A Third-Party Model for Expanded Access Programs
1. We can deliver large EAPs, far in advance of FDA approval.
2. We can do it in today’s regulatory environment.
The ALS Emergency Treatment Fund. Jess Rabourn
www.ALSETF.org
February 27th, 2014. Washington DC.
Hosted by EveryLife Foundation for Rare Diseases
Aligning the interests of current patients, industry, and regulators
Drug Companies
ALS-ETF
Regulators
Patients
Experience, focused in Expanded Access Programs
Medical Advisers
CRO Network
Business Advisory
Science Advisory
Richard Bedlack, MD, PhD
Cato Research
Clinical Operations
DeNovo Biomarkers
Richard Barohn, MD
Kaizen Research
Manufacturing Control
UCSF
Terry Heiman-Patterson, MD
INC Research
Regulatory Affairs
Jackson Labs
Gerard Kennealy, MD
ICON plc
Biostatistics
Iron Horse Diagnostics
Lynn Klein, RN
Legal / Liability
Decision factors for EAP sponsors
Perceived Challenges:
Perceived Benefits:
•Possibility of adverse events impacting
regulatory filing
•Fulfillment of unmet patient needs (NEED for
OPTIONS)
•Regulatory policies that restrict access to
these drugs
•Collection of additional “real-world” data
outside of the clinical trial setting
•Risk of impairing enrollment in trials
•Development of network of physicians
educated on the proper use of the drug
•Drain of internal company resources
•Different EAP regulations offshore
•Generation of data on drug usage patterns to
help forecast demand
•Possible impact on reimbursement once drug
is launched
•Establishment of KOL relationships prior to
launch
Source: 2008-2009 Survey conducted by IDIS via
Pharmaceutical Executive
Do the factors balance?
Commercial Risks
Address Patients’ Immediate
Treatment Need
Additional Regulatory Risks
Medical Monitoring Concerns
Expense
Economic Reward
Q: Do the factors balance?
A: Depends on the company’s situation
For lean companies in poorly understood diseases…………..
Big Regulatory Risk
Uncontrollable Costs
Uncertain Economic Reward
501c3 Public Charity.
Intrinsic alignment with patients  Allows for:
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Regulatory Negotiation, representing patients’ needs first, then company’s
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Charitable Resources
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Advocate EAP launch early in development cycle
Cost recovery
Clear agreement on findings of AEs
Our own meetings with review division
Project financing
In-kind, pro-bono, at-cost services
Clinic Relationships
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ALSETF providing a service to participating clinics, not the other way around
Cost partnership; no profit margin
The evolution of expanded access business models?
No EAP.
Fail
Traditional EAP.
Not meaningful in size.
No NPO engagement. Expensive.
Nonprofit 3rd party sponsor.
Multi-stakeholder involvement.
Little to no cost to drug company
How can more EAPs be done at meaningful size?
1. Our target community is dying patients who have no
access to clinical trials.
1. Fail enrollment criteria
2. Cannot come frequently to clinic
3. Trial is closed / over-enrolled.
Tens of thousands
unable to
participate in
research.
Cost Recovery = Scalability
These organizations launched scalable humanitarian platforms.
Others help in expanding the reach. (individuals, Oxfam, UNICEF)
Is this the best we can do?
1. Cost recovery - Expandable Access Program
2. EAP sponsored by 3rd party non-profit partner
3. Managed regulatory risk
4. Minimal cost to drug company
LEARNING FROM PATIENTS!
*Learning from Patients*: Responder Profiling and Marker Discovery
Large EAPs can enable discovery of subgroup
markers for more targeted trials, companion
diagnostics, swifter marketing approval, and more
personalized clinical care.
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ALIGNMENT OF INTERESTS
Patients / Drug Companies / Regulators
(and Researchers & Clinics, too)
SUMMARY
1.
FDA cannot offer access programs. IND sponsors do.
2.
FDA policies already permit what we want to do.
3.
A nonprofit 3rd party can make EAP feasible.
4.
Early, Expandable EAP for meaningful impact
5.
Learning From Patients
6.
Not just for ALS! Franchise this to other diseases.