Burden of disease studies

The projects included in this pillar are closely related to analyses of the epidemiological and treatment cost burden of different cancer types, the cost of cancer drug development, and the socioeconomic impact of cancer on patients and families.

In line with the original intent to establish the DKFZ as the leading German competence center and a national point of reference in the field of cost of cancer and cancer care, a series of reviews was initiated with Forum, the official journal of the German Cancer Society (Deutsche Krebsgesellschaft, DKG), covering the available evidence on the burden of disease and societal and health care costs of the most prevalent cancers. Of these publications, two consisted of the principles of measuring cancer costs in Germany, and three involved epidemiological and costing estimation reviews for major cancer types in Germany. In addition, a literature review of the economic burden of pancreatic cancer was expanded to Europe.

Selected Publications:

D. Hernandez, F. Wagner, K. Hernandez-Villafuerte, M. Schlander:
Economic burden of pancreatic cancer in Europe: a literature review.
Journal of Gastrointestinal Cancer, published online ahead of print 2022 Apr 26.
DOI: 10.1007/s12029-022-00821-3

D. Hernandez, T. Ran, K. Hernandez-Villafuerte, M. Schlander:
Krankheitslast von Darmkrebs in Deutschland: Epidemiologie und Kosten.
Forum, 2022: published 24 Nov 2022.
DOI: 10.1007/s12312-022-01154-9.

D. Hernandez, P. Giri, A. von Both, M. Schlander:
Krankheitslast von Lungenkrebs in Deutschland: Epidemiologie und Kosten [Lung cancer disease burden in Germany: epidemiology and costs].
Forum, 2022: 37, 387-404.
DOI: 10.1007/s12312-022-01123-2.

D. Hernandez, A. von Both, M. Schlander:
Krankheitslast von Prostatakrebs in Deutschland: Epidemiologie und Kosten [Disease burden of prostate cancer in Germany: epidemiology and costs].
Forum, 2022: 37 227-234.
DOI: 10.1007/s12312-022-01075-7.

D. Hernandez, K. Hernandez-Villafuerte, M. Schlander:
Die Messung der Kosten von Krebserkrankungen in Deutschland. Teil 2 – Wirtschaftliche Belastung [Determining the cost of cancer in Germany. Part 2—economic burden].
Forum, 2022: 37 42-48.
DOI: 10.1007/s12312-021-01026-8.

D. Hernandez, K. Hernandez-Villafuerte, M. Schlander:
Die Messung der Kosten von Krebserkrankungen in Deutschland: Teil 1 – Epiemiologie und Krankheitslast [Determining the cost of cancer in Germany. Part 1 – epidemiology and disease burden].
Forum, 2021: 36 406-410.
DOI: 10.1007/s12312-021-00988-z.

M. Schlander, K. Hernandez-Villafuerte, C. Thielscher:
Cost of cancer in Germany [Kosten der Onkologie in Deutschland].
Forum, 2018: 33 (5) 330-337.
DOI: 10.1007/s12312-018-0481-5

There is only limited information available on the real-life patterns of health care utilization and associated costs attributable to a diagnosis of lung cancer in Germany. We aim to identify health care utilization patterns and cost estimation of lung cancer treatments by making use of administrative claims data from one of the largest health insurance companies in Germany. In a first study, we introduce the identification strategy of lung cancer patients and we estimate lung cancer incidence in Germany addressing different patient characteristic, as well as lung cancer survival in relation to comorbidities. Furthermore, in two additional studies, first, we measure medication costs and identify prescription patters of lung cancer patients in Germany. And second, we estimate attributable inpatient costs and examine impatient treatment patterns of lung cancer in Germany.

Preliminary results suggest that impatient costs attributable to lung cancer are around €25,000 per patient and close to €18,000 per year survived. Large differences were identified by age groups and place of residence, while sex and comorbidities did not seem to affect cost figures. Length of stay declined considerably over the 12-year period analyzed. Moreover, outpatient prescription costs are estimated at approximately €12,000 per patient, being these lowest during the initial phase of treatment and highest in the terminal phase. Outpatient prescription costs per patient were also observed to increase significantly within the time frame of analysis.

