Considering caregiver value at JP Morgan
Paul Kidwell, patient advocate and rare caregiver, brings us the first in a regular series of his brand new Openly Caring column, exclusive to RARE Revolution. Here Paul will be giving a voice to the experience of caregivers within the rare ecosystem, drawing on his own experience and from his insights as an advocate in the space
Written by Paul Kidwell, communications and patient advocacy consultant and co-host of the podcast, InSickness. Men and the Culture of Caregiving
As the entire biopharmaceutical industry makes its way back home in the wake of the 43rd Annual JP Morgan Healthcare Conference this past week, my hope is that the caregiver voice reached a crescendo in the discussions and presentations; particularly those focused on rare diseases. The rare caregiver voice is incredibly important and should be more than a mere talking point. Our stories help ensure that the perspectives and experiences—both anecdotal and real-world—of rare caregivers and patients who live with one of the more than 7.000 rare medical conditions, are clearly understood and integrated into the development of new treatments and healthcare policies. Without this voice the conference becomes the “sound of one hand clapping” and industry drug hunters miss out on critical information that might help lead them to creating life-impacting patient-centered treatments.
If they don’t know already, those attending the JPM event should start to recognise what a valuable resource they have in caregivers. Engagement with caregivers helps provide the runway towards better, more accessible, and more effective treatments. Caregivers bring to the table a valuable asset around drug hunting that is dialled into the creation of patient-centred treatments for rare diseases. Rare disease caregivers come equipped with minds of scientists and hearts of healers.
Influence felt in each phase of drug hunting
Throughout the patient journey there is no one closer to a patient than the caregiver— they witness a medicine’s effectiveness, it’s trajectory, the emotional “slings and arrows” a patient endures, and the ancillary experiences of drug prices, “do’s and don’ts with HCPs, and the challenges living within a feudal healthcare system. Also unique to the rare disease caregiver is the frustration experienced as they support a family member or loved one who is sick with an inexplicable illness; for which there is little medical knowledge and even less validating research. The majority of those in this position are left mostly to their own resources as they spend countless hours seeking information on a mysterious rare condition, knowledgeable researchers, and anyone else who may be going through the same situation and feeling similar frustration.
If you saw BIO CEO, John Crowley, walking through Union Square during the past week I hope you stopped to ask him to share his story. He’d likely tell you how extremely priceless caregiver data is and the large role it can play in improving the understanding, design, and success of rare treatments
In the care of a rare patient, a caregiver observes in real-time how a medication is working, or not, how long a patient might remain symptomatic, and when a medication kicks in. It may be anecdotal, but it has the potential to be as insightful as any trial data point in informing a clinician the direction a therapy is taking. The information is actual and may do a significantly better job of aiding in the creation of patient-centric therapies that enhance outcomes. This is especially important in rare diseases where researchers do not have the luxury of gleaning data from large patient groups and each interaction with a caregiver throughout the clinical continuum becomes a treasured kernel of detail, from trial design to commercial launch.
Timing and location; it’s still everything
Identifying and agreeing upon a trial’s endpoints is often a major hurdle in a trial’s start and understanding the meaningful insights of a caregiver can help identify endpoints that matter most to patients and their families. For example, caregivers may be able to highlight specific symptoms, side effects, or functional changes that would indicate a treatment is not working, beyond what traditional medical assessments might show. This can lead to more meaningful and effective clinical trials.
Caregivers are there from day one when a trial begins and have an intimate view of how a patient may respond to a therapeutic in the all-important early stages when clinicians are trying to understand what the safest and most tolerable path forward may look like through a series of stops and starts. As caregivers—and this is especially true for many rare disease caregivers who are supporting young children suffering from a pediatric condition—our ultimate goal is to assure that the person for whom we provide care, lives a life of optimal safety and health. Since caregivers are closely involved in day-to-day triage and management of a person’s illness, we can offer insightful commentary and somewhat empirical data regarding side effects or adverse events.
The timing of these observations is key as we can see almost immediately any concerns that may adversely affect a patient’s health and/or quality of life. Early is always better and our 24-7 view allows us not to overlook side effects that may fly under the radar in traditional clinical settings. This home-based, time-sensitive information is vital for assessing the full scope of a drug’s safety and effectiveness.
When in doubt; care
In my own life I had a situation where I made a medical decision because a health emergency popped up with my wife and I have learned through this 18-year experience that a trip to the emergency room (ER) is always the last choice. This is particularly true for someone like my wife who has cognitive issues and is in a wheelchair. After returning from a brief hospital stay where her doctors modified her dementia medicines the discharge orders clearly stated when to administer the new meds. Which included bedtime when she was already taking a modest sleep sedative.
The next morning, she awoke largely disoriented and stayed that way through most of the day. It was a Saturday and after speaking to the attending ER physician (who was not part of my wife’s team) I was left with the option of a disruptive ER visit or rely on my wits and let this temporary situation run its course and at bedtime eliminate the sleep aids. Four months later, following this regimen was the right decision as she has never again displayed that type of behavior. With the incident in the rear mirror, my wife’s medical team concur.
Not just another pretty clinical face
As a final thought for the hordes of dealmakers at JP Morgan shuffling the cards around rare disease collaborations. Rare caregivers are not sitting idly by looking for behavioural and physiological aberrations. We have stored incredible amounts of patient detail that can be helpful in paving the way towards approval, market acceptance and compliance.
Want a seamless approval? Patient and caregiver perspective is what agencies like the FDA covet as they weigh decisions that ensure real-world acceptance. So, ask a caregiver. Want physicians to prescribe your medicines that are reflective of the actual needs of patients? Or maybe you need someone to advocate on behalf of your rare therapeutic. Someone who can talk about its impact on their spouse, child or parent. Sounds like an opportunity for a caregiver. Finally, adherence is often the key to ensuring patient health. In my role as a caregiver, I administer over 20 pills daily beginning at 7:00 a.m. and finishing at 9:00 p.m. It’s a responsibility I take very seriously. We all do. No need to ask.
We are delighted to welcome Paul Kidwell as a regular contributor and supporter of the caregiver voice throughout 2025. For more information on Paul’ ‘s podcast check out https://www.insickness.org/
