Last summer, Congress enacted the Food and Drug Administration Safety and Innovation Act (FDASIA), which included the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). We reported in April that FDA will be meeting with patients to get their perspectives about 20 disease areas during the course of PDUFA V (2013-2017).
In addition to these meetings, FDA recently announced a new way to communicate and work with patients—FDA’s Patient Network, run by FDA’s newly named Office of Health and Constituent Affairs (formerly the Office of Special Health Issues). Richard Klein, director of FDA’s Patient Liaison program within this office noted that FDA needs help from patients, and will get such insight and information through the new Network.
FDA has a long history of working with patients and patient advocates representing specific groups, beginning with the AIDS crisis of the 1980s. Since then, FDA has reached out to advocacy groups, such as the National Council of Rare Health Disorders and other disease-specific groups, to ask for their input on proposed regulations and guidance documents.
FDA Commissioner Margaret Hamburg dedicated a post on FDA’s Voice Blog discussing how FDA will “help patients have a bigger voice” through this new Network. Hamburg stated that “When patients better understand the intricacies of how medical products are studied, reviewed, assessed and brought to market, their input will be that much more focused and valuable. We hope, with the launch of this new web site, to expand the role of patients beyond the select group of patient representatives and to engage a wider audience of patients in new and broader ways.”
According to project manager James Valentine, M.H.S., the Patient Network helps to bring the unique perspective of patients, family members, caregivers and patient advocates to the decision-making processes of the FDA. There are a number of ways to become involved, including becoming a patient representative on a committee that advises FDA on key issues. Valentine explained in an interview (see below) that FDA now has “over 170 active patient representatives who represent over 105 different disease areas, and that continued to grow,” and FDA has “almost universal participation of patient representatives on all FDA advisory committees for all medical products.”
He also referenced Section 1137 of FDASIA, which “mandates that [FDA] look into ways to expand patient representatives’ participation in agency sponsor meetings.” Valentine explained that while FDA has historically included patient reps in sponsor meetings, the agency will now “look at a systematic approach to implement it across all of the three medical product centers (drugs, devices and biologics).” One hurdle, however, is conflict of interest screenings, which may be difficult “given the short periods of time between when a sponsor requests a meeting and when it is scheduled.”
Accordingly, FDA will use the same process it does for Advisory Committee conflict screening to see if patients have made “any comments … in the public domain that would reflect a pre-decided position about a particular regulatory decision” that would prevent the potential patient representative from being objective.
Consequently, Valentine also noted that there is some difficulty finding “unconflicted” patient representatives from the rare disease communities who are increasingly involved in product development and clinical trials. As a result, he noted that FDA “tries to be flexible, and grants waivers in situations where there is no other alternative to the person in terms of their level of expertise or conflicts.”
Valentine explains that the three-phase program began in 2011 with the launch of the twice-monthly newsletter Patient Network News. A recent issue includes information on a product that has been linked to potentially serious eye infections; an upcoming public meeting on breast cancer; a story on active ingredients in children’s medicines; and information on pet health. “It’s a good way to find out just what FDA is up to—and in ways that are relevant to you, the individual,” Valentine says.
The annual Patient Network meeting, the second of which will take place this summer, is the second phase. “We’re gearing up for it now,” Klein notes.
By popular demand from patient organizations, Klein says, the meeting will focus on “demystifying the FDA, by providing a basic primer about medical product regulation.” “We need to demonstrate that from both FDA’s and industry perspectives medical product development isn’t a simple 1-2-3 recipe,” he adds.
FDA launched the Patient Network site to engage patients and consumers as partners, and encourage them to share ideas and concerns about the medical product development process. “When it comes to FDA’s regulatory process, we feel very strongly patients should have a say,” notes Klein. “The new website provides an excellent place to start.”
According to Klein, consumers have complained that they find it difficult to locate the information they need on the agency’s website, and when they do, it is targeted for industry use. “This website provides a concentrated, one-stop forum from which we can reach patients and vice versa,” Valentine says. The website connects patients, caregivers, and advocacy groups to the agency through
- e-mail,
- webinars,
- live chats with senior agency officials.
