Perceptions of Oncologists, Healthcare Policy Makers, Patients and the General Population on the Value of Pharmaceutical Treatments in Oncology

Cancer is one of the leading causes of morbidity and mortality worldwide, with 14.1 million new cancer cases and 8.2 million cancer deaths [1]. It is estimated that within two decades, cancer rates will double to approximately 22 million cases [2]. Regardless of this increase in the number of new cancer cases each year, patients’ survival has improved due to the drugs that are currently available, together with the progress of cancer prevention and diagnostics [2].

The study and understanding of molecular mechanisms related to cancer has allowed identifying and developing novel target treatments. Some new therapies delay tumor growth, opening up the prospect for patients’ long-term survival and turning cancer into a chronic disease [3]. At the same time, expenditure on cancer drugs has grown steadily [4]. It has been argued that the high cost of contemporary cancer drugs may signal even greater costs for future drugs [5]. Consequently, the debates regarding the rise of healthcare costs and the development and value of new medical technologies are increasingly present.

In today’s economic context, governments have the challenge to provide high quality and innovative care to meet the population’s health needs most effectively while managing health care budgets and safeguarding the basic principles of equity, access and choice. For this reason, to control healthcare costs, governments need to apply evidence-based principles in evaluating the new medicines, often using “additional cost per additional health benefit” to measure “value for money” [6].

Currently, there is intense debate regarding the value considerations of new cancer drugs. The American Society of Clinical Oncology (ASCO) has proposed a conceptual framework to assess the value of cancer treatment options through their clinical benefits (efficacy), toxicity (safety) and cost (efficiency) [7]. Moreover, the European Society for Medical Oncology (ESMO) has undertaken the development of a validated and reproducible tool to assess the magnitude of the clinical benefits of anti-cancer interventions, the ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS) [8]. There is a growing need to define and adopt explicit decision criteria in the choice of new cancer drugs, and there is an agreement on the need to consider the opinions and preferences of the society. Although the definition and adoption of explicit decision criteria in the choice of new cancer drugs is a major breakthrough, it is important to note that the assessment of the value of any cancer treatment (efficacy, safety, quality of life, cost) may be different depending on healthcare systems and stakeholders. Therefore, decisions related to resource allocation should reflect the opinion and preferences of the society. Including citizen input in government priority-setting approaches is becoming more relevant and there is growing demand for governments to make resource allocation decisions in line with social values. Some studies have reported that in general, citizens and payers priorities for funding a new cancer drugs are different. Payers prioritize efficiency defined as health gains per dollar, while general population or patients prioritize equity understood as equal access to medicines independent of cost or effectiveness [9].

Understanding the value of a new cancer drug from different perspectives, including those of the general population and patients, can provide insight regarding the factors that should determine the value of new oncologic treatments. The objective of this work was to conduct an exploratory and qualitative study to investigate the main factors that drive innovation in oncology, and to determine the importance of each attribute from different perspectives: healthcare policy makers, oncologists, patients and the general population in Spain.

In cancer care, value is often understood as treatment benefits or quality-of-care weighed against economic cost. Some approaches have been developed in order to define the value of new cancer drugs. The conceptual framework proposed by ASCO is based on clinical benefits (efficacy), toxicity (safety) and cost [8], while ESMO-MCBS is mainly based on the magnitude of clinical benefit, QoL and toxicity [9]. Despite these approaches, value considerations may differ among stakeholders. The results of this study, that aimed to explore the main factors that drive innovation in oncology from different perspectives, revealed that gains in QoL, survival, safety, cost and innovation are perceived differently by different groups of stakeholders. For example, although improvements in QoL are considered an important added value for new cancer drugs by all the respondents, there were differences among participants regarding the value assigned to QoL when compared to survival.

Differences were also observed related to economic factors and safety. Healthcare policy makers gave greater importance to the economic impact related to oncology treatment costs than other participants. Compared with other participants, a higher proportion of patients agreed to select a treatment that provides additional survival despite increasing the risk of side effects.

Similar results are described in other studies. In the United States, study findings revealed that oncologists value length of survival highly compared to improvements in QoL when taking chemotherapy decisions [14]. A study conducted in Israel identified differences in the perception of the value of cancer health among family physicians and oncologists [15]. Family physicians valued life-prolonging and QoL-enhancing interventions roughly equally, while oncologists valued interventions that extended survival highly compared with those that improved only QoL. It has been suggested that these findings may have important implications in the coverage and reimbursement decisions of new cancer treatments [14, 16]. Although achieving gains in survival was considered highly important among all participants, healthcare policy makers demanded a greater survival improvement, compared to the other respondents, in order to justify the added value of an innovative cancer treatment.

With regard to cancer drug cost, some studies had reported that cancer patients are largely desensitized to the cost of their treatment, mainly because of their insurance coverage, meaning that drug prices are not significantly constrained by patients’ willingness to pay [17] and in some cases, the value of treatment exceeds the total amount paid by them and their insurer [18]. However, nowadays, mainly due to the economic sustainability of health system has become a cornerstone of global economic policies, this is changing. The rising cost of cancer drugs is creating unintended financial consequences for patients. Cancer patients must face potential financial stresses related to out-pocket expenditures (medical or non-medical expenses), loss of earnings (patient and relatives), being increasingly more concerned about cancer drug cost [19, 20].

In line with our results that showed that all participants agree that patients should receive information regarding the budgetary impact of cancer treatment, even though they do not have to pay for it, some studies conclude that many clinicians consider that cost information should be shared with patients rather than making clinical recommendations based on cost without patients’ input [21].

A review that aims to identify and compare the preferences of patients, the general public and payers to determine the values that should shape public funding decisions for new cancer drugs, suggests that payers consider many factors and prioritize efficiency in funding decisions, while patients and the general public consider fewer factors and prioritize access to cancer treatments with the potential to save or extend life [9].

Finally, as per the safety of new cancer drugs, a recent study that aimed to investigate the attitudes of physicians, healthy control subjects and melanoma patients towards a specific drug showed that patients strived for a longer life regardless of the side effects and tumor response rates, whereas physicians were reluctant to use therapies with a higher incidence of side effects and marginal cancer response benefits [22].

These findings need to be understood in the context of sample biases and ambit of the study. A convenience sample of general population was used, not being representative of the general population. Even though the sample size of the study is not very large, there is a vast range of sample sizes used in qualitative studies, with the most common sample size being between 20 and 30 interviews [10]. Further research is needed to establish the magnitude of meaningful differences in the views of cancer treatment innovation in the Spanish Health System.

This study describes an overall view of the similarities and differences in the gains in drug safety, QoL, survival, compliance with patients’ clinical characteristics and costs as a means of evaluating the perception of the added value of a new cancer treatment by different stakeholders in Spain. The differences observed in the study highlight the need to consider the perspective of different stakeholders in the assessment of a new cancer drugs to obtain more informed decisions when deciding on the most appropriate treatment to use, and contribute to the debate on the definition of the value of innovation in oncology addressing the different perspectives.

In conclusion, since QoL, survival, safety, cost and innovation are perceived differently by stakeholders, this should be considered during the assessment of a new cancer drug.