Standardizing patient outcomes measurement to improve haemophilia care

To improve our understanding of the treatment of persons with haemophilia (PWH), better individual patient data collection and comparison of outcomes are required 1. The annual global survey of the World Federation of Hemophilia (WFH) provides an overview of the number of patients and their access to treatment 2. While this has proven to be a valuable tool to assess progress of care and services in different countries and for advocacy, it does not provide data on how individual patients are treated and what their long-term outcomes are. Such data on individual PWH have been collected in very few centres around the world 3. However, it is becoming increasingly clear that to achieve a high quality of care, comprehensive annual evaluation of PWH and its systematic documentation is very important 4. In fact, the importance and need for standardizing outcomes assessment in all chronic diseases is being increasingly recognized 5. There is a need therefore to identify a core set of data that should be evaluated and documented in a standardized format for this purpose universally. The WFH has created such a data collection format as a universal case report form (U-CRF) and posted it on its website, (http://www.wfh.org/en/u-crf). This commentary describes the process followed to create such a data collection format and an overview of its content. The WFH is committed to not only increasing access to therapy that PWH have around the world but also to improving the quality of care that they receive 6. Towards these goals, the WFH makes multipronged efforts including training for healthcare personnel and lobbying with governments to create appropriate infrastructure at haemophilia treatment centers (HTCs) and to procure safe and effective therapeutic products. Over the last 5 years, there has been special emphasis on increasing awareness of outcomes assessment particularly from the musculoskeletal perspective and their documentation. To bring together the progress in this field that has been made through the efforts of different groups around the world, a special section was created on the WFH website (http://www.wfh.org/assessment_tools) where the commonly used instruments have been listed with explanations on their use. For creating the data collection platform, a U-CRF, an ad hoc group of individuals with experience in such data collection from different perspectives was appointed. This group was charged with the task of creating a set of data forms that could be used for documentation of the relevant diagnostic, therapeutic and outcome data in PWH being managed in different healthcare environments. The group identified the domains described in the European principles of care as a comprehensive list of care and outcome parameters for which data should be collected on individual PWH 7. They also reviewed the existing CRFs being used in some of the major registries and cooperative group studies and extracted the most promising features from all of these to create a unified modular U-CRF which could be used anywhere in the world. Currently, it is estimated that less than 10% of the HTCs around the world collect the minimal required data (as in the MDS form) and less than 1% would be able to collect what is required in the C-CRF. With time and a system in place, more HTCs should start collecting these data for the care of PWH at their own centres, including the more comprehensive data set. This will lead to better care of PWH and will also provide prospectively collected data enabling the comparison of outcomes among different patient cohorts. While the primary goal of this exercise is to provide a uniform template for data collection at each HTC to help the management of individual PWH, the WFH hopes that this will ultimately lead to the development of meaningful national and international registries. To encourage this, the WFH has established a web-based option for hosting data from HTCs that may not have the capacity to host their own data. It must be emphasized that even if the data is hosted and held in a WFH-supported database, the individual HTC would continue to ‘own’ their data and its use for any purpose, research or reporting by others, would require permission from the HTC. Data from groups of HTCs can be pooled into national or regional registries. Through these efforts, the WFH would provide the platform for what could become a World Bleeding Disorder Registry. It will be very important to take into account the existing major registries in the world in the field and create a harmonious system for data sharing. Such a platform could then also be tapped to carry out prospective and retrospective studies to answer specific questions in the management of PWH, for which formal studies may be extremely difficult. Models showing how to effectively maintain and use such a registry exist for other diseases, such as the blood and marrow transplant field 8. The WFH will need to establish an appropriate independent institutional data management group to host and manage such data. While the WFH would generate a certain set of aggregate annual statistics from the data submitted and make these data available to all stake holders, requests for specific projects from researchers will need to be handled through a defined process, consent for use of data being always with the HTCs providing the data. As mentioned above, there are several successful examples of such an approach working very well in data management systems for other diseases. Overall, these are the first steps by the WFH towards building a culture of outcome assessment in PWH at every HTC in a systematic and sustained manner and providing a framework within which this is possible. Ultimately, this should contribute to considerably improved care for PWH around the world. Its success will depend on the acceptability of this concept and on the active participation and cooperation by all HTCs and other stakeholders who would like to see better haemophilia care being provided around the world. The WFH would like to acknowledge the contribution of the ad hoc working group for creating the U-CRF consisting of Marijke van den Berg, Alfonso Iorio, Michael Makris, Margareth Ozelo, Flora Peyvandi, Michael J Soucie, Alok Srivastava as well as the support from WFH staff involved with this project. The authors stated that they had no interests which might be perceived as posing a conflict or bias..