Electronic patient records (EPRs) and databases are not new concepts for people with hemophilia. Generally, electronic patient records are developed as soon as patients are diagnosed by hemophilia clinicians or hematologists with a special interest in hemophilia, or at the establishment of a new hemophilia society. Initially these records are populated with only the most essential details (e.g., name, date of birth, and location), and may include a patient identification number if data were collected in a hospital.
Early data tends to capture the most basic of facts and do not necessarily include the type or subtype of bleeding disorder due to the lack of ability for diagnosis. The information captured expands as hemophilia care, laboratory testing, and treatment occur; extending to bleeding disorder types and subtypes, comorbidities, family history/connections, records of bleeds, number of hospital visits, inhibitor history, product use, and treatment type.
We so often hear that it takes significant funding and resources to establish EPRs. In fact, at the very beginning of some registries the start-up costs are practically zero since EPRs are established by an individual in the hospital or the hemophilia organisation using the most basic and easily accessible computer programs (e.g., Microsoft Excel). The downsides are that these files often reside in one location, are updated on an ad hoc basis, and are not accessible by others in different locations. This leads to duplication of patient records and laboratory tests, and inaccurate treatment information. Enabling multiple users to access EPRs requires more investment, though this may be less of a problem with the use of file-sharing programs across the internet. However, we now enter the area of data protection and privacy. Ensuring that a person’s personal health information is kept in accordance with accepted privacy laws makes the process more costly, but should be considered an investment for the future development of hemophilia care and monitoring of treatment and its outcomes.
Within a patient organization (NMO), a registry is essential for survival and development. It has numerous uses, from the basics of finding a phone number to strategically locating patient meetings or targeting treatment and services. It is a patient organization’s best resource. In some countries, this is the primary registry, and it is updated by dedicated volunteers and/or staff members. The advantage is timely access to specific information on the people for whom NMOs are advocating. Although in some countries the NMO registries hold more details than some hospital registries, the significant disadvantage is that these are not connected to hospital data. Therefore, NMO registries will never accurately reflect the patient’s hospital visits, treatment logs, physiotherapy records, surgical procedures, and other data that are at the core of lifelong treatment of hemophilia. However, even if a national registry collects every possible piece of information, the NMO should ideally maintain its own registry. Without data, patient organizations are limited to fighting with just emotional arguments, which for most governments is not sufficient.
Most localized registries come in the form of hospital and insurance company registries, which share similar advantages and disadvantages. Local registries generally collect patient information with reasonable accuracy. They are connected to a patient’s medical history and contain reports from multiple disciplines. They can be used to determine the level of prescription of treatment, effect of treatment and potential adverse events such as inhibitors. Local registries can also be used to identify annual trends—number of new patients, approximate bleed rates, and volume of product purchased. There are several disadvantages to this approach. People change hospitals and/or insurance companies, and often this leads to duplication issues within the system. There is also generally no uniform method for data collection, which gives rise to inaccurate information and less accurate prediction in planning services and treatment volumes for hemophilia. Other issues are an inability for these registries to be connected to each other in the future and that there is limited transparency in what is collected.
National patient registries clearly provide the greatest benefit for patients and clinicians and the state. We are still waiting for the utopian version—where hemophilia and other medical information are connected—but a few countries are close to achieving this model. In the United Kingdom, the National Health Service allocates a unique patient identifier to all residents and all medical treatment is linked to this identifier. In parallel, the United Kingdom Haemophilia Centre Doctor’s Organisation registry allows clinicians to view a patient’s history and compare to other medical co-morbidities. This functionality will become increasingly useful as we face an ageing hemophilia community.
In the situation where unique patient identifiers are not currently available, some countries have developed web-based registries that clinicians can access from any location, regardless of the diverse I.T. systems used across hospital services. Web-based systems can be accessed within hospitals and also by local hematologists in a distant centre or even by nurses during home or district visits. The only real disadvantage is that other medical details are difficult to access outside of a hospital setting.
A rare disease registry, which mirrors the web-based model, is an interesting model currently used in France. A major difference compared with a hemophilia registry is that it captures a larger patient population receiving treatment for a wide range of conditions. Rare disease registries often require an extensive cross-collaborative approach with access to specific government or regional funding. It is a highly effective model, but it must be noted that with a wide range of conditions being captured, hemophilia data may not reach the necessary level of detail.
