible.
- Plan the evidence needed to validate a surrogate endpoint to a regulator's satisfaction.
Session 2Accelerated Approval and Conditional Marketing Authorisation Routes
- Compare accelerated approval, conditional marketing authorisation and other expedited pathways.
- Plan the confirmatory evidence commitments that follow a conditional or accelerated approval.
- Prepare a briefing document requesting early regulatory dialogue on an expedited pathway.
- Assess the risk of withdrawal if confirmatory studies fail to verify the expected benefit.
03Patient and Advocacy Engagement in Rare Disease Development
2 sessions · 8 points
Session 1Patient Registries and Real-World Evidence Generation
- Design a patient registry data model that captures outcomes relevant to regulators and payers.
- Plan data governance and consent arrangements for a multi-country rare disease registry.
- Use registry data to support post-approval safety and effectiveness commitments.
- Combine registry evidence with trial data to strengthen a product's long-term value story.
Session 2Partnering with Patient Advocacy Groups
- Involve patient advocacy groups in selecting outcomes that matter most to patients and families.
- Plan a patient-reported outcome strategy that captures quality-of-life data in the trial.
- Build a communication plan that keeps advocacy groups informed through development milestones.
- Address the practical and ethical considerations of patient involvement in protocol design.
04Rare Disease Market Access and Pricing
2 sessions · 8 points
Session 1Health Technology Assessment for Ultra-Orphan Products
- Prepare a health technology assessment submission that addresses limited comparative evidence.
- Present cost-effectiveness analysis appropriate to small populations and high uncertainty.
- Address committee concerns about extrapolating short-term data to lifetime outcomes.
- Build a value story that reflects disease severity and absence of alternative treatments.
Session 2Managed Entry Agreements and Outcomes-Based Pricing
- Structure a managed entry agreement that ties payment to real-world treatment outcomes.
- Negotiate data collection commitments that satisfy both payer and manufacturer requirements.
- Model financial risk-sharing arrangements appropriate to an ultra-orphan pricing strategy.
- Plan a re-assessment process that revisits pricing as further outcomes data becomes available.
What the participant receives
4 course modules
A structured syllabus
8 training sessions
across 5 days
32 detailed points
Applied, detailed content
Accredited attendance certificate
On completing the programme
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