The DMO:
Improving the
Quality, Quickness
and Quantity of
Access to
Alzheimer’s
Disease Treatments
By Whitney Davis
2011 WISE Intern
University of Arkansas
August 4, 2011
US Food and Drug
Administration
 FD&C
Act passed in 1938 (safety)
 Kefauver Harris Amendment in 1962
(efficacy)
 Only entity legally responsible for
approval of:

Medical devices, food, vaccines, veterinary
treatments, radiation-emitting products,
cosmetics, tobacco products, and drugs
for all diseases, conditions, and products
Wheel
chairs
Heart
Monitors
Imaging
Systems
Catheter
Hearing
Aids
Defibrillator
Xray
Systems
MS
Biologics
Dietary
Supplements
Animal
Feed
Vaccines
Arthritis
Pacem
akers
Alzheimer’s
Disease
Drugs
Tanning
Products
Food
Additives
Vet
Products
Food
Lou
Gehrig’s
Smallpox
Infant
Formulas
Cardiov
ascular
Disease
West
Nile
Virus
Tobacco
Products
Asthma
Breast
Implants
Blood
Products
HIV
Cosmetics
Radiation
-emitting
Products
Medical
Devices
Hepatitis
Addison’s
Disease
Cancer
Fertility
Drugs
Lupus
SARS
Artificial
Hearts
Influenza
Heart
Valves
Overview:
Alzheimer’s DiseAse

A progressive, degenerative disorder that attacks
the brain's nerve cells, or neurons, resulting in
behavioral changes and loss of memory, thinking,
and language skills

6th Leading Cause of Death in US

Average life expectancy after diagnosis is 8 years

Age is greatest risk factor

5.4 million people live with AD
Economic Impact of AD
 Total
cost for care: $183 billion
 Annual cost of care: $18,500 - $36,000
 Unpaid care in 2010: 14.9 million family &
friends provided 17 billion hours = $202.6
billion
 Costs US businesses $60 billion a year
 Medicare payments are 3x higher
 Medicaid payments are 9x higher
Phase
IV:
Safety
&
Efficacy
Current FDA Challenges
Safety
Efficacy
Safety
&
Efficacy
To Develop A Drug:
o Average Time: 10 years
o Average Cost: $1 billion
o User Fee: $1 million
Current AD Treatments
Name:
Phase: Use:
Success:
Completed:
Methylene
Blue
2
Prevent
aggregati
on of tau
protein
fibers
Positive
Yes
PBT-2
2
Prevent
Positive
aggregati
on of betaamyloid
into
plaques
Yes
Solanezumab 3
Labproduced
antibody
to betaamyloid
N/A
No
Government Reactions

Fast Track




Accelerated Track




product must concern a serious or life-threatening condition and has to
have potential to address an unmet medical need
allows manufacturers to submit sections of their NDA to the FDA as they
are ready rather than submitting a complete application at one time
can meet with FDA so that components of clinical study design and
presentation can be clearly defined
accelerates approval of a drug that provides a “meaningful
therapeutic benefit…over existing treatments”
approval based ongoing clinical trials
instead of survival rates or disease progression, a “surrogate endpoint
that is reasonably likely…to predict clinical benefit” is used
Priority Review


Intended for products believed to address unmet needs
shortens the approval decision from 10 months to 6 months
Results
o
o
o
Manufacturers have requested Fast
Track designation for 569 drugs since
the Fast Track program was set into law
74.5% of those drug requests were
granted
Of products with Fast Track
designation, 10.6% of the drugs were
approved
Current FDA Challenges
 Economically
Unmanageable Amount of
Responsibility
 Single Option for Drug Developers
 Inefficient In Accurately Perceiving
Demand
 Can’t specify around a particular disease
 Blanket “safety” definition
Economically Unmanageable
Amount of Responsibility
o
o
o
Needs funds to match human resources & facilities to
responsibility
Financial resources get spread thin
More funds with little production
Economically Unmanageable Amount of
Responsibility
 Highly
Single Option
restricted
early access
except through
clinical trials
 No personalization
 No incentive to
improve:



Trials
Cost
Access
Inefficient In Accurately
Perceiving Demand
 Revenue
not
directly affected
by demand
 Revenue always
guaranteed
The DMO: Direct Market
Option





After submitting an IND, drug developer will have option to
pursue DMO parallel to the conventional FDA approval
track
Can legally gain approval by free market organization
Only requirement: drug developer must enter into a
contract with organization in which the drug developer
possesses liability for unknown or unlisted adverse effects
Drug developer can run clinical trials set under different
conditions and methods by a free market organization
Organization responsible for:


post-market research
fining the company for false labeling and advertising as seen fit.
Open More Options


More approved drugs
Better specialize



tradeoff of risk vs.
benefit
potentially earlier
access to drugs
More competition to
meet demand

more efficient trials



faster access
cheaper drugs
cheaper user fees
More Accurately Meet
Demand
 Revenue
directly
affected by
demand
 Can specialize &
require less
resources
 Focus funds for
better quality
Questions?
Whitney Davis
IEEE – WISE Intern
wxd003@uark.edu