Transcript Caitlin Rothermel and Meg Franklin

The Dossier
Developing Effective
Academy of Managed Care Pharmacy
and Global Value Dossiers
Caitlin Rothermel, MA, MPH
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Market Access Around the World
Just as health care systems vary worldwide, payer assessments
differ from country to country, and even within the same country
Figure: Evidera
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Global Market Access: Key Issues
To successfully launch a drug in multiple markets, you have to
understand the particular requirements of each payer
• Worldwide, payers weigh data in different ways when deciding whether to
pay for a new drug, and what its price should be
• Public agencies look at clinical benefit, budget impact, and overall costeffectiveness (CE)
– The UK evaluates clinical and budget impact, but bases its decisions on CE
– A German agency looks separately at clinical benefit and affordability, weighting
both to decide
– A Swedish agency evaluates costs and benefits through a societal lens
• Private agencies look at clinical benefit and budget impact
– This includes most private U.S. payers
• Dossiers address the common concerns and information needs of a range of
payers
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What Is A Global Value Dossier (GVD)?
With increasing medical costs, aging populations, and budget
deficits, countries and payers need to develop objective
requirements for coverage and reimbursement
• A GVD is a comprehensive, internal document used to communicate the
value of a product to payers and other relevant stakeholders
• It includes available evidence on the global burden of the relevant illness, as
well as the clinical, economical, and humanistic value of the new treatment
• It provides a comprehensive reference base that can be tailored for use in
different markets
• Typically, GVDs are developed by global headquarters with local affiliate
input; they are generally prepared in a modular format
• GVDs are also internal tools for product launch education—to ensure that
all team members are on the same page in terms of value messaging; they
can also serve as the basis to develop Health Technology Assessments
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Dossiers In Value Story Communication
A value story communicates unmet treatment needs and shows how a
specific product can fill those needs; a dossier assembles this scientific
information and messaging into a single reference document
Disease state,
practice
patterns
Why should payers &
providers care about this
disease?
Current
Treatments
What do payers &
providers need to know
about current treatment?
Product
Effectiveness &
Economic Value
Why should payers &
providers cover your
product?
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What Are Value Messages?
• Value messages are developed in collaboration with clinical, marketing,
commercial, and other departments
• Each message should be supported by scientifically accurate evidence
• Message testing may include primary research with payers, physicians, and
patients
• Draft value hypotheses
should be prepared
early in product
development
(~Phase 2) to
help guide clinical
development and the
selection of trial endpoints
• Final messages are written
after clinical data are available
Barrows S. ISPOR 14th Annual International Meeting, May 2009.
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Examples, Value Messages and
Target Audiences
Patients
Increases length of life over first 9 months
of treatment vs normal care
Prescribing
Physicians
Improves cognition and neurological
functioning from day 28
Regulatory
Authorities
Provides statistically significant, clinically relevant
reductions in 28-day all-cause mortality and an
acceptable risk-benefit profile vs standard care
Pricing &
Reimbursement
Authorities
Provides cost-effective benefits based on
cost per life-year gained and/or cost per
quality-adjusted life year
Barrows S. ISPOR 14th Annual International Meeting, May 2009.
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Global Value Dossier
What’s In the Dossier? (1 of 3)
1. Introduction
• Purpose of/how to use the GVD
2. Burden of Disease (The Problem)
• Disease background
• Epidemiology, including prevalence/incidence, mortality,
comorbidities
• Humanistic burden (eg, health-related quality of life,
functional status, symptoms)
• Unmet treatment need with current therapies
• Sources of information: systematic literature review of peerreviewed literature, national/international professional
organizations, federally sponsored research organizations
Rycroft CE. J Eur Med Writers Assn. 2010;19(3):211-212.
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Global Value Dossier
What’s In the Dossier? (2 of 3)
3. Product Value (The Solution)
• Clinical value: efficacy and effectiveness, safety and
tolerability
• Patient-reported outcomes, quality of life value: healthrelated quality of life, functional status, compliance,
satisfaction/preference, caregiver burden
• Economic value: cost-effectiveness, budget impact, associated
decrease in health care utilization
• Sources of information: sponsored and other studies of
product
Rycroft CE. J Eur Med Writers Assn. 2010;19(3):211-212.
