Burden-of-Illness Scales: Helping or Hurting?

Burden-of-illness scales help us understand how a disease impacts patients beyond its symptoms, identifying the most problematic emotional, social, and economic aspects. I also thought they helped FDA submissions, prescriber promotions, advocacy activations, and payer access discussions. I loved how “patient-centric” they were.

Sure, they had limitations, but doesn’t everything?

No Longer Academic

Newly diagnosed with Celiac Disease, those limitations got personal when, as an advocate, I came up against “Celiac is not a serious disease” objections from policymakers.

Here’s lookin’ at you, FDA.

I’d developed Personal Network Analytics by then, so when I studied the Celiac Disease burden scale, I understood FDA’s reaction in a new way. One question stood out: “Do you have a problem socially because of Celiac? Select one: Often, Sometimes, Rarely, Never.”

Socially? OK, where?

Socially, in my own home? No, I don’t bring gluten into the house. Guests bring gluten-containing hostess gifts; I can deal with that.

Socially, in someone else’s home? Yes. I’m “chill” about the food because I’m there for the fellowship, but to the hostess, I’m the “guest from hell.”

At a ballpark? Yes.

At a wedding reception? Yes.

At a client’s catered lunch meeting? Yes.

At church? The late Pope John Paul II twice ruled that Communion wafers must be wheat, so Bishops invalidated the First Communions of little kids who received rice wafers instead. Imagine that burden. I can’t.

This is but one example of the inadequacy of burden scales. You need to see it only once to recognize it elsewhere. Today’s inadequate burden scale metrics are negatively impacting every phase of a product’s lifecycle and creating barriers to the medicines patients need. Margaret’s experience is instructive.

Margaret’s Chronic Cough Burden

Margaret (not her real name) is married, with an adult son, a part-time job that provides health insurance, and a home-based business. She’s active in her community, a member of an adult choir, and makes a two-hour round trip each week to spend a day helping her elderly parents. She was diagnosed with Atopic Dermatitis as a teenager, but is an otherwise healthy non-smoker who rarely uses healthcare services.

In  June 2024, an occasional morning cough worsened. When OTC cough and allergy medications weren’t effective, she began a 16-week journey. Her Primary Care Physician referred her to a Pulmonologist, who then referred her to an Allergist, who in turn referred her to an Otolaryngologist.

Score	Daytime	Nighttime
0	No cough	No cough
1	Cough for a short period	Cough on waking only
2	Cough for more than two short periods	Wake once or earlier due to coughs
3	Frequent coughing, which did not interfere with usual daytime activities	Frequent waking due to coughs
4	Frequent coughing, which did interfere with usual daytime activities	Frequent coughs most of the night
5	Distressing coughs most of the day	Distressing coughs preventing any sleep

Cough Symptom Score

Cough Symptom Scores are a straightforward way to rate the cough (for her, a “5” for both daytime and nighttime).

Time and Cost Burdens

The scale could not capture her time and costs, however.

• 20 HCP, 12 Pharmacy, and eight diagnostic (lab and radiology) visits.
• 19 prescription changes, with a final, complicated regimen of six drugs multiple times daily.
• 14 hours of travel time and 25 hours of wait time at HCP offices and diagnostic clinics.
• $4,500 in MD copays and $563 out-of-pocket medication costs.

Otherwise “Hidden” Burdens

Nor could the Cough Symptom scale capture other types of burden.

• She went from being a caregiver to needing care; no longer able to care for her parents and requiring care from her husband, son, and son’s girlfriend.
• Constant fatigue left her unable to work in her own business, so her elderly parents assumed responsibility for some of her bills.
• Difficulty breathing left her unable to answer phones at work, hold long conversations, or sing, so she cut back on work hours and resigned from the choir.
• Fearing an infectious cause, physicians suggested she not visit her immunocompromised best friend.
• Fearing a mold-related cause, physicians suggested she avoid the family’s cabin, limiting her summer fun and that of family and friends she entertained there.
• Adding to her time and costs, though she never received a definitive diagnosis, all four physicians want her to continue regular appointments with them.

One burden was especially tragic. One of the specialists recommended a biologic medication, but its formulary tier made it unaffordable. We suggested she explore whether a patient assistance program might be available to help, but she was too exhausted to follow up.

Imagine if market access teams had experiences like hers to help secure more favorable formulary placement. Might they be more successful? 

Margaret’s Additional Atopic Dermatitis Burden

Margaret did her best to return to everyday life, and though her condition improved somewhat, she never fully returned to her pre-cough normal. It is alongside that reality that the burden of Atopic Dermatitis (AD) becomes relevant. As those who understand the condition would know, she experiences periodic flares, especially when she is under stress.

