US formulary dossiers

A US formulary dossier brings together a manufacturer's clinical and economic evidence for payer decisions. The AMCP format organises that evidence into a single reference document supplied in response to an unsolicited request.

Purpose and recipients

The Academy of Managed Care Pharmacy (AMCP) introduced its Format for Formulary Submission in 2000, providing consistent, structured product information for cross-product coverage and reimbursement evaluations. ¹ By 2016 the format had undergone several updates, and requesting agencies could customise it. ^{1, 2} It standardised clinical and economic information supplied by manufacturers to US payers and formulary decision makers following unsolicited requests, with comparisons of effectiveness, safety and economic value against competitors particularly important. ^{3, 4}

Recipients in 2016 included national and regional insurers, managed care organisations, pharmacy benefit managers, hospital systems and state Medicaid agencies. ^{2, 5} Pharmacy and therapeutics committees or medical-technology review committees evaluated dossiers for access and reimbursement decisions. ² The dossier answered committee questions in a single comprehensive reference document; other payer dossiers often requested similar information. ^{6, 2}

Under the 2016 account, a dossier could include information outside the product's approved label only in response to an unsolicited request, because federal law prohibited commercial promotion of off-label uses. ²

Six sections

The AMCP dossier had six sections in the 2015 and 2016 descriptions. ^{7, 8}

1. Executive summary: the product's clinical and economic value.
2. Product information and disease description.
3. Supporting clinical evidence: summaries of key studies.
4. Economic value and modelling report.
5. Other supporting evidence: for example, published and unpublished studies supporting labelled and off-label indications, with evidence-table spreadsheets for all studies.
6. Supporting information: for example, cited references and economic models.

^{7, 8}

In 2015, an AMCP dossier commonly ran to about 100 pages and, unlike several international assessment formats, did not require a systematic review. ^{7, 3}

Clinical and economic evidence

Key information included clinical, real-world outcome, quality-of-life and cost data. ² Trial results commonly supplied efficacy and safety; observational studies or medical databases supplied real-world effectiveness, and patient-reported outcomes supplied health-related quality of life. ²

The economic report could include a cost-effectiveness model comparing two products' costs and health outcomes, or a budget impact model estimating the effect of adding the product to a payer's formulary. ² Budget models projected per-member per-month costs from drug costs, healthcare cost offsets, adverse-event costs and expected use in the covered population. ² Manufacturers could provide a spreadsheet so committees could run analyses with their own internal data. ²

A hepatitis C example using Sovaldi illustrates committee questions and the evidence answering them. ^{9, 4}

1. What are unmet needs, current treatment practices, safety and efficacy? Phase 3 studies, hepatitis C and advanced-liver-disease guidelines, market research and claims analysis inform the answer.
2. How prevalent and severe is hepatitis C within the plan population? Epidemiological studies, registries and claims analyses show the affected population and treatment needs.
3. What drives treatment costs? Cost-of-illness studies consider direct costs such as visits, tests and treatment, and indirect or intangible costs such as reduced productivity, absenteeism and reduced quality of life.
4. How would adopting Sovaldi change medical-service use and direct costs? Phase 4 observational studies, retrospective claims analyses and predictive models using phase 3 data can project resource utilisation.
5. Is Sovaldi cost-effective against standard therapy? Cost-effectiveness studies can give cost per cure, while cost-utility studies give incremental cost per quality-adjusted life-year gained.
6. Is it affordable? Budget impact modelling estimates net costs against current management, for example for 100 patients with advanced liver disease prescribed Sovaldi.

^{9, 4}

Development and the writer's role

Dossier preparation typically involved medical writers, health economists, a project manager and others, with cross-functional contributions from medical-team and regulatory leaders. ^{6, 10} Some companies had staff dedicated to payer deliverables, including writers. ¹⁰ In 2016, development was described in three stages. ¹⁰

1. Strategic planning. Establish the value proposition, available evidence, gaps such as a needed literature review, responsibilities, processes and timelines. Writers can help with timelines, key sources and articulating value with the team.
2. Content development. Produce several drafts for team and senior-management review, tailoring to the requesting agency and reusing existing company material where possible. Keep the main evidence succinct and details in appendices. Writers can conduct the literature review, select key evidence, draft and revise, and address reviewers' comments.
3. Final review and approval. Senior executives review the final document; writers can address approvers' comments. Once approved, the dossier can fulfil unsolicited payer requests.

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