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Global Value Dossier
What’s In the Dossier? (3 of 3)
4. Country-specific Information
• Enables adaptation of GVD for different markets and
populations; includes:
–
–
–
–
Epidemiology
Economic and health-related quality of life burden
Country-specific clinical considerations, including key comparators
Country-specific economic considerations, including reference
drugs
– Country-specific value message considerations (value may vary by
country related to differences in available comparators, treatment
guidelines, and physician awareness of adverse events)
Rycroft CE. J Eur Med Writers Assn. 2010;19(3):211-212.
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Writing GVDs: Practical Issues
• Generally no predefined format, but still highly structured and
visually appealing
– Movement towards “iGVDs” – to view on tablets and mobile devices
• Brevity and organization are key! Avoid the urge to use the GVD as a
“data dump”
• Typically developed in
collaboration with multiple
stakeholders—ie, many people
provide input (and revisions)
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Market Access In the U.S.
In the U.S., the key
healthcare payers are a mix
of governmental and
private organizations
• U.S. Centers for Medicare and
Medicaid Services
• Managed Care Organizations
• Indian Health Service
• Department of Veterans Affairs
• Pharmacy Benefit Management
• Consortia, Employers
• Individual Market
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What Is the AMCP Dossier?
• The AMCP Dossier is the U.S. industry standard by which managed
care organizations request evidence-based information to evaluate
pharmaceuticals, biologics, and vaccines for formulary placement,
coverage, and reimbursement decisions
• The AMCP Dossier Format provides a standardized template to
present clinical and economic evidence
– Developed to improve clarity and
transparency, to streamline evidence
acquisition and decision-makers’ review
process
– Users include national and regional health
insurers, pharmacy benefit managers, federal
agencies, hospital systems, and state Medicaid
agencies—representing >150 million
Americans
AMCP: Academy of Managed Care Pharmacy
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Placeholder: AMCP Dossier Format
Release/Update History
• 1999
– Academy of Managed Care Pharmacy (AMCP) Committee organized
• 2000 – AMCP Format Version 1.0
– AMCP Board of Directors approves AMCP Format
• 2002 – AMCP Format Version 2.0
• 2005 – AMCP Format Version 2.1
• 2009 – AMCP Format Version 3.0
– Substantial format revisions from version 2.0 to 3.0
• 2013 – AMCP Format Version 3.1
– Updated to include:
• Companion Diagnostic Tests
• Comparative Effectiveness Research
• Specialty Pharmaceuticals
• 2015/2016 – Next update likely?
Jackson J. AMCP Format for Formulary Submissions (slide presentation). February 2014.
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How Are AMCP Dossiers Distributed?
The Unsolicited Request Process
• Requires an unsolicited request from the health plan or
equivalent—product dossiers cannot be sent to/viewed by a health
system without an unsolicited request
• Processes for unsolicited requests established by the U.S. FDA; key
elements of manufacturer’s safe harbor to provide medical
information in response to an unsolicited request
– Must be truly unsolicited, without prompting, suggestion, or
solicitation by the drug manufacturer or its employees
– Focus on data rather than company-generated discussions of data
– Prepared by individuals with scientific and medical training
– Does not deliberately go beyond the scope of the request
– Does not include promotional materials
– Objective, balanced, and scientifically rigorous
AMCP: Academy of Managed Care Pharmacy; FDA: Food and Drug Administration
AMCP/FDA Letter. March 26, 2012. Available at: www.amcp.org/WorkArea/DownloadAsset.aspx?id=14941
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AMCP Dossier: Not a Promotional
Document
“The quality and comprehensiveness of evidence
within the dossiers remains an issue. Plans are
warning that if the evidence dossiers are hijacked
by commercial interests in the companies or from
outside, they will not use them. They desire a
scientific communication—not promotion.”
