The Academy of Managed Care Pharmacy is spearheading a renewed push for federal policies allowing biopharmaceutical manufacturers to share off-label economic data on drugs with formulary managers and is also urging a more expansive view of what that data can include.

The organization released a set of recommendations that seek to clarify Sec. 114 of the FDA Modernization Act of 1997. The provision was meant to serve as a regulatory safe harbor for dissemination of health care economic information but manufacturers have been reluctant to take advantage of it because they lack guidance on what kind of claims and supporting data FDA would consider to be acceptable ([A#14150422003]).

In the nearly 20 years since Sec. 114 went into effect, FDA has yet to release guidance on what is permissible under the rule, despite numerous requests from industry. Some issues were addressed by the House-passed 21^st Century Cures legislation ([A#00150504012]).

But industry was hoping for more extensive changes to the law, and it is unclear whether the counterpart bill in the Senate will tackle Sec. 114 issues.

Payers are on board with the campaign to clarify and broaden Sec. 114 because they have a growing need for health care economic information that can help with the management of costly new therapies, AMCP points out.

“Effective communication of [health care economic information] is more important than ever, as biopharmaceuticals entering the market today are highly complex and often very expensive,” AMCP CEO Susan Cantrell said in a release.

“With the US health care system increasingly focused on the concept of providing value, it is essential that pharmaceutical product value is accurately measured and disseminated. These recommendations are a major step forward in addressing a barrier that has prevented health care decision makers from obtaining such information.”

The recommendations were developed with input from pharmaceutical manufacturers, managed care firms, health care providers and policy experts at a “consensus forum” held in early March. As a next step, the recommendations will be reshaped as a draft guidance and offered to FDA and Congress as the basis for possible future action. Biopharma companies provided funding for the forum and development of the recommendations.

As written, Sec. 114 allows manufacturers to proactively disseminate health care economic information to a “formulary committee or other similar entity.” The information must be “based on competent and reliable scientific evidence” and “shall not be considered false or misleading ... if the information directly relates to” an approved indication.

Some of the AMCP recommendations target changes and clarifications that can be made by FDA through guidance and regulation and others would require congressional action to achieve.

Defining Key Terms

Among the proposals aimed at FDA is one that asks that the definition of “health care economic information” be expanded beyond cost to encompass “any analysis that identifies, measures, or compares the economic, clinical or quality of life consequences for any treatment. This includes the cost and resource utilization of a drug or health technology relative to another drug, health technology or no intervention.”

Many of the suggestions for what constitutes sharable economic data seem like they could also be endpoints for clinical trials (see box).

The Economics Of Health Care Data

AMCP's recommendations for what should constitute economic data that can be shared with payers include information that might also be considered clinical endpoints, such as:

Hospitalizations
Emergency department visits
Adherence
Quality of life
Adverse events

In addition to formulary committees, “other similar” entities receiving the information should include organizations “who make health care decisions for patient populations,” such as payers, accountable care organizations, integrated delivery networks and physician practices involved in risk-sharing arrangements, as well as organizations that evaluate health care economic information or develop value frameworks and compendia, according to the proposal.

AMCP argues the “competent and reliable scientific evidence” standard should not translate into a requirement for clinical trials, an issue raised by the Pharmaceutical Research and Manufacturers of America in a 2012 white paper on the law ([A#00121015019]). Instead, the evidence should be defined as “truthful and non-misleading tests, analyses, research studies, models or other evidence,” AMCP states.

”Such evidence would be based on the expertise of professionals in the relevant area and be derived using methods that are transparent, disclosed, reproducible, accurate and valid,” AMCP explains.

The statement that information appropriate for dissemination “directly relates to an indication approved” should be interpreted to cover “information about a product that may vary from the parameters utilized in a randomized controlled trial, such as dosage forms, settings, or populations studied” as long as it is still used within the approved disease indication, AMCP says.

Info Needed 18 Months Before Approval

AMCP believes the statement “directly relates to an indication approved” should be modified by legislation to state only that information “relates to an indication approved.” Such a change was included in the House Cures bill.

“’Relates’ can also mean a drug or other health technology indication that is not specifically stated in the label,” AMCP points out. However, it adds, “claims regarding intended indications versus approved indications would have to be specifically addressed by the FDA to provide clarity on what is permissible under this part of Sec. 114.”

Congress should consider providing health care economic information to patients, possibly through advocacy organizations with appropriate scientific expertise, AMCP suggests. The organization notes, however, there was concern among some forum stakeholders about providing information directly to consumers and ultimately “no consensus was reached” about the best approach to take.

The possibility of providing payers and other entities access to health care economic information for drugs in the pipeline 12 to 18 months prior to approval is also raised in the proposal. “Early dissemination … would allow payers to build this information into forecasting and premiums, since waiting until approval is often too late,” AMCP explains.

Attendees at the forum “agreed these are important areas of possible expansion for safe harbor, but it may not be within the spirit or original intent of Section 114,” the group notes.

“Therefore, further discussion in this area as to how other laws or regulations, such as expanding scientific exchange provisions, could be amended to provide this types of access to [health care economic information] for [patient advocacy groups] and pipeline medications are warranted.”

Rx Economic Information-Sharing Concept Getting Renewed Push

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