- open meetings and a calendar of such meetings
- announcements of opportunities for patients to comment on FDA regulations
- reporting side effects on MedWatch, which Valentine said FDA is working on creating a “consumer form” of adverse event reporting
There are sections on what FDA does, FDA advisory committees, FDA initiatives, and frequently asked questions, and a video explaining the role of the new Network. The website also helps patients, caregivers, and advocacy groups find information on:
- getting involved in the regulatory process;
- finding relevant clinical trials, including helping to expand access to investigational products through FDA’s “compassionate use” rules;
- finding additional treatment options;
- getting information about diseases, including Cancer, HIV/AIDs, Diabetes, and Cardiovascular; and
- Getting information about their medications, including whether they have been issued a warning label or recalled from the market, whether there are other, off-label uses of the drug, or if anyone has had an adverse reaction to their medication.
On the separate page designed for “additional treatment options,” FDA discusses “off-label use of approved drugs” and notes several questions patients should ask their doctors about such uses:
- Is there information available to support the off-label use of this drug to treat my condition?
- Is this off-label drug likely to work better than an approved drug?
- What are the risks and benefits of off-label treatment with this drug?
- Will my health insurance cover off-label treatment with this drug?
Designing the Patient Network Website
“We’ve worked hard to make it easily navigable and user friendly,” Valentine says. RAPS interviewed James Valentine about the Network and its importance. Valentine reiterated the goal of the new website: Getting patients “informed” and “involved” to “help FDA help patients have a bigger voice.” The website gives patients a “home” for getting educated about FDA regulated products.
He noted that while some patients will use the site for information, FDA hopes that others will become “advocates for their patient community and that it will allow them to provide FDA with their unique patient perspective, allowing [FDA] to make better-informed decisions with that perspective.” Valentine emphasized that the site will facilitate “a broader amount of patient input and participation in current mechanisms and evolving mechanisms for patient input.”
To help make the Patient Network website user friendly and easy to navigate, FDA engaged patients and patient advocates through focus groups to figure out what patients needed from FDA. The agency then did “usability testing to make sure that the site really worked for them and contained what they wanted.”
With respect to training and educating patients, Valentine emphasized that the “core educational content of the website” is focused on “the role that industry plays in drug development, that FDA plays in development, and how they intersect.” As he pointed out, the Network website for the first time “put those complex processes into a clear and step-by-step self-guided explanation that doesn’t require that patient to get FDA on the phone to explain it to them.”
For example, patients can “Learn how Drugs and Devices Get Approved” and the site takes them through the following steps (with easy to navigate buttons) using simple, easy to understand language:
- Discovery and Development
- Preclinical Research
- Clinical research (including valuable descriptions of Phase I through Phase IV clinical studies
- FDA review
- FDA Post-Market Safety Monitoring
Valentine said that FDA plans to “continually assess the content of the website and to add to it and change it so if we find people are asking common questions, we can build more content to meet those needs. If people do have individual, specific issues, our goal is still to connect those people with our front-line patient liaisons” he added.
With respect to patient or patient community input, he noted that information will vary.
“For example, some patient groups don’t have any approved therapies and they’re trying to advance the regulatory science around their disease area to get outcomes that can be used by industry to develop products; others may have many therapies, and may be more interested in the safety profiles of products.”
He added that incorporating patient input into FDA’s regulatory decision is important, and that the agency is not necessarily “focusing on a specific product, but rather on the current state of the unmet medical need within a community and the severity of the disease so that reviewers across the agency can take those considerations into account, regardless of the product type coming down the pipeline.”
Ultimately, Valentine said that the most interesting part of this project is its placement in FDA’s transition of “being entirely a regulatory agency to a public health agency with a public mission.” “The patient network is at the core of that public health mission. We’re trying to improve the public’s health, and I truly believe that patient input will allow us to make better-informed decisions, and ultimately better decisions, that better meet the needs of the patient community and improve the public health.”