-Sean Sullivan
University of Washington
AMCP: Academy of Managed Care Pharmacy
Sullivan S. FDA Unsolicited Request and the AMCP Format. ISPOR 2012. Available at:
http://www.ispor.org/meetings/WashingtonDC0512/releasedpresentations/IP4-Sullivan.pdf
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AMCP Dossier Format Version 3.1
What’s In the Dossier? (1 of 8)
Section 1.0: Executive Summary –
Clinical and Economic Value of the Product (2.5 pages)
 1.1 Clinical Benefits (≤ 1 page)
 1.2 Economic Benefits (≤ 1 page)
 1.3 Conclusions (≤ ½ page)
The manufacturer should articulate a value argument to justify
expected expenditures for this product in the context of its
anticipated effects on the clinical evidence, health outcomes, and
economic consequences for the healthcare system
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AMCP Dossier Format Version 3.1
What’s In the Dossier? (2 of 8)
Section 2.0: Place of Product in Therapy
Subsection 2.1: Product Description (≤ 20 pages)
•
•
•
•
•
•
•
Generic and brand name, and therapeutic class of the product
All dosage forms, including strengths and package sizes
NDC for all formulations
The ASP and WAC cost per unit size
AHFS or other Drug Classification
U.S. FDA-approved indication(s) and date
Pharmacology, pharmacokinetics/pharmacodynamics
AHFS: American Hospital Formulary Service; AMCP: Academy of Managed Care Pharmacy; ASP: Average Sales
Price; FDA: Food and Drug Administration; NDC: National Drug Code; WAC: Wholesale Acquisition Cost
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AMCP Dossier Format Version 3.1
What’s In the Dossier? (3 of 8)
Section 2.0: Place of Product in Therapy
Subsection 2.1: Product Description (≤ 20 pages, Cont’d)
• Contraindications/warnings/precautions/adverse effects
• Interactions(drug/drug, drug/food, drug/disease)
• Dosing and administration
• Access, e.g. restrictions on distribution, supply; prescribing
restrictions
• Co-prescribed/concomitant therapies
• Big Table: Concise comparison of prescribing information of
target product and primary comparator products in same
therapeutic area
AHFS: American Hospital Formulary Service; AMCP: Academy of Managed Care Pharmacy; ASP: Average Sales
Price; FDA: Food and Drug Administration; NDC: National Drug Code; WAC: Wholesale Acquisition Cost
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AMCP Dossier Format Version 3.1
What’s In the Dossier? (4 of 8)
Section 2.0: Place of Product In Therapy
Subsections 2.2.1, 2.2.2, 2.2.3, and 2.3
• 2.2.1 Disease Description (≤ 2 pages)*
– Format as bullets and tables
• 2.2.2 Approaches To Treatment (≤ 2 pages)*
– Format as bullets and tables
• 2.2.3 Relevant Treatment Guidelines and Consensus
Statements
• 2.3 Evidence for Pharmacogenomic Tests and Drugs
* Can be expanded to 3 pages
for Specialty Pharmaceuticals
AMCP: Academy of Managed Care Pharmacy
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AMCP Dossier Format Version 3.1
What’s In the Dossier? (5 of 8)
Section 3.0: Supporting Clinical Evidence
Subsection 3.1: Summaries of Key Clinical Studies Supporting Labeled Indications
(≤2 pages/study)
a.
b.
c.
d.
e.
f.
g.
h.
i.
j.
k.
l.
m.
Name of the clinical trial or study and publication citation(s)
Study objective, location and date(s)
Trial design, randomization, and blinding procedures
Setting, inclusion, and exclusion criteria
Patient characteristics
Dropout rates and procedures for handling dropouts
Treatment, including dosage regimens, washout period
Clinical outcome(s) measured (delineate primary vs. secondary endpoints)
Other outcomes measured (e.g., patient-reported outcomes) and principal
findings
Statistical significance of outcomes and power calculations
Validation of outcomes instruments (if applicable)
Generalizability of the population treated
Study limitations, as stated by the authors
AMCP: Academy of Managed Care Pharmacy
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AMCP Dossier Format Version 3.1
What’s In the Dossier? (6 of 8)
Section 3.0: Supporting Clinical Evidence
Subsections 3.1.1., 3.1.2., 3.1.3, 3.1.4
 3.1.1 All Other Relevant Published and Unpublished Clinical Studies
Supporting Labeled Indications
 3.1.2 All Published and Unpublished Data and Clinical Studies
Supporting Off-Label Indications
 Additional items: a) U.S. FDA CDER Office of Drug Safety; b) Access
information for ongoing clinical trials (at clinicaltrials.gov).
