The AMCP Format for Formulary Submissions. Version 3.0 Summary of Revisions

Transcription

1 The AMCP Format for Formulary Submissions Version 3.0 Summary of Revisions Like Version 2.1 of the AMCP Format, which was approved by the AMCP Board of Directors and released in April 2005, Version 3.0 is part of an ongoing effort to issue contemporary evidence standards and to address user comments and concerns. Since the release of Version 2.1, FMCP staff has collected numerous comments from managed health care systems, PBMs and the pharmaceutical industry. A formal call for comments netted over 50 pages of constructive recommendations for changes. The majority of comments came from health outcomes experts within pharmaceutical companies although a few health plans did submit comments. As opposed to comments received in advance of previous revisions, recommendations were largely supportive and extremely detailed. Based on these recommendations, the growing emphasis on comparative effectiveness research and the rapidly changing face of health care delivery, it was determined that the AMCP Format needed a major overhaul. Many of the comments were incorporated into the new version and then vetted by the FMCP Format Executive Committee and other experts in managed care and the pharmaceutical industry (see Acknowledgements). The AMCP Format is now in its 10th year and has improved the content and flow of evidence between industry and payers in the US. The quality of the dossiers remains an issue. Plans are warning that if the evidence dossiers are hijacked by commercial interests in the companies, they will not use them. FMCP staff and Format Executive Committee members continue to hear sporadic reports that health plans are receiving AMCP Format-based dossiers that they have not requested. Manufacturers and healthcare systems need to exercise greater vigilance to ensure strict adherence to FDA s unsolicited request requirements. Summary of general changes: 1. Updated the Acknowledgements 2. Sections were renumbered 3. Added an Executive Summary in lieu of Section 4 Product Value and Overall Cost 4. Added Section 5 Other Supporting Evidence 5. Made significant changes to other sections with the goal of clarifying requirements, improving usability and removing redundancies 6. Revised page limits 7. Removed requirement for print copies of relevant studies but manufacturer must supply upon request and provide links to free references 8. Added revised P&T committee monograph template to Appendix 9. Added to the Appendix a checklist to guide formulary decision making developed by the University of Illinois-Chicago CERT with funding from grants from the Attorney General Consumer and Prescriber Education Grant 1

2 Program (Principal Investigator, Gordon Schiff, MD) and AHRQ (Principal Investigator, Bruce L. Lambert, PhD) Preface and Introduction: 1. Deleted all of the Introduction 2. Retained a few sections of the Preface 3. Added section on Unsolicited Requests 4. Clarified the issue of manufacturers preparing multiple dossiers to comply with other organization s guidelines, e.g. WellPoint and Regence New Section 1.0: EXECUTIVE SUMMARY 1. Based on numerous recommendations, this section replaces Version 2.1 s Section 4 Product Value and Overall Cost but serves the same purpose as the principal opportunity for a manufacturer to communicate the value of its product. However, specific content, format and page requirements were added. New Section 2.0: PRODUCT INFORMATION AND DISEASE DESCRIPTION 1. Section 1.1, Product Information: Verbatim language from the Package Insert (PI) should not be supplied here. If there is not substantive data and information that can be provided beyond what is in the PI, these sections should be left blank and the reader referred to the copy of the PI which is in the Appendix. Basic product information should be included (appropriate sections of the PI including a brief discussion of what the product is, and any significant attributes that define the products place in therapy (e.g. kinetics, AE profile, etc.). In these situations, there should be more information provided that what is in the PI. The PI should not be included in the dossier, but in the appendix. 2. Included WAC and ASP. AWP is becoming less important. 3. Section 1.1.g and h. Included requirement for a listing of approved indications, as well as significant off label uses and potential new indications being studied for the drug. 4. Section 1.1. n, Deleted requirement for Market Share information 5. Section 1.1.o, Further defined Co-prescribed/concomitant Therapies to include if the drug should be used in combination with other agents or treatments. 6. Section 1.1.p, Revised to indicate that this is a comparison of PI information. If direct head to head trials have been performed on the drug and its comparators, it should be so noted and the reader referred to the discussion of these trials in Section 2 of the dossier. 7. Section 1.2.1: In general, keep disease description short and concise just enough to give reader a good overall sense of disease. Not necessary to go all the way to 4 pages if it can be accomplished in less. 8. For multiple indications, create separate 2.2 sections for each indication. However, some indications can be considered one larger indication, e.g. use for induction for disease X, and also indicated for maintenance 9. Stressed the importance of describing how the plans are to use the product in the appropriate subpopulations, not just overall population of a disease, e.g. patients with very high cholesterol vs. all patients with any elevation of cholesterol. Put another way, manufacturers should describe the percent of the total population that is represented by the subpopulation where the therapy is most effective for the indication. 2

