Developing Effective Academy of Managed Care Pharmacy Dossiers Caitlin Rothermel, MA, MPH 1 The...

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Developing Effective Academy of Managed Care Pharmacy Dossiers

Caitlin Rothermel, MA, MPH

The Dossier

Breakout Group

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Dossiers In Value Story CommunicationA 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?

3Barrows S. ISPOR 14th Annual International Meeting, May 2009.

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

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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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Value Message Development Exercise: Vivglucor™

• Break into up to 3 groups to develop draft value messages for this drug (3-5 statements)

• Develop value messages targeted to the following stakeholders

– Prescribing Physicians

– Patients

– Payers

– Regulatory Agencies

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• A new drug to treat type 2 diabetes• Interacts with gut biome to alter bacterial composition,

improve metabolic syndrome symptoms, and reduce blood glucose levels

• Mean 2-year sustained A1C reduction in clinical and real-world trials, -1.75%

• Ongoing cardiovascular trials (2-year data) shows no negative signal; drug also lowers blood pressure (~9 mm Hg systolic)

• Associated with ~5 kg weight loss, sustained at 2 years• Adverse events: moderate hypoglycemia, constipation, and/or

flatulence at treatment initiation, typically resolves in 3-6 months

• Weekly rectal administration/infusion (either by trained professional or patient partner)

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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

AMCP: Academy of Managed Care Pharmacy

– 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

Caitlin P Rothermel

Define MCOS

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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 SullivanUniversity of Washington

AMCP: Academy of Managed Care PharmacySullivan 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)

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

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)

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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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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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Big Table, Example

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Section 2.0: Place of Product In TherapySubsections 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


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Section 2.2.2, Sample

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Section 3.0: Supporting Clinical EvidenceSubsection 3.1: Summaries of Key Clinical Studies Supporting Labeled Indications (≤2 pages/study)

a. Name of the clinical trial or study and publication citation(s)b. Study objective, location and date(s)c. Trial design, randomization, and blinding proceduresd. Setting, inclusion, and exclusion criteriae. Patient characteristicsf. Dropout rates and procedures for handling dropoutsg. Treatment, including dosage regimens, washout periodh. Clinical outcome(s) measured (delineate primary vs. secondary endpoints)i. Other outcomes measured (e.g., patient-reported outcomes) and principal findingsj. Statistical significance of outcomes and power calculationsk. Validation of outcomes instruments (if applicable)l. Generalizability of the population treatedm. Study limitations, as stated by the authors


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Section 3.1.1, Sample

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Section 3.0: Supporting Clinical EvidenceSubsections 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: Center for Drug Evaluation and Research; FDA: Food and Drug Administration; HTA: Health Technology Assessment

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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


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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 modelAMCP: Academy of Managed Care Pharmacy

What Are the Most Common Types of Health Economics Models/Analyses Relevant to Dossiers?

Cost-effectiveness Measures costs in dollars and reports outcomes in natural health units (e.g., mmHg reduction) or ratios (differences in cost/difference in outcomes)

Cost-utility Measures outcomes based on years of life and quality of life obtained with treatment

Cost-benefit Enumerates and compares costs and benefits achieved in monetary terms

Budget impact Estimates effect of intervention on overall cost to organization or health plan

Berger et al. ISPOR Book of Terms. Health Care, Cost, Quality, and Outcomes; ISPOR, 2003. Rascati KL. Essentials of Pharmacoeconomics. Wolters Kluwer, 2011.

Cost-effectiveness Analysis and Incremental Cost-effectiveness Ratio (ICER)

Bonk RJ. Conversance with Fundamentals of Health Economics. AMWA Pocket Training.Rascati KL. Essentials of Pharmacoeconomics. Wolters Kluwer, 2011.

• Used in cost-effectiveness and cost-utility analyses• Answers the question, “How does one treatment

compare with another in terms of costs and outcomes?”– Incremental costs are costs incurred for treatment excluding up-

front investments (eg, diagnostic instruments)

• The ICER is the ratio of the difference in costs, divided by the difference in outcomes

• If the ICER calculation produces negative numbers, one treatment is dominant (more effective and less expensive); the other option is then dominated by this treatment

• More often, the outcome it is not so clear cut

Cost-utility Models, Key Concepts


• A cost-utility analysis (CUA) takes patient preferences, commonly known as utilities, into consideration when measuring health consequences

• Typically applies a special unit of measure, the QALY (quality-adjusted life year)

A QALY combines, in a single measure, gains or losses in both quantity of life (mortality) and quality of life for each treatment option

Measured on a scale of 0 (death) to 1 (perfect health); typically reported as “cost per QALY obtained”

The use of this single, common measure enables comparisons of outcomes across multiple studies, and even multiple disease states

QALYs are most applicable to research or population-based decision-making; they do not translate very well for day-to-day decisions

Cost-benefit Analysis, Key Concepts


• All outcome measures are valued in monetary units • Very useful for health program comparisons; one

treatment approach can be compared directly to an alternative (including “do nothing”)

• Allows for comparison of programs / interventions with entirely different outcomes

• If the net benefit of the intervention is positive, the intervention should be funded

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Budget Impact Model—What Is It?

• A budget impact model (BIM) estimates the impact of an intervention on overall cost to a health plan or organization

• Typically, it measures the net cumulative cost of a specific treatment for a given number of patients in a specific population—likely to use the “payer perspective”– Example: How much will it cost our organization to treat patients

with recurrent multiple myeloma, using Drug A vs Drug B?

• BIM is a common modeling approach for payer audience—the format and presentation can take many forms


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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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• A new drug to treat type 2 diabetes• Interacts with gut biome to alter bacterial composition,

improve metabolic syndrome symptoms, and reduce blood glucose levels

• Mean 2-year sustained A1C reduction in clinical and real-world trials, -1.75%

• Ongoing cardiovascular trials (2-year data) shows no negative signal; drug also lowers blood pressure (~9 mm Hg systolic)

• Associated with ~5 kg weight loss, sustained at 2 years• Adverse events: moderate hypoglycemia, constipation, and/or

flatulence at treatment initiation, typically resolves in 3-6 months

• Weekly rectal administration/infusion (either by trained professional or patient partner)

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Model Development Exercise: Vivglucor™

• Based on your understanding of economic models, what kind of model would be best to use for this product? Discuss the pros and cons of:

– Cost effectiveness

– Cost-utility

– Cost-benefit

– Budget Impact

Writing AMCP Dossiers: Practical Issues

• 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]

AMCP: Academy of Managed Care Pharmacy 29

• 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 Dossiers are a mix of more traditional medical writing and health economics writing

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Thank you!

The Dossier

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