A Third-Party Model for Expanded Access Programs

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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 todays regulatory environment.
The ALS Emergency Treatment Fund. Jess
Rabourn www.ALSETF.org February 27th, 2014.
Washington DC. Hosted by EveryLife Foundation for
Rare Diseases
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Aligning the interests of current patients,
industry, and regulators
Drug Companies
ALS-ETF
Regulators

Patients
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Experience, focused in Expanded Access Programs
Medical Advisers
CRO Network
Business Advisory
Science Advisory
Richard Bedlack, MD, PhD Richard Barohn, MD Terry
Heiman-Patterson, MD Gerard Kennealy, MD Lynn
Klein, RN
DeNovo Biomarkers UCSF Jackson Labs Iron Horse
Diagnostics
Clinical Operations Manufacturing
Control Regulatory Affairs Biostatistics Legal /
Liability
Cato Research Kaizen Research INC Research ICON
plc
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Decision factors for EAP sponsors
Perceived Challenges
Perceived Benefits

• Fulfillment of unmet patient needs (NEED for
  OPTIONS)
• Collection of additional real-world data
  outside of the clinical trial setting
• Development of network of physicians educated on
  the proper use of the drug
• Generation of data on drug usage patterns to help
  forecast demand
• Establishment of KOL relationships prior to
  launch

• Possibility of adverse events impacting
  regulatory filing
• Regulatory policies that restrict access to these
  drugs
• Risk of impairing enrollment in trials
• Drain of internal company resources
• Different EAP regulations offshore
• Possible impact on reimbursement once drug is
  launched

Vs.
Source 2008-2009 Survey conducted by IDIS via
Pharmaceutical Executive
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Do the factors balance?
Commercial Risks Additional Regulatory
Risks Medical Monitoring Concerns
Address Patients Immediate Treatment Need
Vs.


Expense
Economic Reward
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Q Do the factors balance?
A Depends on the companys situation
For lean companies in poorly understood
diseases..
Big Regulatory Risk Uncontrollable Costs
Uncertain Economic Reward
Vs.
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501c3 Public Charity.
Intrinsic alignment with patients ? Allows for

• Regulatory Negotiation, representing patients
  needs first, then companys
• Advocate EAP launch early in development cycle
• Cost recovery
• Clear agreement on findings of AEs
• Our own meetings with review division
• Charitable Resources
• Project financing
• In-kind, pro-bono, at-cost services
• Clinic Relationships
• ALSETF providing a service to participating
  clinics, not the other way around
• Cost partnership no profit margin

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The evolution of expanded access business models?
Traditional EAP. Not meaningful in size. No
NPO engagement. Expensive.
Nonprofit 3rd party sponsor. Multi-stakeholder
involvement. Little to no cost to drug company
No EAP. Fail
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How can more EAPs be done at meaningful size?

• Our target community is dying patients who have
  no access to clinical trials.
• Fail enrollment criteria
• Cannot come frequently to clinic
• Trial is closed / over-enrolled.

Tens of thousands unable to participate in
research.
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Cost Recovery Scalability
FairWater BluePump
These organizations launched scalable
humanitarian platforms. Others help in expanding
the reach. (individuals, Oxfam, UNICEF)
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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
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LEARNING FROM PATIENTS!
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Learning from Patients Responder Profiling
and Marker Discovery
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Large EAPs can enable discovery of subgroup
markers for more targeted trials, companion
diagnostics, swifter marketing approval, and more
personalized clinical care.
ALIGNMENT OF INTERESTS Patients / Drug
Companies / Regulators (and Researchers
Clinics, too)

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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.