Download Stephen Smith Medidata Solutions Worldwide

21st Century Cures
Legislation:
Modernizing Drug
Development in
Pursuit of Cures
How Laws are Made. House, Senate, President
Stephen Smith
Chief Patient Advocate
Medidata Solutions Worldwide,
Volunteer Committee on Federal
Legislation: National MPS Society
Email: [email protected]
Twitter: @SteveSmithPlans
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Since the 1970s only 500 treatments have been approved for 7000 rare diseases
The federal drug
and device
approval
apparatus is in
many ways the
relic of another
era.
How Laws are Made. House, Senate, President
The 30th of April 2014
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TimeLine: 21st Century Cures
May 2014: start cross-stakeholder discussion
May 2015: Passed 51-0 out of Energy &
Commerce Committee: Unanimous!
July 2015 Passed U.S. House 344-77: strong bipartisan vote!
Fall 2015: in Senate HELP Committee. Contact
your Senators!
Late 2015/Early 2016: POTUS Signs?
21st Century Cures
July 2015:
“Never before have we had such a clear, crossstakeholder definition of what is wrong with
drug development and what we need to do to fix
it. “
Stephen Smith, Patient Advocate since 1990
2012
Patient-Advocacy-Driven Changes
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• Removing barriers to increased research collaboration.
• Incorporating the patient perspective into the drug development and
regulatory review process.
• Measuring success and identifying diseases earlier through personalized
medicine.
• Modernizing clinical trials.
• Removing regulatory uncertainty for the development of new medical apps.
• Providing new incentives for the development of drugs for rare diseases.
• Helping the entire biomedical ecosystem coordinate more efficiently to find
faster cures.
• Investing in 21st Century science and next generation investigators.
• HR 6 helps keep and create jobs here at home.
Highlights of 21st Century Cures
Creating a 21st Century Data Infrastructure
Accelerated Approvals
Incorporate Patient’s Risk Vs. Benefit
Validate biomarkers
Conduct long-term, natural history studies
Incentives to focus on higher hanging fruit
Data Sharing, Re-use, from new sources
Use of Apps
Use of mHealth devices
Use of data: Real time, patient reported, EHR, Medicare
Example: Three Incentives in 21st Century Cures
The Creating Hope Act – up for renewal
Rare Pediatric Disease Voucher reduces FDA review by 4 months
The Open Act
6 months patent extension for repurposing a big disease drug
into a rare disease drug.
The Awakening Dormant Therapies Act
6 months patent extension for creating drug from a project that
had been shelved
Rare Pediatric Disease Voucher
Awarded
Voucher by FDA
For Developing
Disease
Bought Voucher
from Awardee
For This Amount
BioMarin
Vimizim
MPS IVa
Regeneron
$67.5*
Asklepion
Cholbam
Bile Acid
Synthesis
Disorder
Sanofi
$245
United
Therapeutics
Unituxin
Neuroblastoma
Abbvie
$350
* Regeneron used its voucher successfully to speed approval of Praluent a PCSK9 inhibitor
for patient with HeFH, or patients with Clinical Atherosclerotic Cardiovascular Disease.
Repurpose for Rare Disease
Awaken Dormant Projects
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Google:
Twitter:
Steve Smith Patient Advocate
@SteveSmithPlans
Email
Full 21st Century Cures Info
[email protected]
energycommerce.house.gov
Contact your U.S. Senators now!
Ask them to support 21st Century Cures Senate.gov
Aka: Innovation for Healthier Americans
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