Transcript Slide 1

MBSW, May 23, 2012
Improving the Effectiveness of
Comparative Effectiveness:
Perspectives and
Developments from the DIA
Working Group
Matt Rotelli
Director – PK/PD
Eli Lilly and Company
Outline
Overview of Comparative Effectiveness Research
What is the Problem
DIA CER Scientific Working Group
What we are doing
How we are approaching it
Contact info – we need YOU
Company Confidential
Copyright © 2000 Eli Lilly and Company
2
Background
Cost of healthcare increasing
Increasing demand for understanding relative value of
treatments and procedures
Increasing awareness and decreasing tolerance for
potential risks
Increasing demand for personalized medicine
Company Confidential
Copyright © 2000 Eli Lilly and Company
3
Different lenses
Regulators: Is it safe? Is there substantial evidence of
efficacy?
Payers: Is it worth it?
Physicians: What will help this patient?
Patients: What are my choices? What will happen to
me with each? How much will it cost?
For the last 3 consumers of health care information,
these are relative questions
Company Confidential
Copyright © 2000 Eli Lilly and Company
4
CER or PCOR
Comparative (clinical) Effectiveness Research or
Patient Centered Outcomes Research aims to provide
answers on the relative effectiveness of treatment
options and paradigms
American Recovery and Reinvestment Act of 2009 and
Patient Protection and Affordable Care Act of 2010
establish bodies and sustained funding to prioritize and
conduct CER (including exploring methodology and
developing standards)
NOTE: Cost-effectiveness is out of scope
Company Confidential
Copyright © 2000 Eli Lilly and Company
5
PCORI’s Proposed Research Budget
Distribution
PCORI’s National Priorities for Research and Research Agenda (PDF) – adopted by the
PCORI Board of Governors on May 21, 2012
Company Confidential
Copyright © 2000 Eli Lilly and Company
6
CER Landscape
Identifying priority research areas
Comparing delivery systems
Hierarchies of evidence
Observational studies vs. RCTs (+pragmatic trials)
Internal validity vs. external validity
Ways to improve internal validity of observational
studies
Company Confidential
Copyright © 2000 Eli Lilly and Company
7
Observational Studies Increasing
A version of this article appeared May 3, 2012, on page A1 in the U.S. edition of The Wall Street Journal,
with the headline: Analytical Trend Troubles Scientists.
Company Confidential
Copyright © 2000 Eli Lilly and Company
8
Critical Fallacy
“When I need to make a decision, some information is
better than none.”
Only true if the information is not
• Misinformation
• Noise
Evidence needs to be assessed in light of
the research operating characteristics
Company Confidential
Copyright © 2000 Eli Lilly and Company
9
Redefining the problem
It is not where research falls in a hierarchy that
determines it’s weight of evidence, but rather the
reliability and validity of the research
Both RCTs and Observational Studies are essential –
we must understand the properties of each that can
increase the reliability and validity
We can seek to mimic or approximate properties that
make one method reliable and valid in other types of
research
Company Confidential
Copyright © 2000 Eli Lilly and Company
10
DIA CER Scientific Working Group
Opportunity Statement:
The use of Comparative Effectiveness Research (CER)
is increasing and there is a need for continuing noncompetitive collaboration among staff from regulatory
agencies, pharmaceutical and biotech companies, and
academia to share ideas and advance the science of
CER.
Company Confidential
Copyright © 2000 Eli Lilly and Company
11
DIA CER SWG
Goal:
To improve the fidelity (reliability and validity) of CER
used for making health care decisions.
Company Confidential
Copyright © 2000 Eli Lilly and Company
12
DIA CER SWG
Vision:
Comparative Effectiveness Research is designed,
analyzed, interpreted, and communicated appropriately
for use in health care decisions.
Company Confidential
Copyright © 2000 Eli Lilly and Company
13
DIA CER SWG
Mission:
To facilitate the appropriate use of CER and
contribute to progress in CER methodology by:
•
Creating a scientific forum for the discussion, evaluation, and
development of methods and software for the design, analysis, and
interpretation of CER.
Company Confidential
Copyright © 2000 Eli Lilly and Company
14
DIA CER SWG
Mission:
To facilitate the appropriate use of CER and
contribute to progress in CER methodology by:
•
Providing education on and promoting the dissemination of methods
and best practices in CER, setting expectations for high quality
standards, and sharing experiences on case studies.
Company Confidential
Copyright © 2000 Eli Lilly and Company
15
DIA CER SWG
Mission:
To facilitate the appropriate use of CER and
contribute to progress in CER methodology by:
•
Engaging in dialogue and advocacy efforts with industry leaders, the
scientific community, and regulators to develop a world-wide
consensus position on when and how to consider the use of CER.