3 10. Created a new section, for treatment guidelines, consensus statements, etc. This new section is to describe the guideline s position on the therapy. Include international bodies, e.g., NICE e Included requirement for description of whether expected outcomes are cure or disease management; palliative; or relief of symptoms, etc. Describe the clinical evidence to show certain clinical markers are linked to disease outcomes, e.g. LDL lowering -> outcomes f Included requirement for Description of REMS or other risk management strategies such as registries, restricted distribution channels, etc. 13. Clarified language in Section 2.3 on pharmacogenomics, New Section 3.0 SUPPORTING CLINICAL INFORMATION 1. Cleaned up wording to improve consistency and clarity (e.g., what does the word pivotal mean?). 2. Provided detailed guidance on content, length and format of narrative summaries and evidence tables of clinical and economic studies 3. Created new subsection for unpublished data and clinical studies supporting off-label indications. For unpublished data that are not otherwise accessible, added requirement for inclusion of as much detail as may be disclosed. 4. Included requirement that references to secondary literature sources such as Cochrane or AHRQ reviews and clinical guidelines should be brief - no more than one page. Also added statement that links to the original sources, if they are free, are sufficient in lieu of printed copies. 5. Included relevance criteria for inclusion and exclusion of studies. For drugs that have a large number of studies, dossiers are to include only the most significant ones and just list others. 6. Instead of print copies of clinical and economic studies, dossiers should include citations for all known relevant published studies in the bibliography section. Reprints of relevant published studies should be available upon request. Where possible, manufacturers are to provide a link to original sources if they are free. 7. Dossiers are to list all trials that are currently underway and document that they are listed in New Section ECONOMIC VALUE AND MODELING REPORT 1. Changed Section heading from Modeling Report to Economic Value and Modeling Report to reflect availability of economic data from a variety of sources 2. Provided brief guidelines for the conduct and reporting of RCT-based economic evaluations, (Rely on ISPOR guidelines) 3. Clarified the following: definition of budget impact model (BIM) and costeffectiveness analysis (CEA) models, model type requirements, model perspective, requirement for provision of a model, process of provision of a model and level of accessibility within a model a. Changed Section 3 heading from Modeling Report to Economic Value and Modeling Report to reflect availability of economic data from a variety of sources 3

4 b. Provided brief guidelines for the conduct and reporting of RCT-based economic evaluations, (Rely on ISPOR guidelines) c. Cleaned-up section with thorough re-write d. Clarified that patient-reported outcomes (other than utilities) should be reported in clinical section. e. Provided structure for reporting model i. Overview: population, perspective, time horizon; Structure; Explicit assumptions, Clinical inputs; Cost and utility inputs; Base-case results; Sensitivity analyses (incl. key drivers); Discussion; Implications ii. List all relevant RCTs, and if data from some trials not used, explain iii. Continue to emphasize Table 1 inputs, Fig. 1 structure, List 1 of assumptions, Table 2 results, Fig. 2 sens. Analyses f. Described transparency g. clear description of structure, inputs, results; access to model 2. Definition and requirement of model types a. Clarified definition of budget impact model (BIM) and cost-effectiveness analysis (CEA) model i. Financial model: assessment of drug cost only ii. Disease-based BIM: drug cost, ADE costs, and disease offset costs iii. Disease-based CEA: healthcare costs and health outcomes, including costeffectiveness, cost-consequences, and cost-utility analyses. iv. Explain pros and cons of different approaches b. Clarified model type requirements i. Require or strongly recommend disease-based CEA ii. Consider provision of BIM, acknowledge potential usefulness (cite ISPOR guidelines) iii. Note that drug cost-only financial models do not address clinical or economic value, and thus are not relevant for the AMCP Format 3. Modeling methods a. Clarified that more than one perspective can be used (e.g., payer, societal), but payer perspective should be included. b. Timeframe could also be variable, i.e., 5-yr, 10-yr, and lifetime. c. Discounting should be applied at 3% for costs and effects, but undiscounted results for clinical events and life expectancy are also useful. d. Briefly note strengths/weaknesses of analysis types (e.g., CEA vs. CUA), but don t provide recommendation e. Briefly note strengths/weaknesses of model types (e.g., decision analysis, Markov, DES), but don t provide recommendation 4. Model provision and accessibility a. Clarified requirement for provision of a model i. Emphasize that provision of an analysis of the clinical and economic value of a product is fundamental to the Format process ii. Require explanatory statement if a model is not provided 4

5 b. Clarified process for model provision i. Strongly recommend delivery of a model to users for use on their own time ii. If concerns exist about unsupervised use of the model, arrange a meeting between user and manufacturer to demonstrate model use before delivery of model iii. Strongly discourage limited access to a model (e.g., only when supervised or analyses run by manufacturer only), noting this raises significant barriers to model validation. c. Clarified level of accessibility within a model i. Should be user-friendly software, spreadsheet software is recommended ii. *All* inputs must be modifiable iii. All formulas/equations driving the model should be accessible within the model iv. In cases where complex software or modeling approaches are used, key risk equations that drive the model must be provided in text format as part of the model. 4. Software code that is not a component of the model is not required to be accessible. New Section 5.0: OTHER SUPPORTING EVIDENCE 1. Summaries and evidence tables for relevant economic studies are removed from the Modeling Section and included in this new section. This section will also include other types of non-rct supporting evidence such as meta-analyses and systematic reviews of clinical, quality of life and economic outcomes, comparative observational studies of effectiveness and harms, assessments of adherence or persistence, studies Emphasized similarities of economic data requirements to other health technology assessment bodies requirements. 5

6 6