FDA officials are leaning toward a single document to inform patients about their medications, but whether and how that can be accomplished remain unanswered questions.

One document is "the logical proposal," Center for Drug Evaluation and Research Director Janet Woodcock said in addressing a Sept. 24-25 workshop convened by the agency to receive input on providing effective drug information to consumers.

Achieving that goal requires a collaborative effort by stakeholders, she noted. "We're all going to have to change the way we do things ... and that's going to be very complicated."

Currently, depending on a medicine's risk profile, patients could receive a Consumer Medication Information packet, a patient package insert and a Medication Guide when they pick up a prescription.

To simplify that literature regimen, FDA's Risk Communication Advisory Committee recommended during its February meeting that a single document be dispensed (¹ Also see "FDA Poised To Overhaul Distribution Of Consumer Medical Information" - Pink Sheet, 9 March, 2009.).

But FDA has many issues to resolve if it pursues that option - such as how to provide concise information about risks and benefits, standards for effective patient literature and the distribution mechanism.

"The path forward is not clear to us," Woodcock said. The purpose of the workshop is "to develop a clear path forward. If we can devise one, we will take that path."

Consensus Hard To Come By

To help FDA find that path, representatives of drug companies, pharmacies, consumers, CMI vendors and others spent a day in small groups critiquing four prototypes of a single consumer information document developed by FDA.

While the exercise was useful, there was a "vast difference of opinion" among participants, CDER Office of Executive Programs Director Deborah Henderson concluded in recapping the effort.

In fact, a number of things appeared on some participants' lists of the prototypes' strengths and on others' lists of the documents' weaknesses, she said.

Some Agreement On Benefits Info

There was consensus that "benefit" means what a drug does, rather than its indication, she said. As an example, she cited agreement that the benefit for the hypothetical drug discussed in the prototypes would be reduction of joint inflammation, rather than treatment of rheumatoid arthritis.

Whether it is possible to provide consumers with quantitative information that makes sense of multiple trials with different endpoints and patient populations was an issue raised by participants, she said.

Despite that concern, "there was consensus that this is an important thing to look at, that we need to have more research and more testing on our ability to explain to patients [their drugs'] effectiveness."

"Loose" consensus formed around the tenet that benefit information is absolutely necessary for patients to receive, Henderson said, while a slim majority thought:

Benefit information should precede risk data;

There should be a timetable for when the benefit is expected to occur;

Quantitative information should be provided on the severity and frequency of side effects; and

There should be directions for use and information on how to get more information.

The Academics' Approach

During a research-based panel discussion, Michael Wolfe, Northwestern University, suggested that to keep information manageable for patients, "need-to-know" information should be provided upfront. Patients can then be referred to other sources for a more comprehensive discussion of the medication, he said.

Such an approach avoids information overload, Almut Winterstein, University of Florida, agreed.

When developing a specific consumer leaflet, the most important information points can be identified through feedback with a small group of potential users of the medicine, Theo Raynor, University of Leeds, said.

Once a leaflet has been designed, patients' ability to understand and retain the information can be assessed in a larger study, he noted, although further testing may not be necessary for documents with content similar to that in a previous leaflet.

Henderson, in an interview, agreed that such "validation" testing is needed. The current MedGuides, patient package inserts and CMI were developed without good research on whether they work for patients, she pointed out.

"If we're going to spend our energies and our efforts developing information for patients, we ought to ask the patients whether it's useful and go out there and, on some periodic basis, evaluate whether it's meeting their needs. ... I feel certain that we would not go forward with putting anything new out there without doing some testing."

Before FDA staff can make any proposals to either tweak or completely overhaul the consumer information system, it must go through the workshop discussions, as well as comments to the docket, which closes Nov. 25.

The task likely will fall to the intra-agency multidisciplinary group that organized the workshop, according to Henderson, who expects Commissioner Margaret Hamburg and Principal Deputy Commissioner Joshua Sharfstein to be briefed on the workshop and potential next steps.

Improving patient understanding of their medications and how to take them can be important to public health, a driving issue for FDA under Hamburg and her deputy.

Although Henderson noted in opening the workshop that "we are working toward, or think we are working toward, a single document solution," she stressed during the interview that that is only one possibility for improving the system, and a number of options remain on the table.

CMI Review As Part Of NDA Approval

These range from guidance to vendors on how to improve the existing CMI, to having manufacturers propose the material for FDA review as part of its assessment of proposed labeling.

The latter option is supported by the Biotechnology Industry Organization, which favors a single document. FDA should set standards for presenting consumer information, providing a template for content and format, BIO Director for Science and Regulatory Affairs Andrew Emmett suggested during the workshop.

Manufacturers should draft the documents for their therapies, he said, pointing out that companies and FDA "have the most detailed knowledge of the risks and benefits of their particular products."

The consumer information document should be reviewed by FDA as part of the drug approval process and there should be a process for post-market revisions as well, he suggested.

Once approved by the agency, the document should be posted to an electronic database and "it shouldn't be altered on the way to the pharmacy."

Many biologics are administered by physicians, so the document could serve as a useful tool for discussing information with patients, he added.

Henderson told the workshop that using a single format for CMI could be complicated by the requirement for mandatory distribution of Medication Guides that are part of a Risk Evaluation and Mitigation Strategy. It remains unclear whether a single document is sufficient to inform patients about drugs with a serious risk, she noted.

An alternative is that a single document for a drug with a serious risk would be different than that for other drugs. "You might get only one thing, but it might be a different thing," she said.

The quality of consumer medication information has been as issue for some time. Congress in 1996 directed FDA to allow the private sector to provide CMI to patients and proceed to regulation only if goals were not met (² Also see "FDA REFORM ACTION DELAYED UNTIL SEPTEMBER AS NEGOTIATIONS CONTINUE OVER AUGUST RECESS; "MEDGUIDE" AUTHORIZED BY FISCAL 1997 FDA APPROPRIATIONS LEGISLATION" - Pink Sheet, 5 August, 1996.).

Those goals - that 95 percent of all new prescriptions be accompanied by useful CMI by 2006 - were missed, according to a 2008 survey. The study found that while 94 percent of prescriptions are accompanied by CMI, only 75 percent of the information meets minimum criteria for usefulness (³ Also see "FDA Risk Communication Advisory Cmte. Will Assess Pharmacy Leaflets’ Utility" - Pink Sheet, 22 December, 2008.).

That translates to 71 percent of patients receiving useful information about their medications.

- Cathy Dombrowski ( ⁴ c.dombrowski@elsevier.com )

Single Patient Drug Leaflet Logical, Complicated To Achieve - Woodcock

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