MODDERN Cures Solution

MODDERN Cures Solution Modernizing Our Drug and Diagnostics Evaluation and Regulatory Networks Marc Boutin Executive Vice President & COO National Health Council May 2011

The mission of the National Health Council is to provide a united voice for people with chronic diseases and disabilities. © National Health Council

Despite many medical breakthroughs, significant unmet need remains for millions of people suffering from chronic conditions. © National Health Council

© National Health Council ALZHEIMER’S DISEASE CASE STUDY 100 + YEARS CLINICAL TRIALS 1990s Testing of early drugs to treat symptoms 1990s-Present Tests of diagnostics to identify plaque in brain TRANSLATIONAL RESEARCH 1976-Early ‘90s R&D of first drugs for symptoms 1976-Present Search for drugs to treat or prevent disease 1980s-2001 Research Dx test to detect amyloid BASIC RESEARCH 1906 First Identified 1976 High prevalence acknowledged FDA EVALUATION/APPROVAL 1990s-Present Total of 4 drugs approved to treat symptoms Still waiting for diagnostic tools to accurately identify patients and treatments to prevent, slow, or arrest disease.

© National Health Council ALS CASE STUDY 140 + YEARS CLINICAL TRIALS 1993 Trials on Riluzole begin 2009 FDA approval of SOD1 antisense and stem cell trials TRANSLATIONAL RESEARCH 1950s-Present Research conducted to identify treatments 1950s Nerve growth factor identified 1990 Growth factor CNTF found to increase survival of motor neurons BASIC RESEARCH 1869 First identified 1939 Lou Gehrig diagnosed 1993 SOD1 gene mutation found in familial ALS 2000 NINDS issues first RFA for ALS research FDA EVALUATION/ APPROVAL 1996-Present Riluzole approved by the FDA It extends life approximately 3 months in some patients No other approved treatment available Still waiting for a cure, diagnostic tools, and treatments that slow or stop the progression of ALS. Meanwhile, the average patient dies within 2-5 years of diagnosis.

© National Health Council PKD CASE STUDY CLINICAL TRIALS 2000 CRISP Disease progression and TKV 2011 Tolvaptan Phase III trial Additional Trials: somatostatin, sirolimus & bosutinib
TRANSLATIONAL RESEARCH
1995 – Present:
Identification of disease pathways
cAMP
Vasopressin
mTOR
AKT
BASIC RESEARCH 1757 First identified 1957 First demographic study 1994 PKD1 gene identified 1996 PKD2 gene identified FDA Evaluation/ Approval 2010 PKD Outcomes Consortium: Analyze patient registry data for validation of TKV as a clinical endpoint for CT’s No therapies have been approved to treat PKD. Still waiting for treatments to slow or stop progression. Meanwhile, 50% of PKD patients will go into ESRD by age 60. Dialysis and transplantation remain the only options available. 250 + YEARS

Addressing Unmet Need
Moses H, Dorsey E, Matheson D, Thier S (2005). "Financial anatomy of biomedical research.". JAMA 294 (11): 1333–42. doi:10.1001/jama.294.11.1333. PMID 16174691
Dorsey ER, Thompson JP, Carrasco M, de Roulet J, Vitticore P, et al. 2009 Financing of U.S. Biomedical Research and New Drug Approvals across Therapeutic Areas. PLoS ONE 4(9): e7015. doi:10.1371/journal.pone.0007015

BARRIER #1: Drug compounds not developed because of insufficient legal protection © National Health Council

Why Patents Don’t Always Work
Compound is reasonably likely to succeed
A discovery was publicly disclosed before a patent application was filed.
A new treatment was patented, but not approved by the FDA. The patent has since expired.
A patent application was filed but withdrawn prior to approval.
© National Health Council

Stages of Research and Development © National Health Council Drug Discovery Preclinical Phase Clinical Trials FDA Review 5,000 to 10,000 Compounds  250 Compounds  5 Compounds Clinical Phases I II III  One FDA-Approved Drug 3 to 6 Years 6 to 7 Years 0.5 to 2 Years

Lost Opportunities © National Health Council Company Product Description Development stage Disease Abbott ABT-491 Elastase inhibitor Clinical Trials Asthma Novartis Recombinant AT-1 Alpha1-Antitripsin Phase II Emphysema Dupont CMP-777 Proteinase inhibitor Phase I/II CF, Emphysema Cortech/United Ther CE-1037 HNE inhibitor Phase II CF, Emphysema, ARDS Cortech/Ono CE-2000 series HNE inhibitor Pre-clinical CF, Emphysema Dyax EPI-HNE-4 HNE inhibitor Clinical Bronchitis, CF, Emphysema Amgen SLP-1 Recombinant inhibitor Pre-clinical Asthma Biogenidec BG-12 Fumarate derivative Phase III MS

Create a new class of drugs called dormant therapies
Address unmet need
Where there is no available treatment or
Where the new treatment
Improves outcomes or
Reduces risk
Have insufficient patent protection
Emphasize therapies to stop or slow disease progression
MODDERN Cures Solution #1 © National Health Council

BARRIER #2: Lack of alignment between market incentives and personalized medicine © National Health Council

Use data protection to encourage
co-developed and companion diagnostics
Advance personalized medicine:
Get the right treatment to the right patient
at the right time

BARRIER #3: Delayed access to FDA-approved diagnostic tools © National Health Council

© National Health Council PathVysion: HER-2 DNA Test to Identify Candidates for Herceptin Breast Cancer Therapy
DIAGNOSTIC DEVELOPMENT
1994 Initial Development Begins
PRE-VALIDATION VALIDATION
REGULATORY SUBMISSION
1999 FDA Approval
2000-04 CMS
Reimbursement Code
2005 Coding Implemented
BIOMARKER DISCOVERY PRODUCT LAUNCH 11 YEARS

Allow diagnostic manufacturers to apply for temporary national code until permanent code can be assigned
Temporary assignments should occur quarterly throughout the year

Identify key factors to be considered when determining payment amount for new diagnostic tests
Impact on Care
Laboratory Charges
Private Insurance Rates
Establish independent advisory panel comprised of diagnostic experts, including patients

Estimated Outlay and Revenue Effects of MODDERN Cures Solution (Outlay Increases are positive number) © National Health Council Provision 5-Year Cost or Revenue Loss 10-Year Cost or Revenue Loss Accelerate the Release of Guidelines – – Improve the Coding and Reimbursement System $5 million $10 million Companion Diagnostics for Existing Drugs/Co-developed Companion Diagnostics negligible $10 million Dormant Therapies – – Total Cost $5 million $20 million

Ensures that science, not the law, drives the development of new therapies
Promotes targeted therapies
Guarantees immediate access to FDA approved diagnostic tests
Rationalizes the reimbursement system
MODDERN Cures Solution © National Health Council

Marc Boutin Executive Vice President & COO National Health Council [email_address]

MODDERN Cures Solution

by National Health Council on May 11, 2011

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