The US Food and Drug Administration (FDA) recently set out to get patients and patient groups considerably more involved in the regulatory process through its Patient Network, a new website and initiative that the agency hopes will clarify the processes by which regulated healthcare products come to be approved and marketed.
For more information, please see our 23 April 2013 article, "Patients First: FDA's New Website Aims to Make Regulations Clear to Everyone, Invite Involvement."
Regulatory Focus recently sat down with one of the architects of that initiative, James Valentine, program analyst at FDA's Office of Health and Constituent Affairs, to explain a bit more about the program and what the agency hopes to achieve from it.
Regulatory Focus: James, tell us a little bit about yourself and the Office of Health and Constituent Affairs
James Valentine: My name is James Valentine, and my background and training is in public health. I am a patient liaison in FDA's Office of Health and Constituent Affairs within the agency's Office of External Affairs. The Office of Health and Constituent Affairs is the agency's primary patient liaison and patient advocacy office within the agency.
We've actually been in this role since the late 1980s when there was the HIV/AIDS crisis. We were created as the AIDS relations staff back then because there was no one to interface with the advocates who were trying to have their voices heard at the agency. We've evolved over time and had many name changes, and now we're the stakeholder liaison office.
But I'm on the patient liaison team within the office and have helped manage the creation of this website.
RF: What is the philosophy behind this patient network initiative? What's the underlying theme of what you're trying to accomplish?
JV: In terms of what our goals were for the creation of the site, I think our launch slogan really helps to capture that: "Get informed, get involved: Help FDA help patients have a bigger voice." We really wanted to provide a resource for any patient or patient advocate that is at all interested in FDA-regulated medical products or issues to have a home for getting educated about these products, how they're regulated and how they can be a part of that regulatory process.
So I guess we knew that some patients will only come to the site to get some information, but we hope that other patients will actually become advocates for their patient community and that it will allow them to provide FDA with their unique patient perspective, allowing us to make better-informed decisions with that perspective. That's kind of our goal for the site.
Now, thinking of what we wanted out of the site, we really wanted to centralize what was otherwise disparate information that is important to patients and patient advocates in a central location and make that information usable and also written in such a way that the patient audience could comprehend it. From the very beginning, we engaged patients and patient advocates through focus groups. Even though our office has a lot of history working with the patient advocacy community, we didn't want to just create a site that we thought would best serve their needs. We figured that they are the best people to tell us what they need from FDA, so we engaged them through focus groups and then subsequently through several rounds of usability testing to make sure that the site really worked for them and contained what they wanted.
I think that based off of the limited amount of feedback that we've gotten so far that people seem to think that the site is usable and very logically laid out for what the patient needs. I think that was something we realized that the current website, although it's very information rich, just isn't organized for the lay audience in terms of either finding information or using that information.
RF: You mentioned giving patients a bigger voice. Is that a recognition by FDA that patients don't have an appropriate level of influence within the regulatory process?
JV: No, I think that it reflects an interest that the agency has in continuing to grow their influence. The website really is not creating a new patient advocacy program, per se. It is really facilitating a broader amount of patient input and participation in current mechanisms and evolving mechanisms for patient input. The primary program for patients to really participate in regulatory decision-making is that patient representative program, and that has continued to grow over the years. We now have over 170 active patient representatives who represent over 105 different disease areas, and that continued to grow. We have almost universal participation of patient representatives on all FDA advisory committees for all medical products.
FDA Safety and Innovation Act (FDASIA) Section 1137, a little provision contained toward the end of the law, mandates that the agency look into ways to expand patient representatives' participation in agency sponsor meetings.
RF: What does that provision, Section 1137, entail and how are you incorporating it?
JV: That provision, very broadly, wants FDA to look into strategies to include patient representatives in those sponsor meetings; what the law calls "appropriate agency meetings." This is something we have historically done in regards to oncology and AIDS, but now we're going to look at a systematic approach to implement it across all of the three medical product centers (drugs, devices and biologics).
We're actively looking to come up with the strategies, but there are some hurdles that we will need to figure out how to navigate. Mainly, conflict of interest screenings given the short periods of time between when a sponsor requests a meeting and when it is scheduled. But we are very supportive of that provision and hope to have by the fall those strategies laid out and start to implement them.
RF: How is FDA approaching the training aspect of regulatory in regards to these patients?
JV: This is something that we've thought is very important for the patient advocacy community in terms of their success in engaging with the FDA. Historically, our office has provided training or educational content in one-on-one settings with these groups by phone or email, starting at the high level of product regulation (e.g. FDA 101) and working our way down to the specific issues that the patient is most concerned about.
We've always had this philosophy that the patient can be the best advocate if they're educated about the role that industry plays in drug development, that FDA plays in development, and how they intersect. And that really was the focus of the core educational content of the website, which are those drug and device development sections that I think 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.
That said, our goal is not to just get people to the website. Our goal is to be able to allow people to ask questions. We plan 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.
I don't think we ever plan on building out the site to try to address the specific issues of every single disease area. I think that would be a little too difficult to do. Just to maintain something like that would take too many resources. One of our goals is really to allow any and all patients and patient advocates an equal opportunity to get that first foot in the door.
RF: How is FDA going to accommodate the increased input it hopes to get from these patient groups? Is FDA looking to guide patients to the federal docket to comment, or is there a more comprehensive way to incorporate patient comments into guidance documents and reviews?
