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

ALS-ETF

Aligning the interests of current patients, industry, and regulators

PatientsRegulators

Drug Companies

Experience, focused in Expanded Access Programs

Medical Advisers

Richard Bedlack, MD, PhD

Richard Barohn, MD

Terry Heiman-Patterson, MD

Gerard Kennealy, MD

Lynn Klein, RN

CRO Network

Cato Research

Kaizen Research

INC Research

ICON plc

Business Advisory

Clinical Operations

Manufacturing Control

Regulatory Affairs

Biostatistics

Legal / Liability

Science Advisory

DeNovo Biomarkers

UCSF

Jackson Labs

Iron Horse Diagnostics

Perceived Challenges:

•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

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

Source: 2008-2009 Survey conducted by IDIS via Pharmaceutical Executive

Decision factors for EAP sponsors

Commercial Risks

Additional Regulatory Risks

Medical Monitoring Concerns

Expense

Address Patients’ Immediate Treatment Need

Economic Reward

Do the factors balance?

Big Regulatory Risk

Uncontrollable CostsUncertain Economic Reward

Q: Do the factors balance?

A: Depends on the company’s situation

For lean companies in poorly understood diseases…………..

• Regulatory Negotiation, representing patients’ needs first, then company’s– 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

501c3 Public Charity. Intrinsic alignment with patients Allows for:

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 Program2. EAP sponsored by 3rd party non-profit partner3. Managed regulatory risk4. 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.

ALIGNMENT OF INTERESTS

Patients / Drug Companies / Regulators

(and Researchers & Clinics, too)

=

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.

SUMMARY