Building a better database can improve advocacy, research efforts

There is no one-size-fits all approach in data collection, but there is a credo: quality data needs to be trustworthy, appropriate, understandable and powerful, said Alfonso Iorio, McMaster University, Canada, as he kicked off the Sunday morning session “Show Me the Data! Challenges and Opportunities in Data Collection.”

“The more we move toward better treatment, the more data we will need,” Iorio said. “What was enough data a few years back will not be enough a few years down the road. It’s our responsibility to set up data collection we can follow for generations.”

The session featured a series of panelists addressing topics ranging from the keys of data collection to how to build a national patient registry.

Randall Curtis, Hemophilia Council of California, United States, said when building a database for advocacy purposes, it’s important to know your target audience. Decision makers often have scarce resources, and hemophilia advocates usually ask for many resources for a small population. “So your data has to be very compelling,” he said.

Legislators have narrow attention spans, so Curtis said it’s important to show data that’s of value to them, and make it short, easy to read and understand. In some countries, reduction of pain and suffering is very compelling. In other countries, it’s compelling that a young man with hemophilia can’t start a family.

“I always try to compare my data to the normal population,” Curtis said. For instance, the benefits of better care for people with hemophilia include reduced emergency room visits and shorter hospital stays, and improving underemployment rates and costs to society through things like lower graduation rates. And remember, “Cost avoidance is almost as good as cost-effectiveness,” he said.

In terms of the ethics of data collection, Margareth Ozelo, University of Campinas, Brazil, said participants should be fully informed of the way their data is going to be used and managed. She also advised that when forming a patient registry for research usage, you can reduce risk by getting patient consent and emphasizing the patient’s confidentiality, privacy and security.

David Page, Canadian Hemophilia Society, focused on the role of patient organizations in data collection. The WFH Global Survey and the European Haemophilia Consortium Annual Survey both include patient data, and Page said many countries’ health technology assessment bodies also require data from patient organizations.

Page said patient organizations have an advantage in collecting data because they provide easy access to patients and caregivers. They also have a lot of credibility. “Unlike market research firms, a patient organization is seen to have an unequivocal interest in patient welfare,” he said. Patient organizations can also efficiently collect data because they can bypass onerous research ethics board approvals.

Page said the disadvantage is that patient organizations can lack the scientific expertise to collect good-quality data. Data collection is also not seen as a role for patient organizations.

Kathelijn Fisher, University Medical Center Utrecht, the Netherlands, listed a hierarchy of patient data collection in order of need: number of patients according to diagnosis; treatment, including prophylaxis and clotting factor consumption, surgeries and number of annual joint bleeds and location; joint status, including a physical exam and imaging; and activities, participation and quality of life.

Jeff Stonebraker, North Carolina State University, United States, said there’s no perfect data, but there are sources for collecting the best data possible. These include the WFH Annual Global Survey, scientific literature, national registries, The Marketing Research Bureau, the World Bank, the United Nations, the World Health Organization, the U.S. Centers for Disease Control and Prevention, survey questionnaires, subject-matter-expert interviews, and the Immune Deficiency Foundation.

Christine Herr, WFH’s data research coordinator, said data is being collected for the 2015 Annual Global Survey, which will be released in October and can be viewed at This comprehensive survey can be downloaded and used in advocacy efforts, Herr said.

The 2014 AGS includes data from 106 countries, validation against humanitarian data, and new sections on hemophilia severity and data sources.

New topics on the 2015 questionnaire include: percentage of the patient population being reported on per country; more information about HIV and hepatitis C infection, common doses of factor administered and frequency, immune tolerance induction, and amount of donated factor concentrates.

The session closed with a look at the successes and challenges of building nationalized, web-based patient registries.

Anne-Louise Cruikshank, South African Haemophilia Foundation, said challenges in building her country’s web-based patient registry included patient underdiagnosing and under-reporting, accuracy problems with data collection, and synchronizing data sent in Excel spreadsheets to a web-based system. Security was also an issue— “Data was only as secure as the office it was locked in,” she said. And healthcare personnel charged with collecting data often became disheartened.

As a result, the foundation came up with a new approach to obtain better data. The University of Cape Town Research Center, in conjunction with a local hematology center, will develop a web-based software that will be hosted on the university servers.

Saeed Ul Hassan, Pakistan Hemophilia Patients Welfare Society, said In 2013, the society submitted an initiative to the WFH to build a national patients registry. The initiative was approved and funded by Novo Nordisk.

The registry, which was launched in June 2015, groups data by diagnosis, age group, chapter/area of country, incidence of hepatitis C and B, joint deformity and more. Hassan said the data is used to monitor trends in health, allocate resources, distribute mechanisms for humanitarian aid and ensure better global data.

In Canada, Page said a lack of financial resources and clinician time to gather data gave the Hemophilia Society the idea of using an already-existing registry. As a result, Canada is using Australia’s registry and adapting it to fit Canada’s needs.