Why Patient Burden Should Be a Core Development Metric in Oncology R&D

Executive Summary

For decades, oncology drug development has been measured through two dominant lenses: efficacy and safety. While these remain essential, they no longer tell the full story of whether a therapy truly delivers value in real-world cancer care. An additional dimension — patient burden — is now emerging as a critical determinant of therapeutic success, especially in populations such as non–small cell lung cancer (NSCLC), where most patients are elderly, medically complex, and often unable to tolerate the aggressive regimens that dominate clinical guidelines.

This article argues that “treatment burden” must become a core metric in oncology R&D, shaping trial design, therapeutic innovation, commercialization strategies, and health-system planning. If oncology is to evolve toward more sustainable, patient-centered care, reducing burden will be just as important as extending survival.

The Reality of Cancer Care Today: Patients Are Struggling With the Burden of Treatment

Modern oncology has grown increasingly intensive. Frequent infusions, cumulative toxicities, extensive monitoring, long travel times, and complex regimens place substantial strain on patients and caregivers.

This burden is not a soft concept — it directly influences adherence, outcomes, healthcare utilization, and quality of life.

The issue is especially acute in NSCLC:

• The median age at diagnosis is 70 years (SEER, 2023).

• Up to 40 percent of patients have significant comorbidities (Owonikoko et al., JCO, 2014).

• Frailty, poor performance status, and limited mobility significantly impact treatment tolerance (Hurria et al., JCO, 2014).

When treatment burden exceeds a patient’s physical or emotional capacity, the consequence is predictable:dose reductions, treatment delays, discontinuations, or refusal of therapy altogether.

Why Patient Burden Must Become a Formal Development Metric

1. Survival Gains Are Meaningless if Patients Cannot Complete Treatment

Clinical trials measure intent-to-treat outcomes, but real-world patients often cannot adhere to complex regimens due to toxicity, logistical challenges, or deteriorating functional status (Schnipper et al., JCO, 2015).

Therapies that are “effective” on paper fail in practice unless burden is manageable.

2. Treatment Burden Predicts Real-World Benefit Better Than Clinical Protocols Do

A patient’s ability to sustain treatment correlates strongly with overall outcomes.High-burden treatments often select for the fittest minority, excluding the majority of real-world patients who are older or frail (Wildiers et al., JCO, 2010).

3. Health Systems Are Strained by Highly Burdensome Therapies

Infusion centers, oncology nurses, and acute-care beds are increasingly overcapacity. Treatments that reduce hospital visits, supportive care needs, or toxicity-related admissions directly relieve health-system pressure (Kimmick et al., The Oncologist, 2015).

4. Global Oncology Access Depends on Reducing Burden

Low- and middle-income regions cannot support complex systemic therapies, but they can adopt simpler, lower-burden modalities that require less infrastructure (Atun et al., The Lancet, 2015).

Without addressing burden, global disparities in cancer outcomes will continue to widen.

5. Payers Are Prioritizing Value, Not Complexity

Reimbursement frameworks increasingly reward treatments that deliver meaningful outcomes without disproportionate cost or resource use (Tefferi et al., Mayo Clinic Proceedings, 2015).

Treatment burden is directly linked to total cost of care.

What Should “Patient Burden” Include? A Modern Framework

Patient burden is multi-dimensional. A robust development metric should capture:

A. Physical Burden

• Treatment-related fatigue, nausea, pain, and functional decline

• Cumulative toxicities impacting daily living

• Vulnerability in older or comorbid patients

B. Logistical Burden

• Time spent traveling to treatment centers

• Frequency of visits and duration of appointments

• Need for caregiver support

C. Psychological Burden

• Anxiety linked to toxicity, uncertainty, and treatment interruptions

• Impact on independence, autonomy, and identity

D. Financial Burden

• Out-of-pocket costs

• Loss of income or productivity

• Transportation and caregiver expenses

E. Healthcare System Burden

• Supportive care requirements

• Hospitalizations

• Infusion chair time and staffing needs

These domains shape outcomes just as much as biological activity.

Why Elderly and Comorbid NSCLC Patients Need This Metric Most

NSCLC disproportionately affects elderly individuals who often:

• Cannot tolerate high-dose systemic regimens

• Have limited cardiopulmonary reserve

• Live with chronic illnesses such as COPD, heart disease, and diabetes

• Face mobility or cognitive limitations that complicate treatment adherence

Yet many modern oncology trials underrepresent these patients, limiting our understanding of true burden in real-world populations (Hutchins et al., Cancer, 1999).

Putting patient burden at the center of development ensures innovation that is designed for the actual population receiving care, not an idealized subset.

A Call to Action: How the Industry Should Integrate Burden Into R&D

1. Build Burden Metrics Into Early Development

Candidate selection should consider complexity of administration, logistical demands, and likely patient tolerability — not just biological potency.

2. Prioritize Low-Burden Modalities

Therapies that reduce systemic toxicity, simplify administration, or target the disease more directly align with real-world needs.

3. Redesign Clinical Trials to Reflect Clinical Reality

Trials should oversample older adults, measure burden explicitly, and incorporate patient-reported outcome tools.

4. Collaborate With Health Systems and Payers

Early dialogue ensures the value proposition reflects not only clinical benefit but also operational and financial sustainability.

5. Educate Clinicians and Stakeholders on the Burden Mindset

Changing what we measure will change what we value — and ultimately what we deliver.

Conclusion

Cancer innovation has long centered on discovering new molecules and achieving incremental survival gains. But as cancer incidence grows, and as the patient population ages, the burden of treatment has become just as critical as efficacy and safety.

By making patient burden a core development metric — particularly for elderly and comorbid NSCLC patients — the oncology community can accelerate progress toward therapies that are effective, tolerable, and accessible to those who need them most.

True innovation in oncology is not just about extending life — it is about ensuring that patients can live through treatment with dignity, independence, and quality.

References

• Hurria A. et al., Journal of Clinical Oncology, 2014

• Owonikoko T. et al., Journal of Clinical Oncology, 2014

• Basch E. et al., JAMA, 2017

• Atun R. et al., The Lancet, 2015

• Schnipper L. et al., Journal of Clinical Oncology, 2015

• Wildiers H. et al., Journal of Clinical Oncology, 2010

• SEER Cancer Statistics Review, 2023

• Kimmick G. et al., The Oncologist, 2015

• Tefferi A. et al., Mayo Clinic Proceedings, 2015

• Hutchins L. et al., Cancer, 1999