Expanded Access Programs: Opportunities and Challenges for Pharma
Expanded access programmes make available investigational drugs to chronically sick or dying patients who have no other licenced product or clinical trial alternative. Driven by clinicians and patients, this highly-emotive area has both benefits and risks for pharma. What are the advantages? Where are the dangers? Does pharma have a moral imperative to engage?
Expanded Access Programs: Opportunities and Challenges for Pharma is a detailed report for industry management who must assess, approve or manage expanded access requests and programmes. Enriched with case studies, the report reveals the “real-world” experience and opinions of 17 senior industry, regulatory and stakeholder experts in the US and Europe and presents critical insights on the current operating environment for expanded access programmes.
Answers to Critical Questions
- Understand clinician and patient drivers for expanded access
- Formulate strategies to respond to requests that meet the needs of the wide variety of stakeholders involved
- Understand the complex and varying regulatory requirements in leading markets
- Balance the risks and “real-world” research benefits of engaging in expanded access programmes
- Assess whether running your own expanded access programmes or engaging a service company is the route to go
- Review the therapeutic areas currently attracting interest for expanded access programmes
- Be aware of the PR benefits and dangers of engaging in expanded access programmes
- Understand how “Right to Try” and CURE legislation may impact this area in the US
- Expanded Access: how best to manage clinician/patient requests?
- Regulation of expanded access programmes varies widely in the US and EU – what are the essentials you need to know?
- Companies are often expected to foot the bill for expanded access programmes – so what are the research and communication upsides that would support your participation?
- What role can service companies play in smoothing access and management between pharma and clinicians/patients?
- Avoiding unintentional consequences: how could expanded access impact the main drive to get the compound to market for all patients?
- Media can be valuable partners in getting awareness of expanded access programme availability, but can also backfire if things don’t go to plan. How can you manage communications and avoid falling into a PR black hole?
- Understand the factors that drive patients and clinicians to submit requests for early access to investigational drugsBe aware of the positive – if complicated - regulatory environment in the US and EU governing early access programmes
- Examine the financial implications: you may get paid, but more often you’ll be footing the bill
- Learn from “real world” experience on how industry is engaging with expanded access programmes and meeting the challenges they present
- Identify the key benefits that accrue in terms of early exposure of your investigational drug in a real world setting
- Appreciate how an early access programme can help support and speed your product application, and also how they can slow progress to regulatory approval.
Pharma and biotech experts
Service provider experts
- CEO and Founder, radiopharmaceutical company
- Chief Commercial Officer, global biopharmaceutical
- Senior Director of Global Pricing and Reimbursement, global biopharmaceutical
- CEO, biotechnology company
- A representative from a leading biotech company
- A representative from a top 15 pharma company
- CEO, pharma company
- COO, biopharmaceutical company
- Senior medical director, pharma company
Patient advocacy groups
- Head of Project Management, Global Access Programmes
- Senior Vice President, Global Access Programmes
- Head, US programme operations
- Director, commercial development
- Marketing Director
- Senior Project Director
- President and co-founder, Marti Nelson Cancer Foundation
- President and founder, Melanoma International Foundation
- Spokesperson, UK's Medicines and Healthcare Products Regulatory Agency
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- 1.Executive summary
- 2.Research objectives and methodology
- 4.Expanded access programmes: An introduction
- 5.Finding a definition for expanded access
- 6.The drivers behind expanded access programmes
- 6.1.Drug provision: The role of the pharma company in expanded access
- 7.The expanded access process from supply to patient
- 7.1.Expanded access in the US: History, regulations and practical applications
- 7.2.Regulations and application for expanded access across Europe
- 7.3.Expanded access in France – the ATU process
- 7.4.Expanded access in the UK – early access to medicines
- 7.5.The role of the service provider in expanded access
- 8.Who pays for expanded access?
- 9.The benefits and challenges for expanded access programmes
- 9.1.Expanded access: Benefits and risks for patients
- 9.2.Sponsor and services companies – benefits and challenges
- 9.3.The role of physicians in expanded access
- 9.4.Expanded access and therapeutic areas
- 9.5.Expanded access by companies
- 9.6.The challenge of communications
- 9.7.Expanded access in the future