Not so long ago, the Food and Drug Administration was completely cut off from patient voices.

Luckily, that's been changing in recent years. The federal agency has upped the ante by creating to advisory groups that include patient advocate POVs from a variety of disease states, including diabetes!

The first Patient Engagement Advisory Committee came together in early 2017, and then by the end of the year the FDA created the first-ever  (PEC), a joint effort with the Clinical Trials Transformation Initiative (CTTI). But don't let that group's name fool you -- this goes beyond clinical trials to create a channel for "patient engagement" on topics ranging from study design, to how technology or mobile health apps are developed, to the process of reviewing new products and evaluating their impact on users, to even how the agency communicates and tracks information.

“Patients are increasingly... spearheading the creation of novel registry platforms that collect health data to track their progress, and facilitate medical product evaluations. We’re fostering interactions with these patient-led registries to help them become vehicles for regulatory change," FDA Commissioner Scott Gottlieb announcing this last year.

We definitely like the sound of all this, as how could it NOT be important to include more real-life perspectives from those who actually live with diabetes and other health conditions?

Even more exciting is that we have TWO diabetes advocates serving in these roles -- D-advocate from Pennsylvania who lives with type 2 and has two T1D children was named to the first committee; while longtime type 1 from Indiana has just recently been named to the PEC.

Cheers to the FDA, and to both of these D-peeps making sure our collective patient voices are heard!

But just how is that being facilitated here?

 

The Patient Engagement Collaborative

Those invited to apply for the PEC are people who either: have "personal disease experience"; are caregivers such as a parent, child, partner, family member or friend; or are representatives of a patient group with direct or indirect disease experience.

On July 12, the 16 individuals were named to this new collaborative.

They include an array of different conditions and life experiences, and as noted, we're excited to see longtime T1D-peep Rick Phillips selected out of more than 800 applicants for an initial two-year term.

"That makes this very special to me," he tells the 'Mine.

Some of the topics on the table to talk about include:

  • making patient engagement more systematic
  • how to improve transparency
  • education and communication on the medical product regulatory process
  • new strategies for increasing patient input
  • new models for patients to collaborate as partners in the medical product development and FDA review process

This all stems from continuing efforts by the FDA to be more inclusive and recognize people on the receiving end of healthcare. This new collaborative is actually modeled after an initiative in Europe known as the European Medicines Agency’s Patients’ and Consumers’ Working Party (PCWP). The US legislative framework allowing for a similar group includes provisions in the  of 2016 and the that aim to expand patient participation and incorporate patient experiences in the regulatory process.

This is what the about this collaborative effort.

As to this selection, Rick shares that he believes his application was buoyed by support from the diabetes and Rheumatiod Arthritis communities (where he's also an active advocate), particularly from the local Indiana JDRF chapter, by Christel Marchand Aprigliano of the , and Mila Ferrer of .

"I'm grateful for those endorsements and I of course hope I will fulfill the trust they placed in me," he says. "I am always open for suggestions about how to improve communication or interactions with FDA. I cannot guarantee your ideas will be adopted. But, I know if we do not raise issues it is unlikely they will be adopted."

Rick encourages the D-Size now to contact him with any ideas directly at [email protected], or via or Twitter or Facebook.

 

FDA and the Diabetes Size now

Of couse, the FDA has already made some great strides in connecting with our own Diabetes Size now in recent years -- from a number of DOC webinars and online discussions (like that time our DOC Crashed the FDA Webinar Server!), to improving its efficiency in how it works to quickly approve devices, to FDA leaders at our #DData events, and most recently launching an accelerated review program for mobile health tools.

In early 2017, the FDA began the process of creating a formal umbrella infrastructure for patient input with a new Patient Affairs Team. Later that year, that group's first act was to create the  made up of nine people who meet periodically to discuss topics such as agency guidance and policies, clinical trial design, patient preference studies, benefit-risk determinations, device labeling, unmet clinical needs, and other related issues on the regulatory front. The .

While the FDA is cautious about how Advisory Committee members talk publicly about their work, our friend Bennet Dunlap was able to give us some general info about his experience in the first months of serving in that advisory role.

“It was phenomenally surreal to be sitting on the other side of the ropes, being the ones listening to all the testimony. We do have to realize that the FDA’s a big agency and, being that I like to use the 'big ship' analogy, they don’t turn on a dime. We have to be patient, and understand that... there’s not going to be 100% overlap (in different groups' agendas) because everyone does have their own mission.”

Overall, Bennet says he’s encouraged by the moves the FDA is taking to include more people. "The FDA is becoming, by intention, much more patient-centric. That’s fantastic."

 

The Diabetes Size now vs. Other Disease States

Bennet says it was helpful sharing stories and hearing from those in other disease states, and recognizing what similarities and differences exist in what we’re all dealing with. He also thought it was helpful how the FDA Advisory Committee organized the room for the first meeting, having members grouped at smaller tables first where they could discuss and summarize thoughts before presenting them to the larger group.

It became clear to Bennet how fortunate we are to have strong national advocacy and scientific orgs like the American Diabetes Association and JDRF, while many other disease states don’t have the like.

“To some extent, they’ve become a voice for the patient. Maybe that’s not always appropriate, I don’t know. They are the voice of scientific research, in my view, more than voice of the patient, but they do bring patients to the Hill and into this process. Other disease states are trying to figure out how to emulate that,” he says.

What our D-Size now does not have compared to other disease states is formal training of grassroots advocates, Bennet notes. Some of the rare disease states have organized processes to help individuals get involved, bringing people up to speed to have one clear message, and teaching advocates how to get behind it. Our lack of that unity may be a result of our large and diverse community.

“There’s a joke about two ninjas parachuting out of a plane and trying to cut each other’s parachute cord. We do that between different types (of diabetes) in our community, with everyone arguing about what our priorities in advocacy should be… We’re all kind of slapping each other in the face, instead of taking a calm, 'let’s go get this done' approach," he says. "There’s a diversity of goals and that’s a two-edged sword.” 

 

Who 'Owns' Patient Engagement?

While all of this FDA change has been happening, it's been interesting to watch some of the online chatter questioning these "patient engagement" efforts -- not just by the FDA and government folk, but also by national non-profits, industry, and healthcare providers.

We often see efforts to "include patients in the process" that never materialize into actual change; a handful of people are included as "token patients" seemingly more for show (to check a box) than with any tangible goals in mind.

And some folks complain it's not for "them" to use the language of engagement or push for it, but rather it should be driven by "us" -- those living with diabetes (or other health conditions).

That's an interesting philosophical quandary, but honestly any "Us vs. Them" situation tends to create silos and negativity. As Bennet says based on his FDA advisory experience, the real need is to move toward an "Us WITH Them" mentality in order to improve the system.

We agree. Here's to working together with regulators, industry, medical professionals, advocacy orgs, and other health condition advocates to move the needle and get a more inclusive, efficient system.

From our perspective here at the 'Mine, mutual engagement is a very good thing.