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Move over Participatory Medicine, Here’s What Participatory Research Can Look Like

I first saw Emily Kramer-Golinkoff on stage at the 2015 Medicine X Conference at Stanford University. I had missed her ePatient talk while I participated in the always intensely satisfying networking but was lucky enough to catch her as part of a panel session. Though Emily was very small in physical stature and waiting all too patiently for her turn to chime in, the moment she did speak, I stopped in my tracks (i.e. looked up from my Twitter feed, which was always ablaze with worldwide, real-time dialogue about the sessions). I was struck at first by her unique voice and speech pattern which stood out amongst the other speakers. Quickly realizing that her voice belied a masterful grasp of health care and research issues and an expression of elegant, simple ideas for change, I remember saying to myself, “Wow. I have to meet her!” Only later, when I finally got to meet Emily, did I learn that she was a stone’s throw from where I live in the Philadelphia area. Funny how I needed to travel 1,000 miles in order to meet this powerhouse from my backyard.

Emily defies traditional definitions, but powerhouse is always fitting.

Emily defies traditional definitions, but powerhouse is always fitting. Trained as a bioethicist from the University of Pennsylvania, she is the engine behind a multi-level approach to changing the game for the advancement of treatments for patients with certain protein production mutations of Cystic Fibrosis (CF) called nonsense mutations. All all while living with the condition 24/7 she:

  • Provides funding for many phases of scientific research
  • Advocates for patients with Cystic Fibrosis
  • Participates in clinical research
  • Influences the design of research
  • Connects disparate scientific fields and scientists
  • Inspires action through her public speaking

Though I’ve only known Emily for a few short years, in that time she has rapidly advanced the cause for her condition as the co-founder of Emily’s Entourage (EE), a nonprofit foundation that raises funds and awareness to accelerate research for new treatments and a cure for nonsense mutations of CF. I was lucky enough to catch up with her recently and was happy to learn that she has a number of clinical programs underway.

Influencing Research Design
At Lilly we strive to responsibly partner with patients in as much of the clinical research design process as possible through our CoDESIGN capability. So naturally, I was very interested to learn how Emily is exerting her influence as a patient in this all-too-often science-centric activity, particularly when scientists may not have the wherewithal to engage patients at this level.

What is important to understand first, is that 90% of the CF community is expected to benefit from life-changing breakthroughs, and the community is already fully dialed into clinical research. Unfortunately, those patients with rare forms of CF caused by nonsense mutations have virtually no treatment options, but of course are literally dying to get into research and help to bring medical advancements for their condition. This is the reason EE was born, with the “laser focus of creating clinical research opportunities for this majorly underserved population.” As Emily simply and elegantly puts it, “clinical research leads to new drugs, which can bring life-saving advancements…clinical research equals hope.”

“clinical research leads to new drugs, which can bring life-saving advancements…clinical research equals hope.”

But building a scientific discovery and development network from scratch is hard. After months and years of building contacts, influencing economic drivers, understanding the scientific supply chain and tirelessly working to convince anyone to pay attention, EE is finally at a place where they are funding interventional drug research. And now a new chapter of hard work is underway for EE to help researchers design patient-centered trials.

Defining the entry criteria for research participation is a topic that many patients wonder and lament about, as it can often exclude interested patients from participation. The rules of research and limitations of resources and methods can often result in a very limited set of patients who match the criteria, and Emily herself has been excluded from many clinical trials due to her low lung function, a result of her advanced-stage CF.

Broadening the Research Viewpoint
In other aspects of research design however, she can influence subtle but profound changes as a result of the patient perspective that sometimes gets missed because certain populations are systematically excluded from trials. For example, her insights have awakened some researchers to the notion that inhalation delivery mechanisms can cause serious side effects particularly for patients with advanced-stage disease that make these medications not usable, often for the sickest patients who need them most. Emily, always the ever-gracious, explains, “There are some correlates that are not reflected in the research…and sometimes [the researchers] just don’t know...there is no mechanism to listen or learn.” And so Emily brings this welcome perspective as a result of her close, authentic relationships with the researchers. I have seen this kind of insight help our researchers at Lilly as well through our CoDESIGN capability. While this isn’t the core capability of the foundation, Emily recognizes that “it speaks to the multi-faceted value that relationships with patients and patient advocacy orgs can bring to companies.”

Emily has also drawn attention to a number of other relationships among comorbid conditions, symptoms and CF. Regarding the relationship between CF and CF-related diabetes, she states, “I know I have an exacerbation when my blood sugar levels start rising.” She also talks about how she can track sleep quality and begin to understand how declining lung health can affect this negatively. Emily is confident that “there are lots of patient-generated data that could benefit researchers as potential endpoints and early indicators of pulmonary exacerbations.” It’s exciting to see how Emily is beginning to unlock a number of ways to look at CF beyond just the conventional lens, which may spur scientists, not normally focused on patients with CF, to come up with solutions that could improve their lives.

While influencing scientific and medical thinking can be a herculean task in itself, true medical advancements can’t happen without real patients getting involved in the research. And once again, Emily has not fallen short there. Over the last few years, EE has built up a sophisticated audience of like-minded patients and advocates devoted to the mission of bringing much-needed therapies to patients with nonsense mutations of CF.

true medical advancements can’t happen without real patients getting involved in the research

Online and Offline Engagement
In addition to growing a community of patients organically through social media, local and national media and speaking engagements, EE has also developed a patient registry for people with nonsense mutations of CF in the hopes of helping researchers expedite development of treatments for their condition. While this “registry” aims to be a “goldmine for research communities,” it is really a collective quest, as Emily would define it, providing a real go-between for researchers and the total patient population who suffers from CF, and possibly defining the archetype for what participatory research might look like. The fact of the matter is that patients and researchers desperately need each other. As such, EE’s core mission is to enable deep collaboration between these two groups in a responsible way that advances the science and empowers patients, all while protecting their privacy, safety and autonomy.

Collectively, all of these approaches bring immense value to the CF community of people living with nonsense mutations. But maybe the most impressive accomplishment of the foundation is the way it has literally changed the enterprise of research for this field. Emily has done her equal share of listening to scientists and researchers, and has responded and mobilized the community in kind. As such, the whole “toolbox of research has been brought to the next level.” EE has developed a sustainable model for providing a litany of new tools and networks for researchers (e.g. organoids, cell lines and cell models with two copies of the nonsense mutation), to help ensure expansive use for scalable research with the hopes of igniting scientists everywhere to find treatments and therapies for patients with this version of CF.

It is with this level of wonder and respect that @LillyTrials is proud to sponsor the 7th Annual Emily Entourage Gala to support this growing participatory research model so that we may learn from it to help all patients everywhere, looking to be responsible partners to advance medical breakthroughs in other therapeutic areas. Meanwhile, like on that panel at Medicine X, Emily waits all too patiently, along with so many others with different conditions, for treatments or cures that will extend or improve their lives. But while she waits, she acts. So let’s all of us act, too. Because…science.

Tags: participatory research, codesign, epatient,

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