The 21st Century Cures Act and Drug Company Promotion of Real-World Data

A new discussion draft of the 21st Century Cures Act, the bipartisan initiative to accelerate development of new medical treatments, includes a section entitled “Facilitating Dissemination of Health Care Economic Information.” A previous version of the bill included only a placeholder heading for these issues. The section makes changes to Section 114 of the Food and Drug Modernization Act of 1997 (FDAMA 114), which regulates promotion of health economic information by pharmaceutical companies to formulary committees, such as a claim that a drug reduces health care costs in a given population.

The Food and Drug Administration (FDA) has never commented on Section 114 and, as we note in a new paper in which we develop hypothetical case studies for Section 114, the lack of guidance about what the original statute allows has hampered the exchange of information. Together, the proposed changes would add clarity and flexibility for pharmaceutical companies to engage with health plans about the potential value of their products, though it will require careful ongoing monitoring by regulators.

Improved Communication

The discussion draft contains a number of changes that, in our view, would help foster better communication between drug companies and payers about the real-world impacts and economic consequences of using pharmaceuticals.

The existing language of Section 114, while vaguely worded, seems to restrict the promotion of health economic information only to information that drug companies collect in randomized clinical trials (RCTs), called “substantial evidence,” rather than information using “real-world” data. For example, it is unclear whether and in what circumstances a company could promote a study using an observational database that analyzes the costs of using the drug in real-world settings.

The new draft amends the clause requiring that a promotional claim “directly relate[s]” to the FDA-approved indication to the less restrictive term “relate[s].” The term, “relates,” would still require clarification, ideally in a future FDA guidance, but it suggests more flexibility around the type of health care economic information (e.g., from database analyses or economic models) that drug companies could provide proactively (as opposed to in responses to unsolicited requests) to health plan audiences.

For example, the “directly relates” language implies a strict standard — a company might not promote findings from a database study if the population differed, even in minor ways, from the population studied in the relevant randomized clinical trials. Under the proposed change, such an analysis might be deemed permissible, though again, the FDA will have to clarify the details.

The draft also adds a provision requiring, where applicable, pharmaceutical companies to provide “a conspicuous and prominent statement describing any differences between the information and the indication approved.” Such a disclosure (e.g., that a promotion is based on a database study, that the population under investigation might differ from those in RCTs, and that retrospective database analyses demonstrate associations but do not indicate causality) should increase transparency and help ensure that health plan audiences are informed and aware that the health care economic information being promoted is not necessarily based on substantial evidence from randomized clinical trials.

Furthermore, the existing statute provides a narrow definition of “health care economic information” regulated by the Section which limits the possible types of economic analyses permitted. The draft expands the definition to incorporate inputs and methods, including “clinical or other assumptions.” Because all health care economic analyses include clinical assumptions, this language would allow a much broader array of health economic analyses to be included, though again FDA clarity will be important.

Finally, the draft clarifies the permitted audience for health economic promotions. These promotions are currently limited to formulary committee members and the undefined phrase, “similar entities.” The draft includes “payor[s]” in addition to the “formulary committee[s], or other similar entit[ies]” covered by the existing statute. That is important because the original statutory language, from 1997, did not state whether payers would be included, although the draft still does not define what is meant by “similar entity” (e.g., does it include physicians working and making decisions for accountable care organizations?).

Preserving Guardrails For Public Health

Critically, the changes would continue to restrict these types of promotions to organizations (i.e., formulary committees and payers), not to general prescribers and consumers.

As we’ve argued before, that would “preserve key guardrails for public health. Promotion would be restricted to organizations that retain strong incentives to be informed and wary consumers of drug-company promotions and that increasingly employ their own experts, mine their own data, and request comparative effectiveness evidence from companies.”

It would confer upon health plans the responsibility to judge the usefulness and credibility of a wider range of health economic claims promulgated by drug companies, therefore providing more data to payers to inform coverage decisions.

As noted, several other conditions must hold if the new legislation is to be successful. First, FDA will need to clarify what types of clinical claims (either explicitly made or implied) are permissible in health care economic information.

An amendment posted to the discussion draft would require the Department of Health and Human Services (HHS) to “issue draft guidance on facilitating the dissemination of responsible, truthful, and non-misleading scientific and medical information not included on the label of drugs.”

Second, ongoing work is needed on standards for observational data and other real world studies. Third, it will be important for the FDA to monitor the field to ensure that health care economic promotions are not false and misleading.

This will be a challenging area for FDA to regulate. For one, various court cases surrounding drug companies’ promotion and First Amendment rights have raised issues about the FDA’s ability to regulate truthful, non-misleading claims. For another, it is challenging for FDA to know what types of promotional claims will be made under the new health economic promotion laws given that they are business-to-business communications.

The language in the Discussion Draft may continue to change as the bill works its way through the legislative process. Still, the proposal to date reflects a step in the right direction.