Medical research depends a lot on the collection of clinical data about patients from blood and other physical tests and measures to determine the health state of those being studied. There may be other tests carried out too about their mental health state to arrive at an objective view of their overall health.
Such measures taken over time can assess the progress made, or otherwise, for an individual or collection of individuals with the same disease.
This is the basic purpose of a patient registry for any illness.
But increasingly in medical research, patients are being asked to say how they feel and how they are coping with aspects of their day to day lives while living with their disease. Whilst a treatment might keep someone alive , if they feel little better or their quality of life remains diminished then the benefit of treatment becomes questionable.
This is part of a patient centric approach to find out what matters to them. There is sometimes a difference between how well a clinician thinks a patients are doing and how patients feel themselves.
aHUS patients’ interest in the clinical, psychological and social-economic impact of having aHUS is reflected in the questions that are asked in topics 4 and 5 of their Global Research Agenda ( see below or click HERE to read more) .
4.2 Can the side effects of treatment using a complement inhibitor be distinguished from those temporary and permanent ongoing ailments which follow initial onset?
4.3 Does the anxiety and self-esteem of aHUS patients vary significantly between treatment types and what can be done to reduce and boost them respectively?
5.1 How does living with aHUS impact on education and work?
Articles about topics like “brain fog” are popular with viewers of aHUS Global Action’s website.
Eculizumab has been transformational for aHUS patients, where it is accessible. It is reasonable for health policy makers, such as the Food and Drugs Administration ( FDA), to want to know how transformative it is from a patient’s standpoint and perception. That is why registries are sought and demanded by health policy makers to provide those answers . Alexion’s aHUS Registry is one of several registries created for the long term observation of aHUS patients, whether treated by eculizumab or not.
Having given informed consent to participate, on their enrolment into the Registry patients base clinical data is supplied, including the patients’ genetic susceptibility variant if known. At that time too each patient, or carer , is also asked “how are you doing?” in a list of questions about tiredness, and how they are coping generally with their illness and treatment, including adverse health events and other issues like employment/schooling.
These are known as “patient reported outcomes” in the registry world, or PROs. ( For more information about PROs in patient registries click HERE).
They give a snapshot of how patients are doing at a moment in time.
After the first two questionnaires are answered , one specifically about fatigue and another about general health related issues, the same questionnaires are given to patients at six monthly intervals thereafter, to update the response to “how are you doing? “ and their completed forms are entered to the Registry database by their hospital . In a twelve month period, patients would be expected to complete four PRO reports , The total number of completed forms entered to the data base in a twelve month period would be 4 X number of patients enrolled.
What patients say in these reports is important. As far as PROs are concerned patients themselves are their own control group. It is important over time that a picture of patient progress , or not, can emerge for an individual, as well as generally for the whole Registry cohort. That is the value of collecting such data. It is an observation of the natural history of a disease over a long period of time
All registries can face the problem of incomplete data.
Incomplete data hampers the quality of registry reports and the quality of research based on them. A registry like the Alexion aHUS Registry ,which has been set up to observe aHUS patients for thirteen years , is going to experience incomplete data , for a variety of reasons, over that time. Not just uncollected medical data but also PROs.
aHUS Global Action was therefore interested in finding out what the extent of unreported PROs is experienced by the Alexion aHUS Registry , and whether any action was needed to improve it.
As an independent investigator Global Action submitted a research ” concept sheet” asking for Registry data about PROs response compliance by patients. The data request passed through the protocols set up to consider any request for data from the Registry and eventually Global Action’s request was approved by ther Registry’s scientific advisory board.
The scope of Global Action’s research was the level of compliance with patient PRO reporting obligations since the Registry was established in 2012 and whether the 2018 change in Registry recruitment policy and “downsizing” of patients that followed had any impact on PRO activity.
Alexion supplied data for only the newly enrolled patients in each year since 2012, the year of the Registry began through to the end of 2019.
In the first year of enrollment in any calender year , patient enrollment is likely to be spread over the year. Therefore in that first year, newly enrolled patients would be effectively “in” the Registry for an average of six months.
In the first year “on average” therefore only two of the four PRO reports would be expected from those newly enrolled patients.
But what did the data reveal about the level of compliance during those years by the newly enrolled patients.
In the second part of “How are you doing?” the Registry data about PRO compliance is presented and examined
Click HERE to go to Part 2.