Percivil Melendez Carrera

Are current ICER thresholds outdated? Valuing medicines in the era of personalized healthcare

According to the Orphanet database, there are, at present, 495 drugs that have been granted orphan or ultra-orphan designation (i.e. affecting no more than 5 in 10,000 and 1 in 50,000 people, respectively [1]). Within the European Union (EU), 88 drugs have received market authorization from the European Medicines Agency (EMA), of which nearly one-third was granted in 2014 and 2015. Given that orphan drugs as well as cytotoxic chemotherapy – or cancer drugs – have been driving the number of new molecular entities approved by the EMA, and its counterpart in the USA, and cost many patients health systems dearly, the issue of access in the age of personalized healthcare is increasingly and inextricably linked to affordability and willingness to pay for better outcomes [2,3]. Indeed, in the absence of EU legislation to guide the prices and use of orphan and cancer drugs among member states, there are considerable differences in patient access to medicines that make the routine application of health technology assessment (HTA) more challenging [2]. Since the market for orphan drugs is small and their prices are associated with high monetary cost, these drugs rarely meet conventional reimbursement criteria, be they formal cost-effectiveness requirements or other therapeutic added value relative to cost hurdles [4]. This also holds for cancer drugs. A recent study which compared the cost-effectiveness of cancer versus noncancer drugs, for example, found that the mean and median of incremental cost-effectiveness ratio (ICER) calculated for an intervention using the former were

The financial hazard of personalized medicine and supportive care.

138,582/quality-adjusted life-year (QALY) and

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55,500/QALY, respectively, compared with

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49,913/QALY and

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31,000/QALY, respectively, for an intervention with noncancer drugs [5]. Among cancer drugs, 45% had ICERs below

Do-it-yourself health care.

50,000/QALY and 70% below

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100,000/QALY. Meanwhile, threshold ICERs based on reimbursement and coverage decisions range from £20,000/QALY in England to €80,000/QALY in the Netherlands [1,6]. While the simplicity and ease of application of the ICER threshold (to just about any drugs and health technologies) make it very appealing, one cannot escape the deficiencies of the QALY as an outcome measure, and the arbitrariness (of the derivation) of the threshold as illustrated in the case of

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50,000/QALY in the USA [7]. Fortunately, orphan, ultra-orphan, and cancer drugs are often reimbursed. In the Netherlands, for example, no drugs have been withheld because of their unfavorable costeffectiveness, as in the case of nivolumab in the treatment of non-small cell lung cancer. Moreover, a special program is in place to support hospitals financially in prescribing expensive orphan drugs. Meanwhile, England has promoted to facilitate patient access to cancer drugs not (routinely) available in the National Health Service through the Cancer Drugs Fund [8]. Although the policy on reimbursement of cancer and/or orphan drugs is recently revised to better capture value for money, there are essentially two thresholds in the UK, given distinct and separate budgets and differential weights on health outcomes. In both the Netherlands and England, negotiations with pharmaceutical manufacturers on effective/actual prices have been instrumental in facilitating reimbursement of and patient access to drugs. In the era of personalized healthcare, the evidence base for a new drug’s efficacy and safety is likely to be smaller, giving a relatively imprecise ICER. Indeed, a systematic review of orphan drugs in oncology found that drugs marketed in the USA have varying levels and quality of clinical evidence and a paucity of evidence regarding economic value [9]. As personalized healthcare advances, there will be more drugs developed targeting a molecular defect or single mutation found in small(er) subsets of patients to prolong survival as well as better quality of life (QoL), but may also generate smaller population benefits [7]. Were the size of the health budget and our collective willingness to pay to remain the same, are we ready to deal with the intended and unintended consequences of investment and disinvestment? Han et al. [10] found, for example, that Canadian physicians have resorted to, among other methods, falsifying claims on access forms to obtain unfunded oral chemotherapies for their patients. With new treatments developed for limited patient populations whose health outcomes may or may not be deemed commensurate to the high(er) price tags of medicines, and given that the majority of EU member states surveyed on their respective orphan drug regulations and policies have costeffectiveness as an HTA requirement [3], we need to deal

Primary Care Transformation as a Solution to the Epidemic of Chronic Diseases

Personalized medicine is revolutionizing the delivery of oncological care, promising benefits both at the patient and health system levels. The cost of targeted therapies, unfortunately, is becoming more expensive and unaffordable. Where supportive care in cancer concerns the prevention and management of the adverse effects of cancer and its treatment and is the thrust of the Multinational Association of Supportive Care in Cancer, financing of and value in personalized medicine is an important area of research and engagement for the association. Discussing patients’ concerns with and identifying those at most risk for the financial hazard of cancer treatment and offering financial counseling and assistance and/or referral to support networks are potential key areas for (exploring and providing) better supportive care. The time is now to turn the concern of patients and their carers, providers, and other advocates regarding the affordability of cancer treatment into a collective cause towards coordinated action.

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Already stretched health care systems face a panoply of challenges, including a high and increasing number of patients with chronic conditions, expensive and rising costs of treatment, and stagnant economies. Not surprisingly, even as specialty pharmaceuticals—or, more broadly, personalized medicine—are touted as the future of medical care because they make more treatments more effective, there are reservations about the value that they offer society as a whole, compared to certain population groups. The findings of Bradford Hirsch and coauthors (Oct 2014) certainly help justify the efficiency and equity concerns of payers and health authorities about the impact of specialty pharmaceuticals. To be sure, we know little about the intended and unintended health, economic, and social impacts of personalized medicine. Advocates claim that personalized medicine provides value for money, as illustrated by the French case of universal molecular testing and targeted treatment of lung cancer patients with epidermal growth factor receptor mutation.1 The sustainability of these benefits, unfortunately, is far from established. Personalized medicine is still both hype and hope. Arguably, the challenge is to produce a preponderance of clear and convincing evidence that specialty pharmaceuticals and personalized medicine are worth their cost.2