The benefits of "Big Data" in health care will be undermined by the Valley of Death resulting from the FDA's drug approval process. The drug approval process should be changed as recommended herein.

1. Coalition to Unleash “Big Data” in
Healthcare
CROSSING THE VALLEY OF DEATH
IN DRUG DISCOVERY
CUB – November 2015

2.  Identifying problems before they happen
 Tailoring medical solutions to a person’s unique DNA profile
 Identifying trends and relationships to identify cures
 Predicting epidemics
“BIG DATA” IS OFFERING TREMENDOUS
OPPORTUNITIES IN HEALTHCARE
The Opportunities are Endless and the Benefits Are Enormous

3.  The risk, cost and delay of the current drug approval process
creates a hostile investment environment
 Preclinical involvement by the FDA creates unpredictable delays
 12 years of clinical trials on average
 Unpredictable outcomes (high risk)
 Over $1 billion in development cost per approved drug
 The result is that although 15,000 new chemical substances
are registered per day, there are only about 25 new drug
approvals per year
 That is, the vast majority of scientific discoveries never cross
the Valley of Death between the lab and regulatory approval
BUT THE CURRENT DRUG APPROVAL
PROCESS CREATES A “VALLEY OF DEATH”

4.  Shift responsibility for safety and effectiveness to qualified
research organizations and focus the FDA on enforcement
 The FDA’s role in pre-clinical research should be limited to surveillance
 Qualified research organizations should be required to provide details
for each trial application and put substantial funds into escrow
 FDA would have 60 days to review each trial application, after which a
provisional license would automatically be granted
 FDA would have the right to file an injunction in Court against any
healthcare solution that it believes is or would be harmful to the public
 If an injunction is granted at any time during the process, the research
organization would lose its escrowed funds and its provisional license
 Without an injunction, the research organization would provide detailed
consumer information one year after commercial launch (in addition to
results for each trial); 60 days after which a commercial license would
be granted and the escrowed funds would be returned
PROPOSED LEGISLATIVE REFORM

5.  The “Valley of Death” in the current drug approval process
is undermining the massive benefits available from the
application of “Big Data” in healthcare
 The proposed changes give clear guidelines and proper
incentives to researchers, the investment community and
the FDA to enable commercial deployment of a much
greater number of safe and effective healthcare solutions
 Congress could implement such an approach cautiously
by implementing it for one or two chronic conditions such
as Alzheimer’s or Parkinson’s disease and/or metastatic
cancer
SUMMARY
For more information, see www.thevalleyofdeath.info

6.  Phase 1 - $250,000 per trial participant
 Phase 2- $100,000 per trial participant
 Phase 3 - $50,000 per trial participant
[Note: FDA would have authority to adjust for specialty applications]
EXHIBIT I. ESCROW DEPOSIT SCHEDULE
Number of
Participants
Incremental
Deposit
Cumulative Deposit
Phase 1 20 - 40 $5 -10 million $5 - 10 million
Phase 2 100 - 200 $10 -20 million $15 – 30 million
Phase 3 1000 - 3000 $50 – 150 million $65 - 180 million
* Deposits returned one year after commercial launch
Illustrative Deposits *

7.  Minimum of 5 large, brand-name organizations representing
research organizations, public interest groups and healthcare
providers is required before a recognized spokesperson is
chosen and lobbying begins
EXHIBIT II: COALITION MEMBERS

8.  Steven A. Zecola has held executive management positions in
large companies, entrepreneurial companies and the federal
government. He has raised over $800 million in capital for start-
up enterprises. He has also worked as a venture capitalist,
consultant and professor, teaching classes in Strategic
Management, Marketing Management, and Entrepreneurship for
an accredited MBA program.
 Zecola has spoken publicly on over a hundred occasions covering
topics from nanotechnology to information technology to
biotechnology to entrepreneurship.
 Zecola has written two books including The Race to Cure
Parkinson’s disease (2006).
 Zecola holds a Master of Arts in Economics from the University of
Virginia and a Bachelor of Arts in Economics from the State
University of New York at Stony Brook.
 Zecola was diagnosed with Parkinson’s disease in 2001.He can
be reached at szecola@gmail.com or 571-926-9692.
EXHIBIT III. PRINCIPAL ORGANIZER