NCATS Director of Clinical Innovation
Mike's Blog
“How Are You Feeling?”… And Why
By: Josh Fessel, M.D., Ph.D., DCI Senior Clinical Advisor
May 1, 2023
As a lung doc and ICU doc (also occasional internist and, rarely, consultant for an autonomic thing or a strange metabolic thing) I’ve helped care for thousands of patients spread across ICUs, the wards, bronchoscopy suites, clinics, and even one unscheduled visit in my barbershop. Not a single one of those encounters began with a chief complaint that mentioned G-protein coupled receptor function, or a concern about a kinase, or anything like that. They all started with a person who didn’t feel well enough to do the things they needed or wanted to do, or a person who was scared about something, or both at once.
People seek care because they hurt, or can’t breathe, or are nauseated, or are so fatigued that it’s incapacitating. People seek care because they can’t see or hear or smell or sense touch or move like they need or want to do. People seek care because blood or something else that’s supposed to be inside their body is now outside their body, and they’re worried about it. People seek care because they (or their loved ones) are worried they might be on a steep path to disability or death.
Have you noticed the commonality? These are all issues that originate with the patient. In the classic teaching of symptom (what a patient tells the healthcare provider about) vs sign (what the healthcare provider observes or measures), these are all symptoms. These are all patient-reported things. Stated differently, the things that people want us to fix essentially all trace back to symptoms.
It is striking, then, that we understand so little about symptoms themselves. Most of our medical practice and a great deal of our medical research is predicated on the idea that if we address the underlying disease/pathophysiologic processes that seem to be producing symptoms, the symptoms will improve in step with the restoration of homeostasis. So, we focus not on the crippling fatigue, but on the anemia or the hypothyroidism or the obstructive sleep apnea that we think are the cause of the fatigue. The MAJOR limitation to this approach is that if there is no identifiable, active, and addressable disease/pathophysiologic process, we run out of things to offer pretty quickly. Telling the person that their blood counts and thyroid hormone levels and such are all fine does nothing to make them less fatigued.
Consider:
- We have essentially no effective treatments for cough, and little understanding of the biology of different kinds of cough. Yet, cough is consistently in the top 10 reasons for a patient-initiated symptom-driven visit to a primary care provider (detailed in this article and this source study).
- Pain is perhaps the #1 overall reason for symptom-driven healthcare visits. We have basically 4 kinds of meds for the long-term treatment of pain – acetaminophen, COX1/2 inhibitors, opioid agonists (full or partial), and GABA potentiators (e.g., gabapentin, pregabalin). You could be generous and count older antidepressants (e.g., amitriptyline) in there. I don’t count topical/local anesthetics like lidocaine patches. For comparison, we have for hypertension: beta blockers, central alpha-2 agonists, calcium channel blockers, thiazides, ACE inhibitors, ARBs, direct renin inhibitors, aldosterone antagonists, direct arterial dilators (e.g., hydralazine, minoxidil), nitrates, peripheral alpha-1 blockers… I’m sure I’m probably forgetting a class or two. And these are all for a thing that is so asymptomatic, we call it the “silent killer."
Patients are keenly interested in feeling better, and healthcare providers are keenly interested in helping them feel better. Over the last 7 years, the number of accredited hospice and palliative medicine fellowships (the subspecialty that is focused entirely on symptom management and helping people feel better irrespective of their disease) has grown by 50%, and the growth of the field overall shows no signs of slowing. But there are some important gaps and associated opportunities.
We don’t understand the biology of symptoms themselves nearly as well as we do the biology of disease processes. That means we don’t have the number and variety of interventions specifically for symptoms that we have for disease processes, and that’s an opportunity for research and growth. As attractive and relevant as mortality is as a clinical trial endpoint, we sometimes focus on it to the exclusion of deep inquiry into really making people feel better (only partial credit for PROs). There are opportunities for innovative trials that incorporate symptoms more thoroughly than we do now. Want to maximally engage participants and communities? Focusing on what matters most to people’s day-to-day lives, what they are or aren’t able to do, and how they feel is a remarkably effective way to engage with people and to be a trustworthy partner.
Advancing basic biology into innovative clinical studies that improve the well being of all people in a disease-agnostic way while engaging more deeply with the communities we serve. That sounds a whole lot like what the CTSAs can do very, very well.
Let the young know they will never find a more interesting, more instructive book than the patient himself.
- Giorgio Baglivi
This Mike's Blog was featured in May 2023's Ansible. Subscribe to receive upcoming Ansible newsletters.