Company Confidential
Copyright © 2000 Eli Lilly and Company
16
DIA CER SWG
Mission:
To facilitate the appropriate use of CER and
contribute to progress in CER methodology by:
•
Fostering diversity in membership and leadership across:
– Geographical locations
– Institutions (e.g. academia, government, industry)
– Training or areas of specialty (e.g. statistics, medicine, epidemiology,
econometrics)
Company Confidential
Copyright © 2000 Eli Lilly and Company
17
DIA CER SWG
Subgroup: Best Practices
Assess published examples in light of existing
guidelines
Identify gaps and develop recommendations
Develop example-based workshop
Company Confidential
Copyright © 2000 Eli Lilly and Company
18
DIA CER SWG
Subgroup: Framework for testing observational
or post-hoc research methods
Obtain realistic observational data – either publicly
available, contributed, or simulated
Identify or inject known relationships
Assess operating characteristics of research
approaches
OMOP approach but focused on head-to-head
comparisons
Company Confidential
Copyright © 2000 Eli Lilly and Company
19
DIA CER SWG
Hopper projects:
Workshop on measured and unmeasured
confounding
Workshop on Bayesian approaches for indirect
comparisons and network meta-analysis
Company Confidential
Copyright © 2000 Eli Lilly and Company
20
DIA CER SWG
We need YOU
Please contact:
Matt Rotelli
[email protected]
(317) 277-8584
Company Confidential
Copyright © 2000 Eli Lilly and Company
21
Extras
Demos
Links
PCORI info
Challenges facing health care and drug development
Company Confidential
Copyright © 2000 Eli Lilly and Company
22
What you find…
Company Confidential
Copyright © 2000 Eli Lilly and Company
23
… can depend on what you look for
Company Confidential
Copyright © 2000 Eli Lilly and Company
24
Signals… don’t go away
Company Confidential
Copyright © 2000 Eli Lilly and Company
25
Links
OMOP: http://omop.fnih.org/
OMOP presentations:
http://omop.fnih.org/Presentations
PCORI: http://www.pcori.org/
FDA: Advancing Regulatory Science Initiative:
http://www.fda.gov/scienceresearch/specialtopics/regul
atoryscience/default.htm
Company Confidential
Copyright © 2000 Eli Lilly and Company
26
Company Confidential
Copyright © 2000 Eli Lilly and Company
27
Company Confidential
Copyright © 2000 Eli Lilly and Company
28
Company Confidential
Copyright © 2000 Eli Lilly and Company
29
Prescription Medicines Account for about
12% of National Health Care Spending
Prescription Drugs
12%
Hospitals and
Nursing Homes
45%
Other
18%
Physician and
Clinical Services
25%
SOURCE: Centers for Medicare and Medicaid Services, National Health Expenditure Accounts 2006 Highlights
Company Confidential
Copyright © 2000 Eli Lilly and Company
30
Developing a new drug
Average cost to discover and develop a new drug:
$800 million to $1.2 billion
Average length of time from discovery to patient:
10 to 15 years
Patent:
20 years from first filing – clock is running during
R&D
Company Confidential
Copyright © 2000 Eli Lilly and Company
31
Declining New Drug Approvals
Increasing industry spending on research is not yielding a proportionate
increase in numbers of new drugs.
50
25
New drugs approved by FDA
# of new drugs
40
20
R&D spending in billions
30
15
20
10
10
5
0
0
1980
’85
’90
’95
‘00
Sources: Food and Drug Administration; PhRMA
Recreated from Wall Street Journal, April 18, 2002
Company Confidential
Copyright © 2000 Eli Lilly and Company
32
Attrition Rates Are High
Even after a drug
reaches human
testing, attrition rates
remain very high.
Only 11% make it to
approval.
Can the pharmaceutical industry reduce attrition rates?, Ismail Kola & John Landis, Nature Reviews
Drug Discovery 3, 711-716 (August 2004)
Company Confidential
Copyright © 2000 Eli Lilly and Company
33
Many patients do not respond
Efficacy Rate (% )
Oncology
Alzheimer's
25
30
Incontinence
HCV
Osteoporosis
40
47
48
Rheumatoid Arthritis
50
Migraine
Diabetes
51
57
Schizophrenia
60
Cardiac Arrythmias
Asthma
Depression (SSRI)
60
60
62
Analgesics (Cox-2)
80
0
20
40
60
80
100
Spear et al. TRENDS in Molecular
Medicine Vol. 7 No. 5 May 2001
Company Confidential
Copyright © 2000 Eli Lilly and Company
34
Drug safety concerns are high
 Over 2.2 million people impacted
annually by adverse drug reactions
including medical errors
Cause Of Death
Number Of Deaths
Heart Disease
652,091
Malignant Neoplasms
559,312
Cerebrovascular Disease
143,579
Lower Respitory Disease
130,933
Accidents
117,809
Adverse Drug Reactions
106,000
National Vital Statistics Reports, Vol. 56, No. 10, March 7, 2008, and Lazarou, Pomeranz, Corey. Incidence of adverse drug reactions in hospitalized
patients: a meta-analysis of prospective studies. JAMA 1998; 279: 1200-1205
Company Confidential
Copyright © 2000 Eli Lilly and Company
35