 3.1.3 Clinical Evidence Spreadsheets of all Published and
Unpublished Trials
 3.1.4 Summary of Evidence From Secondary Sources (Cochrane
reviews, systematic reviews, AHRQ evidence summaries, HTAs)
AHRQ: Agency for Healthcare Research and Quality; AMCP: Academy of Managed Care Pharmacy;; CDER: Center22for
Drug Evaluation and Research; FDA: Food and Drug Administration; HTA: Health Technology Assessment
AMCP Dossier Format Version 3.1
What’s In the Dossier? (7 of 8)
Section 4.0: Economic Value and Modeling Report
(≤20 pages)
•
•
•
•
•
•
•
•
4.1 Abstract
4.2. Introduction / Background
4.3 Methods
4.4 Results
4.5 Limitations
4.6 Discussion
Minimum 2 Figures, 3 Tables (detailed instructions provided)
4.7 Interactive Model
AMCP: Academy of Managed Care Pharmacy
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AMCP Dossier Format Version 3.1
What’s In the Dossier? (8 of 8)
Section 5.0: Other Supporting Evidence
 5.1 Summaries of Other Relevant Evidence (≤2 pages/study)
 Includes retrospective studies, comparative observational studies,
studies of patient adherence/persistence
 5.1.1. Summarize relevant economic studies not covered elsewhere
Section 6.0: Supporting Information
 6.1 References
 6.2 Appendices: Prescribing information, CER addendum, REMS
program documents, etc.
AMCP: Academy of Managed Care Pharmacy; CER: comparative effectiveness research
REMS: Risk Evaluation and Mitigation Strategy
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Economic Models In the AMCP Dossier
• Section 4 of the AMCP Dossier Format requests a
descriptive narrative and an interactive model
(typically Excel-based)
– Allows healthcare systems to apply and evaluate internal
data (ie, membership, prevalence, cost estimates)
• Most common model types:
– Cost-effectiveness (assesses overall clinical risk-benefit
and economic value of drug in relation to other available
drugs/treatments)
– Financial model (minimal approach: financial impact on
pharmacy budget only)
– Budget impact model
AMCP: Academy of Managed Care Pharmacy
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An AMCP Budget Impact Model—What’s In It?
Market Size: Number of eligible beneficiaries
diagnosed with and treated for the disease
Market Distribution: Forecasted market share distribution
of relevant comparators, including the new drug
New Treatment Costs: Impact of new treatment on service delivery; for
example: cost per patient/per claim/per-member-per-month
Transparency: Model inputs, assumptions should be clearly presented, cited
Budget Impact: Estimate all direct costs for existing vs new treatment
scenarios; present incremental cost savings with new vs reference scenario—
apply a specific and relevant time frame, sensitivity analysis
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Issues With AMCP Dossier Economic Models
• A 2011 survey evaluated and compared the
perspectives of payers and pharmaceutical
manufacturers on AMCP Dossier version 3.0
– 31% of manufacturers did not include any economic
model
– An interactive economic model was only prepared by
2 out of 3 manufacturers
– Even in cases where models were provided, they were
not as user-friendly, clear, or transparent as payers
would have liked
AMCP: Academy of Managed Care Pharmacy
Medina RD et al. Evaluating the Value of Clinical and Economic Evidence-Based Information from Dossier Submissions
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Using the AMCP Format. AMCP 2011.
AMCP E-Dossier System
• AMCP supports an electronic platform to facilitate
dossier distribution between manufacturers and
health systems (Dymaxium)
– This secure, web-based platform allows formulary and/or
benefit design decision-makers to easily access, review,
and evaluate dossiers—in compliance with regulatory
standards for unsolicited requests
• Rapidly becoming the standard approach for AMCP
dossier dissemination; different manufacturer
subscription plans available (including free option)
AMCP: Academy of Managed Care Pharmacy
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Writing AMCP Dossiers: Practical Issues
AMCP Dossiers are a mix of more traditional medical
writing and health economics writing
• Highly structured document, based on a defined format; plain
formatting is a plus
• Length depends on number of indications and body of available
research, but typically ~50-200 pages [goal <100 pages]
• Generally a team project—
challenging for a single freelancer
(can be 400-500 hours’ work on
tight turnaround)
• Writers likely to be assigned
specific sections
• Requires close project
management
AMCP: Academy of Managed Care Pharmacy 29
The Dossier
Thank you!
It’s time for Q & A
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