The Food and Drug Administration (FDA) recently published guidance that clarifies how pharmaceutical manufacturers can share product information with payers while remaining compliant with FDA rules on communication. The FDA aims to promote value-based contracting through increased sharing of information, including off-label use of a pharmaceutical treatment. Clinical pathways have an opportunity to utilize this same information concerning use beyond the label as well as benefits and metrics that can assist patients, providers, and payers on most appropriate treatments to use in order to achieve greater value in terms of both clinical and financial outcomes. This is an opportunity for a more comprehensive and timely value-based clinical pathway.
Opportunities for value-based contracts just increased significantly with the recently published FDA guidance that clarifies how pharmaceutical manufacturers can share product information with payers while remaining compliant with FDA rules on communication.1 This guidance permits pharmaceutical manufacturers to provide payers with information about unapproved drugs or unapproved uses of already-cleared medicines. Pharmaceutical manufacturers can negotiate ahead of an approval decision to appropriately craft a value-based payment scheme.
FDA Commissioner Scott Gottlieb, MD, summarized the importance of the guidance in a statement: “This guidance will inform market participants developing contracts that include value-based arrangements [on] how to communicate information about how a drug might impact outcomes that are important to purchasers.”2 These efforts will encourage competitive contracting based on measures of value that matter most to stakeholders without getting in the way of competitive negotiations.
The big news here is that clinical pathway developers can now consider incorporating unapproved treatments as well as value-based metrics into their pathway guidance, which will be of significant benefit to providers, payers, and patients. This approach is consistent with other value-based care efforts by the Health and Human Services, by the Centers for Medicare and Medicaid Services (CMS) through the use of innovative payment models, and, most recently, by the FDA with this guidance. Clinical pathway developers have an opportunity to incorporate this guidance concerning use beyond the label as well as value-based outcomes into pathways to assist patients, providers, and payers on the most appropriate and highest value treatments.
FDA Guidance Breakdown
In communications with payers about unapproved products or unapproved uses of approved/cleared products, pharmaceutical manufacturers must be unbiased, factual, accurate, and not mislead. Communications may include information about the indication(s) sought, such as information from the clinical study protocol(s) about endpoint(s) being studied and the patient population under investigation (eg, number of participants enrolled, their enrollment criteria, participant demographics). Additional information that can be communicated includes:
- Anticipated timeline for possible FDA approval/clearance/licensure of the product or of the new use.
- Product pricing information.
- Patient utilization projections (eg, epidemiological data projection on incidence and prevalence).
- Product-related programs or services (eg, patient support programs).
- Factual presentations of results from studies, including clinical studies of drugs or devices or bench tests that describe device performance (ie, no characterizations or conclusions should be made regarding the safety or effectiveness of the unapproved product or the unapproved use).
The FDA also recommends that pharmaceutical manufacturers provide the following information to payers when communicating information about unapproved products or about unapproved uses of approved/cleared/licensed products:
- A clear statement that the product or use is not approved/cleared/licensed and that the safety or effectiveness of the product or use has not been established.
- Information related to the stage of product development (eg, the status of any study[ies] in which a product/new use is being investigated and how it relates to the overall product development plan, whether a marketing application for the product or new use has been submitted to FDA or when such a submission is planned).
- For communications that include factual presentations of results from studies, FDA recommends that firms describe material aspects of study design and methodology and also disclose material limitations related to the study design, methodology, and results. Firms should also ensure that results are not selectively presented (eg, both positive and negative or null findings should be presented).
In addition, for communications with payers about unapproved uses of approved/cleared/licensed products, firms should provide a prominent statement disclosing the indication(s) for which the FDA has approved, cleared, or licensed the product and a copy of the most current FDA-required labeling.
The FDA also suggests that firms provide follow-up information to payers if previously communicated information becomes materially outdated as a result of significant changes or as a result of new information regarding the product (eg, failure to meet the primary effectiveness endpoint in the pivotal trial) or its review status (eg, an application is determined to not be ready for approval upon completion of the review cycle, or a study is placed on a clinical hold).
Also included in the final FDA guidance was clarity regarding use of health care economic information (HCEI). The FDA has said that HCEI analyses should relate to the disease or condition, the manifestation of the disease or condition, or symptoms associated with the disease or condition in the patient population for which the drug is indicated in the FDA-approved labeling. Table 1 provides examples of HCEI findings that the FDA believes could be considered related to an approved indication of a drug. This is information that could be incorporated into communications or pathways that does not appear within, and may vary in certain respects from, information presented in the FDA-approved labeling.
Value-Based Clinical Pathways
With this new guidance from the FDA, clinical pathway developers can further incorporate important elements into pathways that impact all key stakeholders, including providers, payers, manufacturers, and patients for value-based contracting. These elements include the impact of a pharmaceutical treatment on quality measures or total cost of care—metrics that both payers and providers are being held accountable to. This information is the basis of “why” payers are motivated to provide access or, in some cases, block or promote access.
In addition, this information helps translate the “what” of what critical elements of a treatment matter in terms of value. Articulating this value in terms that matter is essential to ensure that patients have the appropriate level of access and, as a result, achieve the greatest outcomes possible. A good illustration of these elements and the process for articulating value is shown in Figure 1. This illustration shows critical components in a value proposition in the areas of economic and clinical value, which, for payers, translates into lowering costs and increasing revenue through increased membership and quality measure improvement.
This process is, of course, not a one-and-done exercise, as clinical pathway developers have the responsibility of updating pathways as new findings become available. For example, if initial HCEI information supported an off-label use that had been previously incorporated into a clinical pathway but new information results in FDA denial of such a claim; such findings could mean that use would no longer be appropriate. Without changes/updates to the clinical pathway, users may not be utilizing the most current information. Also, if the HCEI information developed over time provides additional evidence of delivery on value-based metrics such as quality measures or total cost of care, for example, this new information should be added to an updated guideline and shared with all users. As new information becomes available through HCEI data at a much quicker pace than FDA label changes, clinical pathway developers will need to have a process for timely incorporation of changes and new information.
Conclusion
Value-based contracts have been talked about for some time without much traction. But now, with value-based care being promoted by CMS and adapted by other payers, and as the FDA allows for greater communication, it looks like the time for value-based contracting has finally arrived. As value-based care gains more momentum, resources like clinical pathways will need to provide their own value to all key stakeholders.
References
1. US Food and Drug Administration. Drug and Device Manufacturer Communications With Payors, Formulary Committees, and Similar Entities — Questions and Answers. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM537347.pdf. Published June 2018. Accessed July 5, 2018.
2. US Food and Drug Administration. Statement from FDA commissioner Scott Gottlieb, M.D., on new efforts to advance medical product communications to support drug competition and value-based health care [press release]. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm610415.htm. Published June 12, 2018. Accessed July 5, 2018.
3. The Access Group (TAG) Payer Value Story Construct [proprietary data]. The Access Group, Berkeley Heights, NJ.