The high cost of research and development (R&D) of new cancer drugs is a frequently cited yet controversial reason for high drug acquisition costs. It also concerns the viability of the business model of the R&D-based biopharmaceutical industry. We analyzed the peer-reviewed literature on the cost to bring a new molecular entity (NME) to market and explored reasons for the wide variation of published estimates. Total average capitalized pre-launch R&D costs were reported to range from $161 million to $4.54 billion (2019 US dollars) Therapeutic area-specific estimates were highest for anticancer drugs (between $944 million and $5.4 billion). Our analysis confirmed a trend of increasing R&D costs per NME over time. Beyond the quantitative analysis, we developed a suitability scoring system to help improve the validity of future research in the field. Extended in-depth analyses of strategic success factors and cost of clinical R&D are being prepared.

Immunotherapy such as chimeric antigen receptor (CAR) T cell therapy is a promising treatment option for some blood cancers, with the possibility of cure. In August 2018, the European Commission approved the use of two genetic gene therapies for oncology indications. We evaluated the costs associated with CAR-T cell therapy, including production costs and costs related to preparation, treatments, and toxicity management, using micro-costing and gross-costing methods. Our results showed for the first time that decentralized production in not-for-profit environments can be less costly and more efficient alternative to the current centralized production mode. Given economies of scale and scope as well as learning curve effects, such as process improvements, we predict father cost reductions of decentralized CAR T-cell production with increasing standardization. This exception is commensurate with the early life cycle stage of this new technology. Our findings may inform pricing and reimbursement policies, and we will share our insights with international partners, such as the "European Fair Pricing Network" (EFPN). For future studies of the overall cost of CAR t-cell therapies, clinical partners are sought.

Selected Publications:

D. Hernandez, C.-Y. Cheng, K. Hernandez-Villafuerte, M. Schlander:
Survival and comorbidities in lung cancer patients: evidence from administrative claims data in Germany.
Oncology Research, 2022: 30(4), 173-185.
DOI: 10.32604/or.2022.027262.

T. Ran, S. B. Eichmüller, P. Schmidt, M. Schlander:
Cost of decentralized CAR T cell production in an academic non-profit setting.
International Journal of Cancer, 2020: 147 (12) 3438-3445.
DOI: 10.1002/ijc.33156.

T. Ran, S. B. Eichmüller, P. Schmidt, M. Schlander:
Reply to: Comments on "Cost of decentralized CAR T cell production in an academic non-profit setting".
International Journal of Cancer, 2021: 148 (2) 516-517.
DOI: 10.1002/ijc.33253.

M. Schlander, K. Hernandez-Villafuerte, C. -Y. Cheng, J. Mestre-Ferrandiz, M. Baumann:
How much does it cost to research and develop a new drug? A systematic review and assessment.
Pharmacoeconomics, 2021: 39 (11) 1243-1269.
DOI: 10.1007/s40273-021-01065-y.

M. Schlander, T. Ran, P. Schmict, S.B. Eichmüller:
Can we reduce the acquisition cost of CAR T cell therapies?
Poster presentation at the ESMO Congress 2020.
Virtual. September 19-21, 2020.
Annals of Oncology, 2020: 31(Supplement 4) S966.
DOI: 10.1016/j.annonc.2020.08.1912.

Health economists are familiar with various perspectives of cost analyses; besides the "societal" and the "payers"' perspectives, sometimes an employer perspective, a citizen's perspective, and the perspective of patients are cited. In applied health economics, the latter perspective has remained mostly lip service. While in the United States the "financial toxicity" of cancer treatments has gained increasing attention, there is a wide-spread perception that European welfare states offer cancer patients effective protection from financial hardship. Yet, also many patients in European countries, which provide universal health care, face financial hardship; their "objective" financial burden is driven by direct medical and non-medical expenditures (including co-payments) as well as by indirect costs, i.e., loss of income (which may not be compensated for by social transfers). Resulting intangible costs include psychological distress, decreased quality of life, and impaired clinical outcomes, including higher mortality. Since outcomes exceed "financial toxicity", we propose using the broader (and more neutral) term "socioeconomic impact" to comprehensively address the financial consequences and their impact on cancer patients (and cancer survivors) and their relatives (for example, caregivers and dependents).

