Measuring what matters

"Not everything that can be counted counts. Not everything that counts can be counted"

Not everything that can be counted counts. Not everything that counts can be counted” 

We are in a golden age of haemophilia research and development. Advances in treatment mean that, nowadays, many patients can successfully manage their disease while maintaining their independence. With bleed rates under better control, the community’s focus is instead shifting to what else matters most to people living with haemophilia – and how to incorporate those factors into research and day-to-day care. 

“Understandably, research in haemophilia has traditionally focused on bleed rates, but these alone just don’t tell the whole story,” says Mark Skinner, a leading advocate for people living with bleeding disorders, who also himself lives with haemophilia A. “We need to start seeing how living with haemophilia affects the whole person, beyond bleeds. We need to tell the bigger story around the personal impact, the societal consequences. And to do this, we need other ways to collect data that measure the emotional effects and quality of life impact of haemophilia and of new treatment approaches, to complement the clinical picture.”

This is where Patient-Reported Outcomes (PROs) come in. Find out how PROs measure the patient perspective, and can help redefine haemophilia care, with the following seven things you should know about PROs:

1. PROs reflect the patient’s own perspective of their disease. 

Information is gathered using standardised questionnaires filled out by the patient or caregiver, as opposed to their doctor or nurse.


PROs give opportunity for people living with haemophilia to provide their input directly, without interpretation, about their condition and its impact on their daily lives.” - Mark Skinner, President, Institute for Policy Advancement

2. PROs are a major focus for haemophilia research.

Pioneering studies, like the ongoing Patient-Reported Outcomes, Burdens and Experiences (PROBE) study, led by Mark Skinner, are using the measures that patients say are most relevant for them. These studies evaluate the emotional and social impact of haemophilia and its treatments. Along with traditional clinical measures, these help paint a more complete picture of the true impact that haemophilia has on individuals and their lifestyles.


3. Research methods incorporating PROs turn patient experiences into measurable scientific data.

Setting up the PROBE study resulted in a validated, detailed PRO survey specifically designed for haemophilia, tested and honed through a global patient-led research network. Uniquely, parents or siblings not affected by haemophilia also completed the PROBE PRO survey. This means that researchers can look at the effect that haemophilia has on, say, education or employment, in people with a similar background. Results are grouped and assessed in a consistent way, to tease out useful patterns or trends.


During the first six years of his life, we had to go to the hospital daily, which was 30 kilometers away from our home. It took us almost four hours to inject him. It was like a job for me. I could not work at the time because it took too much time to have treatment for Michael.” - Mariska, mother of child with haemophilia A

4. PRO data can help deliver better care.

Comparing data in people with or without haemophilia, from before and after a policy change, or between two different countries, can help make a case for new specific care services or management approaches to governments and health authorities. For example, showing that patients’ mobility issues affect getting to work can provide evidence of the need for special transport services; showing how these services increase work attendance and productivity can provide evidence to support a broader policy change or inspire other countries to follow suit.


With the data we are collecting, we are looking to empower patients and patient organisations to move advocacy beyond anecdote and emotion to evidence-based arguments, to galvanise ongoing change.” - Mark Skinner

5. PROs can help drive medical innovation.

Scientists are using PRO surveys in clinical trials to test whether a potential new medicine can improve a person’s quality of life, or whether a patient prefers the new or existing treatment. This information supplements the clinical data on safety and effectiveness, to demonstrate more fully the potential benefits of a new haemophilia treatment, and ultimately help researchers to develop medicines that can make a real difference to patients.


6. PROs help doctors and patients make the right treatment decisions.

A more complete picture of the impact of different treatment or management approaches makes more informed patient-doctor conversations possible. These conversations can result in better shared decisions on care – grounded in deeper mutual understanding of individual patient preferences and goals, and backed up by evidence.

7. New technology will boost PROs and their impact.

Online and mobile methods to collect PROs, including activity trackers and health apps, will allow more information to be collected from more patients more quickly, and without the need for face-to-face appointments.


We all have a role to play

The landscape in haemophilia, including how we are able to gather data, is rapidly changing. The haemophilia community – patients, doctors, scientists, advocacy groups and regulatory authorities – will all need to work together to adapt to the rise of PROs and harness their potential.

That’s why Roche are providing grants to fund important PRO research – such as PROBE – to find out what matters most to people with haemophilia, and are launching awareness campaigns – such as  ReDefine Haemophilia – to spark conversations around what it really means to live life with haemophilia A.


  1. 1963, Informal Sociology, a casual introduction to sociological thinking by William Bruce Cameron, Page 13, Random House, New York.

Tags: People, Science, Culture, Innovation, Society, Patients