Who should pay for these registries? Governments, hospitals, insurance funds, or pharmaceutical companies? The answer is obvious: governments. Even in most developed countries, it is impossible for individuals to pay for hemophilia care, and governments ultimately pay for treatment and services. Unlike many conditions where the cost of the treatment is 50-60% of the total costs (treatment + services), the cost of treatment in hemophilia is almost 90% of the total, so countries without registries are effectively paying for treatment without gaining feedback on real effectiveness.
Hospital or insurance funds could provide financial backing, but these institutions may not have the same priorities when it comes to collecting data in a uniform way. Pharmaceutical company funding is also possible but it is important that is the registry is not based on individual products. In the Netherlands, a national patient registry is being developed which is equally funded by the government and all pharmaceutical companies that provide the country with products for bleeding disorders.
What does the future hold for EPRs? This is strongly dependant on the overall organization of hemophilia care, from the decision to purchase treatment to its administration in individuals. Payers and assessors have difficulty with data from EPR’s as they can have very static information. The ideal future for EPRs for hemophilia needs to be more dynamic. We are seeing the start of active EPRs in the United Kingdom, Australia, Ireland, USA, Germany, and Canada. Gone are the days of seeing patients filling out treatment sheets from memory. Instead, patients are using apps to enter data on bleeds, product use, timing, and pain experienced during or close to treatment administration. Not only is this more accurate, but it prevents vague discussions at clinical appointments.
One individual described his most recent appointment to me: “I was on demand and thought I was fine, but I was surprised to be told I had seven bleeds in the last six months, all on my right side. I switched to prophylaxis and was sent for physiotherapy to balance the strength on my right side. I feel a lot better. It was the best appointment I’ve had in years.” In this case, the clinician was able to use patient feedback to prescribe the correct treatment, not just the amount of factor or the regimen but also the focus for the next physiotherapy appointment.
With the advances in mobile technology we have an opportunity to enhance the capabilities of registries significantly. To avoid the challenge that has already occurred with the existence of multiple registry systems that are not connected, coordination should start as soon as possible. This will ensure that data will be collected in an uniform manner and there will be increased value derived from the data that is collected.
We can also achieve even more with EPRs going forward. We can use EPRs to track and warn patients approaching their personalized trough levels (using pharmacokinetic data and knowledge of a patient’s factor levels when they experience bleeds) and alert healthcare professionals in hemophilia centres when persistent or specific bleeds (e.g. iliopsoas or intracranial) occur. Alerts can prompt a healthcare professional to contact the patient to discuss further treatment or inform the patient to contact their hemophilia treatment centre immediately.
In the future, EPRs may even assist with identifying a bleed. Fewer children and young adults are experiencing bleeds when on prophylaxis, and sometimes have difficulty identifying when a bleed starts. One study is examining the use of accelerometers in smartphones or activity trackers to detect changes in gait, which may be indicative of the start of a bleed. Patients and their families will know relatively quickly whether the individual requires factor or needs to investigate the cause of the bleed.
A great use of EPRs is when treatment use (recorded by the patient) is linked to treatment purchase on a national scale. In Ireland, the system scans product at purchase and again at distribution to three comprehensive care centres, four HTCs, 30 hospitals (adult dose in case of emergency), and the patient’s home. This allows precise stock control and allows emergency hospital product approaching expiration to be rotated into the main comprehensive care centres to prevent wastage and co-ordinate the service. Since the system also tracks products sent to a patient’s home, they can be asked to use older stock first. This may be just one vial a year, buteven one vial per patient per year on a national level could pay for a significant portion — if not all — of the costs of putting a system like this in place in the first year! EPRs that continually generate data, although in their infancy, will flourish as technology advances and costs are reduced.
Data from active EPRs help predict product growth patterns for payers and improve treatment regimens for clinicians and patients. There are many clinicians and patients willing to develop such registries but we need a collaborative effort by all involved in collecting data in a cohesive approach and sharing the data in EPR’s.
For more information on the WFH epidemiological research program and global data collection, click here.