European research into the socioeconomic impact of cancer and cancer care is still in an early stage, and it has been characterized by substantial heterogeneity. Inconsistent use of terminology in existing studies can be traced back to the absence of a coherent conceptual framework for "socioeconomic impact analyses". Thus, in March 2021, we inaugurated an international Task Force within the framework of the OECI Health Economics Working Group under the leadership of Michael Schlander (chair) and Wim van Harten (co-chair), with the objectives to develop a European consensus for a comprehensive conceptual framework, a consistent taxonomy and terminology, and measurement instrument development validation for socioeconomic impact analysis from the perspective of cancer patients, in order to ultimately reduce or eliminate the risk of wasted research efforts due to redundant, overlapping, and incomparable work owing to unnecessarily heterogeneous use of terms and definitions, and to support and facilitate further research in the field.

Further studies into the socioeconomic impact of cancer has been initiated in parallel. Beyond related studies in the context of the COVID-19 pandemic, an analysis using data from the Socio-Economic Panel (SOEP), one of the largest and most comprehensive household surveys in Germany, demonstrated that average job incomes drop between 26% and 28% within the year after a cancer diagnosis was reported. In another project, we analyzed data from the CAESAR study in collaboration with the DKFZ Unit of Cancer Survivorship (Volker Arndt). We assessed the out-of-pocket payments and income losses of breast cancer survivors in Germany and identified their common drivers.

In the context of the OECI Task Force, a number of projects were initiated. These include a bibliometric review, an analysis of terminology and a proposal for a consistent taxonomy, a best-fit framework synthesis, and an educational paper providing an overview of the use of patient-reported outcomes (PROs) in oncology. A further ongoing project concerns the development and validation of a patient-reported outcome (PRO) instrument to measure the socioeconomic impact of cancer on German (and European) patients and their relatives, based upon the coherent conceptual framework developed by the OECI Task Force. The instrument should reflect the relationships among the dimensions of socioeconomic impact on patients / households and their relatives. Also extensions of the original SOEP-based analysis of income losses are being planned.

Selected Publications:

V. Arndt, D. Hernandez, M. Schlander:
Einkommenseinbußen nach Krebs – Ergebnisse aus Deutschland.
Onkologie, 2022: 28 951-956.
DOI: 10.1007/s00761-022-01243-3.

R. Eckford, K. Hernandez-Villafuerte, A. Spier, L. Hirschberg, M. Schlander:
A systematic review of the socioeconomic impact of cancer on patients and families: Are differences among countries being effectively captured and addressed?
Poster presentation at ISPOR Europe 2020.
Virtual. November 16-19, 2020.
Value in Health, 2020: 23(Supplement 2) S478.
DOI: 10.1016/j.jval.2020.08.453.

D. Hernandez, M. Schlander:
Income loss after a cancer diagnosis in Germany: An analysis based upon the socio-economic panel survey.
Cancer Medicine, 2021: 10 (11) 3726-3740.
DOI: 10.1002/cam4.3913.

K. Hernandez-Villafuerte, R. Eckford, A. Spier, M. Schlander:
How to Describe the Socioeconomic Impact of Cancer on Patients and their Families: An Evaluation of Terminology by Global Regions and Healthcare Systems.
Presentation at iHEA 14th World Congress on Health Economics.
Virtual. July 12-15, 2021.

P. D. Pham, J. Ubels, K. Hernandez-Villafuerte, R. Eckford, M. Schlander:
Developing a conceptual framework for socioeconomic impact research in European cancer patients: A best-fit framework synthesis.
Poster presentation at ISPOR Europe 2022.
Vienna, Austria. November 6–9, 2022.
Value in Health, 2022: 25(12, Supplement) S430.
DOI: 10.1016/j.jval.2022.09.2141.

M. Schlander.
PRO ("patient-reported outcomes") und Lebensqualität in der Onkologie.
FORUM 2020; 35: 382-390.
DOI: 10.1007/s12312-020-00841-9.

J. Schneider, D. Hernandez, CAESAR study group, M. Schlander, V. Arndt:
Out-of-pocket payments and loss of income among long-term breast cancer survivors in Germany: A multi-regional population-based study.
Journal of Cancer Survivorship, 2022: 1-21.
DOI: 10.1007/s11764-022-01293-x