Defining patient value in haemophilia care

Once upon a time, before the age of modern medicine, death ór miraculous survival was the most common outcome for many diseases, including haemophilia. Nowadays, haemophilia outcomes also include bleeding episodes, arthropathy and inhibitors, and more patientcentred outcomes such as quality of life (QoL) and participation in society. There is no doubt that these outcomes have improved dramatically in most countries in the past decades, due to increased availability of safe clotting factor concentrates and prophylactic treatment. But has “value” for individuals with haemophilia increased? If so, can the haemophilia community worldwide improve “value” further? And will “value” increase with novel and promising, but costly treatment options? That depends on the definition of value. Value comes down to: is it worth it? Value is about achieving patientrelevant outcomes relative to costs. What value is depends on the role played within the health care field: providers traditionally focus on clinical outcomes (eg clotting factor levels, annual bleed rates), while costs are usually the domain of policymakers and insurers. For people with haemophilia, outcomes are also about QoL (eg pain relief, functional ability) and costs can be both monetary and nonmonetary (eg travel time to the treatment centre, loss in productivity).1,2 For someone with mild haemophilia, the most relevant question may be: Is it worth taking a morning off from work for a routine visit to the treating physician? Is administering prophylaxis to prevent spontaneous bleeding always worth the time investment for an individual with severe haemophilia? Contrastingly, for someone in a developing country with limited access to treatment, the value may be in surviving severe bleeds. Delivering value to patients should be the overarching goal of healthcare provision, argues Michael Porter, professor at Harvard Business School. He is the founding father of valuebased health care, a concept introduced in 2006. This strategy consists of six essential elements that should be implemented simultaneously: (i) organize care into integrated practice units (around the consumer or need), (ii) measure outcomes and costs for every patient (so progress over time can be tracked), (iii) move to bundled payments for care cycles (paying for outcomes rather than services), (iv) integrate care delivery across separate facilities (eliminating duplication of care and optimizing care in each location), (v) expand excellent services across geography (increase catchment area for an excellent hospital) and (vi) build an enabling information technology platform (that helps the parts of an integrated practice unit work together). Together, these elements can improve value of care in many settings. The need is urgent: many hospitals and even health ministries have started to work towards improving value rather than profit.3 How about haemophilia? The first two elements, organizing care into integrated practice units and measuring outcomes and costs for every patient, are the starting points.3 Integrated practice units provide services to people with the same medical condition and needs in terms of outcomes. They do not only treat the medical condition but also related conditions and complications (eg arthropathy, hepatitis C, HIV infections, inhibitors),1,3 all highly relevant for haemophilia. Can and should haemophilia be defined as a single medical condition? Medically, it is clearly defined as factor VIII or factor IX levels below 40 IU/dL, but outcomes and subsequent clinical management are much more heterogeneous:4 functional outcomes and QoL are perhaps similar for individuals with severe arthropathy and people with other orthopaedic conditions, but different for mild haemophilia. Many haemophilia treatment centres worldwide provide multidisciplinary care for haemophilia,5 but true valuebased health care goes further: all team members, regardless of specialty, share the responsibility to improve outcomes, and are accountable for the results.3 The second step is to establish socalled minimum outcomes sets or core sets of outcomes (both clinical and patientreported). These combined sets have already been developed for several conditions, including lower back pain,6 advanced prostate cancer7 and hip and knee osteoarthritis.8 With the help of Delphilike processes and involvement of both patients and different clinical specialists, organizations such as the International Consortium for Health Outcomes Measurement (ICHOM) and the Core Outcome Measures in Effectiveness Trials (COMET) focus on defining outcomes that matter most to patients and that are to be used as effectiveness endpoints in clinical trials,9 with the patient’s voice becoming increasingly important.10 As with value, outcomes are not all similar and equal, but they form a hierarchy.1 Porter divides patientrelevant outcomes into three tiers: (i) health status achieved or retained, for example mortality rates or functional status; (ii) outcomes related to the nature of the care cycle and recovery, for example preventing hospital readmissions, because they are a burden on patients and clinicians as well as on the system; and (iii) outcomes related to the sustainability of health, for example recurrence of health problems.3 A core set of combined clinical and patientreported outcomes does not yet exist for haemophilia. Brian O’Mahony, Gerard Dolan and colleagues11 set off to map value in haemophilia onto the threetiered framework of outcomes. They defined haemophilia outcomes in each tier and