Global Perspectives on the Economics of Cancer Care

VBCM - Meeting Highlights - EHA 2013
Lisa A. Raedler, PhD, RPh

Stockholm, Sweden—A joint session of the European Hematology Association (EHA) and the American Society of Hematology (ASH) was held during the 18th Annual Congress of EHA on June 15, 2013. Ulrich Jäger, MD, President of EHA and Medical University of Vienna, Austria, and Janis L. Abkowitz, MD, President of ASH and University of Washington, Seattle, moderated a well-attended plenary session titled, “Challenges Associated with Delivering Cancer Care in Times of Economic Crisis.”

As they introduced the problem of limited access to cancer therapeutics, Drs Jäger and Abkowitz acknowledged the challenges of the aging population and the plethora of new and expensive agents. They also highlighted the striking disparity in cancer care and drug access around the globe. European countries, for example, operate under varying public healthcare systems and payment models, and use distinct methods for assessing new technology. The US system, by contrast, is in a state of flux as controversial healthcare reforms are realized over the next several years. Regardless of one’s geography and reimbursement model, unequal access to cancer care remains problematic throughout the world.

UK Perspective

Despite its small geographic size, the system of government-sponsored healthcare delivery and the robust technology assessment process in the United Kingdom serve as models to which other countries’ healthcare agencies and pharmaceutical industries pay significant attention. Peter Clark, MA, MD, Chair of the UK’s National Health Service (NHS) Chemotherapy Clinical Reference Group—a committee that provides clinical advice regarding cancer drug assessments to the NHS—reviewed the delivery of cancer care and the cancer treatment evaluation process in the United Kingdom.

Established by the NHS in 1999, the National Institute for Health and Care Excellence (NICE) is charged with establishing and updating treatment standards to ensure equal patient access to modern treatments. One aspect of this is the explicit determination of cost-effectiveness boundaries for new technologies and new drugs. Dr Clark noted that rather than focusing on affordability or on the impact of a new agent on the NHS drug budget, NICE asks, “What do we get for the price?” NICE’s assessment accounts for opportunity costs as well. “What will be displaced?” Multiple stakeholders, including physicians and patients, contribute to all new product assessments.

To measure the health benefits associated with a new cancer therapy, NICE uses the measure of quality-adjusted life-years (QALYs). The relative health benefit of the new treatment is calculated by comparing the present value of expected QALYs with and without the new treatment to the value of older treatments. When combined with the relative cost of the new treatment, this information forms the incremental cost-effectiveness ratio (ICER):

However, the key outcome parameters needed to determine the QALYs for a new cancer therapy—overall survival (OS) and quality of life (QOL)—are often missing or incomplete. Dr Clark lamented that clinical data supporting cancer drug registration often include short-term data (ie, overall response rate and/or progression-free survival), but not OS data. Similarly, QOL data are either not measured or are measured poorly.

NICE accepts as cost-effective interventions and therapies with an ICER of less than £20,000 per QALY. Increasingly compelling reasons are needed for accepting as cost-effective interventions with an ICER threshold of more than £30,000 per QALY.1 Rare diseases, including advanced cancer, are the exception, given patients’ short life expectancy.

Dr Clark emphasized that although NICE approves approximately 80% of all new drugs and interventions, only 50% of cancer drugs have been approved in recent years. Common reasons for cancer therapy denials by NICE include indeterminate ICERs per the provision of crossover data (ie, inability to assess the drug’s individual impact on OS), efficacy data that are very reliant on subgroup (vs intent-to-treat) analyses, and/or high prices (including the lack of price discounts). Dasatinib (Sprycel) and ruxolitinib (Jakafi) were not approved by NICE, because their manufacturers chose not to offer discounted pricing in the United Kingdom.

We cannot underestimate the importance of price discounting—known as “patient access schemes”—in the approval of new cancer therapies by NICE, according to Dr Clark. He noted that pharmaceutical manufacturers’ willingness to discount new drug prices (sometimes up to 55% using straightforward calculations) has substantially enhanced UK patients’ access to new and innovative medications. Although actual discount rates are “mostly confidential,” Dr Clark described “substantial” and “increasing” price reductions. Using a “loss leader” pricing approach, oncology drug makers are apparently willing to sacrifice revenue in the UK market for the ability to leverage their brand’s positive NICE review in communications directed toward other countries.

Dr Clark predicted that the “rational rationing” approach used by NICE will become more pervasive among government organizations of other European countries. Although each country has its own economic system and drug review processes, all face similar issues with rising healthcare costs and “economic gloom.” He speculated that today’s variable pricing agreements among individual countries and pharmaceutical manufacturers may become more consistently based on rigorous cost-effectiveness analyses. For pharmaceutical companies, this translates into a strong need to design clinical trials with “an eye on reimbursement,” which means accurate measurement and robust analysis of QOL.

He admonished practicing hematologists and oncologists to confront the reality of healthcare costs and the need for the rationing of cancer care. “Healthcare spending is unsustainable today. It is not something that is coming tomorrow. It is here.”

Providers managing patients with cancer, he believes, are best served by learning about and actively engaging with their governments’ reimbursement decision-making processes. It is critical for providers to understand not only the clinical benefits of a new therapy but also the assumptions that are made in cost-effectiveness analyses. He cited examples in which oncologists have engaged with pharmaceutical companies, including cases in which the companies have been “tackled” regarding drug pricing.

