Cost Effectiveness vs. Affordability in the Age of Targeted Drug Therapies

A special issue of Current Oncology (ISSN 1718-7729). This special issue belongs to the section "Health Economics".

Deadline for manuscript submissions: 15 March 2026 | Viewed by 2735

Special Issue Editor


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Guest Editor
1. Department of Oncology, McGill University, Montreal, QC H3A 0G4, Canada
2. Division of Medical Oncology, McGill University Health Centre, Montreal, QC H4A 3J1, Canada
Interests: cancer related drug; medical oncology; GU malignancies; sarcoma

Special Issue Information

Dear Colleagues,

In this golden age of targeted drug therapies, the distinction between cost-effectiveness and affordability is crucial in cancer treatment. While groundbreaking in their precision, targeted therapies often come with high price tags that strain healthcare systems and patients alike. Cost-effectiveness refers to the value these drugs offer relative to their cost, typically measured by outcomes like extended survival or improved quality of life. Even when a therapy is deemed cost-effective, it does not necessarily mean it is affordable for patients or sustainable for health systems.

Affordability, conversely, is about the immediate financial impact on patients and healthcare providers. A drug may be cost-effective in the long term but still unaffordable for individuals due to high out-of-pocket expenses or insurance limitations. This growing gap between cost-effectiveness and affordability highlights the need for innovative pricing strategies, better insurance coverage, and policy interventions that ensure equitable access to these life-saving treatments. As we advance in personalized medicine, balancing the promise of targeted therapies with their economic realities remains a pressing challenge for oncologists, policymakers, and patients.

We encourage submissions of original research, reviews, case studies, highly novel case reports, retrospective studies, or perspectives that align with these themes.

Dr. Ramy Saleh
Guest Editor

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Keywords

  • cost-effectiveness
  • affordability
  • drugs
  • financial
  • cancers
  • treatment
  • targeted drug therapies

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Published Papers (3 papers)

