Is it time to think beyond bleed rates?

It is reasonable to assume that all stakeholders in haemophilia care want the best health outcomes from every health care pound. Trouble is, each stakeholder has a unique perspective and different expectations.

Speaking at a recent Bayer Healthcare Hematology Conference, Mark Skinner, President and CEO of the Institute for Policy Advancement and former WFH chair, said that patients have a unique perspective and will consider issues “differently to regulators, drug manufacturers, scientists, clinicians and payers.”

While regulators think about risk and benefit, payers think about cost and benefit and clinicians want to know that the treatment they are prescribing is efficacious “but I as a patient think about how does this affect the life that I am living,” he said.

The challenge is in turning that patient perspective into a measurable value, so that value in healthcare really becomes real value created for patients; this requires breaking the traditional link, whereby value is generally considered as cost.

He said the HERO (Haemophilia Experiences, Results and Opportunities) study had given some insights into the psychosocial burdens of living with haemophilia. It showed that patients often reported that haemophilia had negative impacts on employment, on forming relationships and on their sex lives, and that pain interfered with their daily life. “These are the things that people with haemophilia talk about when they get together,” said Mark.

He also talked about several initiatives underway in which patients were working with companies to generate data that will demonstrate value.

In the Patient Reported Outcomes Burdens & Experiences (PROBE) study, data are being collected on outcomes patients deem relevant to their everyday lives. They include pain (chronic, acute, interference, when it occurs), independence (impact on daily living and activities of daily living), educational attainment and attendance, employment (duration, underemployment and attendance), family life, mobility (impairment, and need for assistance) and current health status.

Data are also being collected on possible explanatory variables including demographic and personal characteristics, as well as on haemophilia-related issues like disease severity, treatment and bleeding history, target joints and impairment.

Mark Skinner noted that when patients were asked about their comorbidities, the expected list of arthritis, hypertension and hepatitis C included a surprise in gingivitis. “Although the importance of oral health is widely accepted, higher gingivitis rates indicate the importance of allied health professionals supplementing the core care team within haemophilia treatment centres,” he said.

Another initiative, the CoreHEM project is aiming to establish a clearly defined minimum “core set” of outcomes to measure, demonstrate and differentiate the effectiveness and value of gene therapy in haemophilia relative to current standard of care. “The annualised bleed rate may not be an appropriate endpoint for an approach that abrogates peaks and troughs, frequent repeat infusions, adherence issues and permits assumption of a normal lifestyle,” he said.

These are interesting and exciting initiatives, with the potential to really put the patient at the centre of care, and making treatment outcomes far more “patient relevant”. Provided, of course, that commissioners of care have the flexibility to consider anything other than just cost.