EHA, ASH, and other hematology/oncology organizations, Dr Clark believes, can and should work together to ensure appropriate product access: share knowledge and best practices, as well as specific cases of successful access and reimbursement.

French Perspective

To preface his remarks, Laurent Degos, MD, PhD, past Chair, French National Authority for Health Board, and Professor of Hematology, Université Paris Diderot, France, reviewed the current “nonuniformity” of health technology assessment approaches used in European countries, as well as the social values of these approaches, as outlined below.

Country Relevant social values Health technology assessment model
England Utility: opportunity cost (quality-adjusted life-years) Cost-quality threshold (<£20,000 per quality-adjusted life-years is reimbursed); utility compared among diseases
Germany Responsibility: willingness to pay Cost-effectiveness: price ceiling is compared with previous drugs for a disease
France Equal access: immediate use and later health technology assessment of precise indications Cost-benefit: multiple parameters are considered (indications, patient volume); price and reimbursement are fixed at an early stage
The Netherlands Regulated competition Multiple insurance companies; supply and demand drive pricing decisions

On the basis of these countries’ collective experiences, Dr Degos recommended moving away from segmented systems of care for patients with cancer. Instead, he argued that healthcare professionals and patients should focus on the care “cycle” or care continuum. By using regionally relevant clinical pathways that consider a drug’s safety, efficacy, and accessibility (reimbursement), he believes that cancer care providers can ensure integrated patient care with optimized outcomes.

Critical questions related to payment in this type of a paradigm shift in cancer care include who will pay, and how? Dr Degos summarized the many “experiments” that are currently under way to assess funding for this “full cycle of care.” The UK’s NHS convenes experts to participate in clinical commissions that are tasked with developing cancer treatment guidelines. Physician practices in the United States are moving toward accountable care organizations that receive bundled payments for patient care. France is evaluating the role of regional health agencies. Dr Degos concluded by predicting that “new politics, new policies, and new regulation” will transform our collective approach to health technology assessment and to cancer care delivery.

The Patient Perspective

Nikos Dedes, a founding member and President of Positive Voice, the Association of People Living with HIV in Greece, was recently appointed a member of the Management Board of the European Medicines Agency (EMA). Mr Dedes concluded the session by offering the patient perspective on the economics of cancer care and on the need for equal access to care throughout the world. He began by stating, “We [as patients with cancer] face a crisis. We have to do more than expect everything.” Cancer care delivery that is sustainable and equitable to all is no longer financially viable.

Mr Dedes asked patients with cancer and their advocates to engage with public and private payers, as well as with the pharmaceutical industry, on these critical questions of access and funding. He substantiated this recommendation by highlighting the poignant example of the patients with HIV community in the early 2000s. In 2003, the prevalence of HIV in Africa was the highest in the world. At the same time, HIV-positive African patients were least able to access antiretroviral therapies that had been identified and approved for use in patients with HIV more than 7 years earlier. Mr Dedes recalled a 2003 quote from Nelson Mandela to illustrate the unacceptability of this situation, “We have failed to translate scientific knowledge into action. This is a global injustice; a travesty of human rights.” Over time, persistent advocacy and lobbying regarding this disparity in HIV drug access compelled pharmaceutical manufacturers to make their products more affordable. Pharmaceutical companies’ solutions included tiered-pricing strategies (ie, lowered medication prices for patients in developing countries), the issuance of voluntary licenses to South African generic drug manufacturers, and drug donation programs.

Today, according to Mr Dedes, we are in a similar predicament with innovative cancer therapies and other expensive medications. He cited 2011 data from IMS Health regarding the use of “valuable, innovative” medicines throughout Europe. This analysis demonstrated a wide disparity in the sales of 11 “innovative” drugs (per 100,000 inhabitants), including bortezomib (Velcade), in 25 countries for which data were available. There was a 35-fold difference in innovative drug sales between selected Western European countries (ie, France, Denmark, and Sweden) and certain Eastern European countries (ie, Poland, Latvia, and Estonia). Mr Dedes concluded by admonishing that as members of the oncology community, we all “need to do whatever is necessary” to overcome this injustice.

In the discussion session, speakers agreed that, ideally, country representatives will agree to put aside differences in philosophy, politics, tax systems, and reimbursement approaches to focus on the vital need for universal access to cancer medications. Keys to success will include active engagement of all principal stakeholders (ie, governments, pharmaceutical companies, and civil society, including consumers), as well as a willingness to risk-share in cost-effectiveness data collection, analysis, and decision-making. Pharmaceutical manufacturers will also need to continue to innovate on pricing (discounting) and to actively consider novel reimbursement strategies, such as “pay for performance.”

To conclude the session, speakers urged the audience—members of EHA, ASH, and other cancer care specialist societies—to advocate for change, “Get involved. Share information. We have to act fast.”

Reference

  1. National Institute for Health and Clinical Excellence (NICE). Incorporating health economics in guidelines and assessing resource impact. In: The Guidelines Manual. London, United Kingdom: NICE; 2007:49-55. www.nice.org.uk/niceMedia/pdf/GuidelinesManualChapter8.pdf. Accessed June 16, 2013.
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