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14 pages, 523 KiB  
Article
Epidemiology, Treatment Patterns, Survival, Healthcare Resource Utilization, and Costs of Dedifferentiated Liposarcoma (DDLPS) in Canada: A Retrospective Cohort Study Using Administrative Databases in Ontario
by Soo Jin Seung, Anisia Wong, Raymond Milan, Nisha Chandran and Albiruni R. Abdul Razak
Curr. Oncol. 2025, 32(5), 273; https://doi.org/10.3390/curroncol32050273 - 9 May 2025
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Abstract
Background: Dedifferentiated liposarcoma (DDLPS) is a rare, aggressive tumour with poor survival outcomes in advanced settings. This study assessed the incidence/prevalence, treatment patterns, survival, healthcare resource utilization (HCRU), and costs for DDLPS patients in Ontario, Canada. Methods: A retrospective cohort study was conducted [...] Read more.
Background: Dedifferentiated liposarcoma (DDLPS) is a rare, aggressive tumour with poor survival outcomes in advanced settings. This study assessed the incidence/prevalence, treatment patterns, survival, healthcare resource utilization (HCRU), and costs for DDLPS patients in Ontario, Canada. Methods: A retrospective cohort study was conducted among DDLPS patients between 2010 and 2022 using administrative databases. Overall survival, all-cause HCRU, and costs (2023 Canadian dollars, CAD) were compared based on advanced disease and resection status. Results: The overall incidence and cumulative prevalence of DDLPS was 0.465 and 1.995 per 100,000 people, respectively. Of all 611 DDLPS cases (64.3% male, median age [IQR]: 67 [57–76] years), 40.3% and 61.0% had advanced and unresected disease, respectively. The median overall survival (mOS) was 69 months [IQR = 15–151] for the entire cohort, but this was significantly lower for advanced and unresected disease (p < 0.0001). Among patients receiving systemic treatments (N = 117), 81.2% were prescribed doxorubicin as first-line treatment. All-cause healthcare costs (2023 CAD) amounted to CAD 34,448 per person-year (PPY), with inpatient hospitalizations being the highest cost driver at CAD 14,522 PPY and 0.8 inpatient hospitalization PPY for all years. Advanced disease had higher HCRU and costs. Conclusions: This is the first comprehensive real-world evidence study that quantifies the high mortality and cost burden associated with DDLPS in Canada. Full article
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12 pages, 980 KiB  
Brief Report
Accelerating Oncology Drug Reimbursement in Canada: Impact of the CDA-AMC Time-Limited Recommendation and pCPA Temporary Access Process
by Catherine Y. Lau, Arif Mitha and Allison Wills
Curr. Oncol. 2025, 32(4), 235; https://doi.org/10.3390/curroncol32040235 - 17 Apr 2025
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Abstract
The complex pathway for new drug reimbursement in Canada has been well documented. Drugs with promising early efficacy data may receive a Notice of Compliance with Conditions (NOC/c) from Health Canada. For oncology drugs that receive NOC/c, the pathway through positive review by [...] Read more.
The complex pathway for new drug reimbursement in Canada has been well documented. Drugs with promising early efficacy data may receive a Notice of Compliance with Conditions (NOC/c) from Health Canada. For oncology drugs that receive NOC/c, the pathway through positive review by Canada’s Drug Agency (CDA-AMC) and subsequent public reimbursement can take over 500 days. To address this challenge, in September 2023, CDA-AMC announced a new Time-Limited Recommendation (TLR) category, and in parallel, the pan-Canadian Pharmaceutical Alliance (pCPA) developed a set of principles and conditions for a Temporary Access Process (pTAP). This accelerated access pathway, the first of its kind in Canada, enables patients with advanced diseases to gain timely access to promising therapies while managing the uncertainties and risks associated with early approvals. This report provides a first assessment of the impact of the TLR-pTAP process on the reimbursement timelines for oncology drugs approved with NOC/c. Methods: The time from NOC/c approvals for oncology drugs between 1 January 2023 to 31 December 2024, to first provincial listings, and the timelines of the Health Canada, CDA-AMC, and pCPA review processes, were collected and evaluated. Results: Nine oncology NOC/c were granted during the selected period, of which three products, Columvi, Akeega, and Epkinly, received provincial listings, and the median time from regulatory approvals to provincial listings is 509 days (IQ range 306–544 days). One drug, Epkinly, has elected to adopt the TLR-pTAP pathway. Compared to the conventional reimbursement pathway—including for the drug Columvi, whose therapeutic profile is similar to that of Epkinly—the new pathway reduced the time to first provincial listing by over 200 days. A stepwise analysis indicates that the most significant accelerator within the TLR-pTAP pathway is the pCPA’s prioritization and processing of the file in parallel to the CDA-AMC’s health technology assessment (HTA) review process, rather than subsequently. Electing to file the HTA submission pre-NOC could have further accelerated timelines. No acceleration in each agency’s review time was observed. Conclusions: Participation in the TLR-pTAP pathway can help mitigate concerns over uncertainties associated with novel therapies while providing timelier access for patients with life-threatening diseases. Full article
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12 pages, 593 KiB  
Systematic Review
Does Real-World Evidence of the Economic Burden of Lung Cancer in Greece Exist? A Systematic Review of the Literature
by George Gourzoulidis, Catherine Kastanioti, George Mavridoglou, Theodore Kotsilieris, Dikaios Voudigaris and Charalampos Tzanetakos
Curr. Oncol. 2025, 32(3), 130; https://doi.org/10.3390/curroncol32030130 - 25 Feb 2025
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Abstract
Objective: This systematic literature review aimed to summarize the economic burden of lung cancer in Greece, identify current data gaps, and support the design of future real-world studies. Methods: A systematic search of studies published in English on the cost of lung cancer [...] Read more.
Objective: This systematic literature review aimed to summarize the economic burden of lung cancer in Greece, identify current data gaps, and support the design of future real-world studies. Methods: A systematic search of studies published in English on the cost of lung cancer was performed in MEDLINE-(PubMed), Scopus, and ScienceDirect. The databases were searched until September 2024, and records were screened based on our eligibility criteria. After conducting the initial literature search, the abstracts and full texts of the identified studies were reviewed and evaluated for inclusion based on predefined criteria. Data from the selected studies were then extracted into a standardized form and subsequently synthesized. Results: Seven studies were included in this review. The reported burden was sourced from hospital data and categorized as direct and indirect costs. Most studies (n = 6) reported direct costs, with one study reporting both direct and indirect costs. The total direct medical cost per patient increased from approximately EUR 16,000 in 2015 to EUR 58,974 in 2023, with drug acquisition costs being the key driver of the total direct cost. Additionally, the cost of end-of-life care during the final six months of a patient’s life was estimated to range from EUR 6786 to EUR 7665 per patient, with pharmaceutical costs comprising the largest proportion of the total cost. One study also reported that indirect costs were considerably higher for patients than for family caregivers. Conclusion: The economic burden of lung cancer has increased substantially over the past decade in Greece. The present systematic review emphasizes the critical need for comprehensive real-world studies on the economic burden of lung cancer in Greece. Addressing the current gaps holistically will yield invaluable insights for policymakers and stakeholders. Full article
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