JV: So, the website will be a great tool for helping to identify the opportunities for patients to become involved.
Each disease community may have different interests. 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. Depending on the needs of different communities, we plan on having those opportunities to give input.
It's really a great time to launch the website now because of new opportunities developing at the agency coming out of FDASIA like patient-focused drug development. It's truly a new evolution in the way that we're incorporating patient input into our regulatory decision-making. We're not 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.
RF: One of the requirements for patients serving on committees is that members need to be "objective." What does that mean to the agency, and how does it plan to evaluate objectivity?
JV: I don't have the exact citation, but it's the same conflict of interest screening that any federal employee has to go through. Whether you're a regular government employee or you're a special government employee like a patient representative, you have you go through the same screening process for financial conflicts and objectivity, which relates to any comments you may have made in the public domain that would reflect a pre-decided position about a particular regulatory decision.
The idea is that we want you to come to the table with your personal experience, which may include information about a product or its competitors (especially in rare disease communities), but also the perspective of other patients in a given disease group. But if you're already decided that a particular product that you know is in the pipeline should or should not be approved, then you're not going to be objective when you're reviewing the application materials that the agency is reviewing. We want people to react to the application as submitted with the backdrop of their and their communities' experiences, but we want to avoid the appearance of non-objectivity.
RF: You mentioned rare disease communities. Increasingly, rare disease communities are involved in the development of products and are more involved in trials. Is FDA concerned about finding 100% un-conflicted patients when dealing with these types of communities?
JV: That's something that becomes difficult as we're trying to find people for these types of consulting roles, because we don't have time to try to find someone that might have less conflict of interest. But the issue isn't even unique to patients. The same issues are present in researchers and scientists serving on rare disease advisory committees as well. The agency is aware of the issue and 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.
RF: Moving beyond advisory committees, are there other initiatives in the works meant to positively affect patients?
JV: Going down the line with adverse event reporting, one of the things being worked on is an actual consumer form for reporting. That's something that has never existed before. Right now, there's the generic MedWatch adverse event reporting form (FDA Form 3500), which isn't consumer-friendly.
We're in the process of developing a consumer-friendly form that will eventually be featured on our website, as we think our audience will be the largest user of this form.
RF: If you're looking back at this program in five years, how will you have defined success for this program?
JV: I think for us, success will be defined by the quality and extent of patient engagement that we've had. At the patient level, rather than the patient-advocate, there's a lot built into the website where we're trying to help facilitate access to investigational products through expanded access programs, as well as helping people to identify whether clinical trials are appropriate to them. We currently spend a lot of time on the phone trying to guide patients through that process.
Success-wise, we can look at the expanded access applications that come to the agency and to see if there is an increase in quality or numbers of those just because people are more aware of it as an option. That would be a measure of success as well.
On the patient advocacy front, I would be looking to see if we have been able to get that broad patient input into our advocacy efforts. So, 'was this website able to allow us to get broad representation of patients in the patient representative program and the patient focused drug development program,' and whatever else the newest evolutions look like in the future-are we able to support those and be adaptive?
I would even like to see a way for patients to directly provide input to the agency using the Internet. Right now it's sort of a gateway to the program, but there is limited amount of benefit with having people comment on dockets or show up to meetings. We're advanced with webcasts and webinars with having people participate remotely, but to even further expand upon that and allow people to provide input without even leaving their computer would be great.
RF: Does FDA have any concerns or hesitancies at this time?
JV: No. We've worked with all the medical product centers throughout this whole process of bringing this website to fruition. Everyone's been very excited about it. I think the agency sees this as a model of how to engage with the lay audience, starting with patients, and I think it's even a model for how to move forward with web content management for the agency.
I think whether you're talking to people in a communications shop or regulatory staff, everyone has seemed pretty excited to see how this expands our engagement with patients.
RF: How are you going to be accommodating the increased number of patients engaging with FDA through this process? How do you make sure one group doesn't box others out of getting attention?
JV: I think that will be something I may have a different response to months from now, but we have a team of people from all different disciplines-medical practitioners, public health professionals, patient liaisons-and we're very much looking forward to this increased activity with the patient community.
We haven't added people, per se, but I think if we're able to spend less time doing some of the basic education that the website will now provide, we can spend more time working with the groups and individuals on their specific issues. I think the support we have from throughout the agency will make it so that we can be successful in doing this. All of FDA's centers are all constantly looking for new ways to engage with patients. We're come a long way from when the agency was being picketed by AIDS advocates. Now, we're at a point where our office is being asked for the patient perspective in all different ways.
I think everyone will be happy because there's an increased need for the patient perspective.
RF: Is the goal here to just generally be more responsive to patients, or to actually have them fine-tune the regulatory process in ways that FDA officials haven't yet been able to?
JV: The patient network is really there to support the framework as it exists and as it evolves. That being said, the patient community has been a driver for changes in the regulatory framework for as long as they've been engaged with FDA, and FDA-in my experience-has always been very receptive to those changes.
Throughout history there is example after example where patients have been able to shed light on things where, traditionally, it has been left to the scientists and regulators to think up what the best process is.
RF: What's something interesting about this process that we should know?
JV: The most interesting part to me is the transition of FDA being entirely a regulatory agency to a public health agency with a public mission. I think this 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.
It's really great to be a part of a program where we're embracing that public health mentality.
The above interview was held on 25 April 2013.