Her Dermatology Life Quality Index (DLQI) score during those 16 weeks was 17 (of a possible 30), meaning AD had a “very large effect” on her life. The severity of her symptoms drove that score, as did the embarrassment she feels about her appearance, and the impact on her leisure activities. Like other burden scales, though, the DLQI has limitations.

Important Life Event and Essentials Limitations

The DLQI does not adequately capture aspects of life that matter.

• When her son wed in a black-tie ceremony in October, she could wear makeup only long enough for the ceremony and photos afterward. “I felt like a queen for two hours; I felt like a monster when my skin told me to take it off.”
• She limits activities to only the very most essential for her household and work to avoid appearing in public for exercise, entertainment, or enrichment.

Self-Administration Limitations

The DLQI and other scales are self-administered, and we learned about that limitation only by accident when her score didn’t align with how she talked about AD. Exploring that difference, we discovered that she had not scored the ten elements against “life before AD.” Over the years, she adapted to the condition, creating a “new normal” that distorted her current ratings. In other words, she accepted that some level of “background” burden would always be present and rated her current burden against that.

When we asked her to rate the burden as the developers intended, comparing it to her life before the AD, her score increased to 25, indicating it had an “extremely large effect” on her life. An instrument like the DLQI could not capture her disease adaptations:

• AD caused “no problem” in her work because she chose to start a home-based business decades ago and later chose a part-time job that allowed her to work remotely. That adaptation helped her avoid any embarrassment she might feel from her appearance, but it also limited her career choices.
• AD caused only “a little” problem with close friends because she limited her social life to the very few people she knows well and who accept her regardless. That adaptation leaves her with a small social circle and risks loneliness, particularly as her friends age and pass away.
• Her current treatment, a generic steroid cream, rates as “no trouble or mess” in the DLQI, but it’s not effective. She is among those who are “resigned” to their skin condition's fate. As probably the most egregious limitation of all, the DLQI does not allow patients to rate treatment effectiveness.

Natural Disaster Limitations

No burden scale, including the DLQI, helps us account for life’s unexpected challenges and extraordinary demands. Imagine Margaret experienced one of the recent floods in North Carolina or Texas. Without a more effective AD treatment, the natural barrier function of her skin would have been compromised, leaving her more vulnerable than others to bacteria, fungi, sewage, pathogens, and other chemicals. Increasing numbers of patients face this reality.

Life’s “Wild Card” Limitations

Within six months of her 16-week chronic cough journey, and as her AD flare continues unabated, Margaret assumed new caregiving responsibilities. No DLQI item accounts for circumstances that make disease burdens exponentially worse.

• Her father passed away. Her mother, who is legally blind from age-related macular degeneration, needs Margaret’s caregiving support even more often.
• Her husband’s three-month journey with back and hip pain was diagnosed as a hip fracture requiring surgery.
• Three months later, while hospitalized for a suspected heart attack, her husband was diagnosed with inoperable cancer, with metastases at several organ and bone sites, and began his cancer chemotherapy journey.

Burden and Value Stories

Those who understand Margaret’s chronic cough and AD conditions would recognize there are newer treatments that might relieve her symptoms, allow her to return to social engagements, support her need to generate income (even at retirement age), and care for her mother and husband.

She’s checked. Getting access to those medicines will not be easy. Some are not listed on her drug plan formulary. Others involve prior authorization hurdles, and it’s unclear whether her physician will be willing or able to navigate those.

My conclusion? The burden scales we depend on to secure FDA approval may be necessary, but they are not sufficient to support patients’ needs for advocacy and access.

We need new insights to succeed at the rest of our job with:

• More compelling messages for HCPs to encourage timely diagnosis, appropriate treatment, and, if necessary, to surmount prior authorization hurdles.
• A rationale for improved clinical guidelines to support more patient-centric care.
• New content to support patient group education and advocacy roles.
• Improved dossiers to secure access to coverage and reimbursement.

Informing and Activating Patients

We’ve seen it time and again: even patients struggle to articulate the burden of a condition. Like Margaret, they underestimate the impact on their life and are therefore less likely to seek new solutions or advocate for themselves.

As Margaret said, “When I saw this data, I cried. I had no idea this was my life. When you’re in the middle of it, you take it day by day and sometimes moment by moment.”

Today, she has new insights that have motivated her to seek better options. Will that be the case for others? I’ve seen it happen, so I’d say, ”yes.”