How are you doing? (2)- PRO reporting in practice

In the first part  of the  “How are you doing?” series about the important role that patients can play in the Alexion aHUS Patient Registry,  we explored the topic of long term observations of what it is like to live after a diagnosis of aHUS. Patient Reported Outcomes (PROs)  provide important data about what the patient is thinking and feeling over time.  But that is true only if PROs are reported and shared on a regular basis.

In Part 2   data, which the aHUS alliance Global Action team sought from the Registry about non compliance in providing PROs , is presented and considered.

Year of enrolment No. of new patients enroled  in year No. of new patients completing at least one Fatigue form Total No. Fatigue forms completed No. new patients completing at least  one  general questionnaire Total No. of general questionnaires completed
2012 21 20 23 19 22
2013 336 279 353 290 369
2014 310 269 384 271 387
2015 380 298 402 300 408
2016 385 292 394 303 407
2017 317 222 291 236 323
2018 40 25 36 25 37
2019 32 19 23 22 26
Total 1821 1426 1910 1473 1988

Table 1 : PROs completed by  newly enroled patients ( supplied by Alexion  aHUS Registry)

From  2012 ( the year the Registry began) until 2019, 1821 aHUS patients were enrolled in the Alexion aHUS Registry.

In 2018, Alexion decided to remove “uneconomic” sites, limit the number of aHUS patients to be followed and stop new patient recruitment in most countries and thereby downsize the Registry to about 900 patients (at Feb 2019) . Recruitment since  2018  has only been undertaken  in Belgium, France, Poland and South Korea. New patient enrollment numbers have therefore reduced dramatically.

There are two sources of data for PROs , a universally adopted Facit- Fatigue form and a General Questionnaire about patient health and activity. Together they provide the measures for  PROs.

In Table 1  information is given for the number of patients enrolled in  each calendar year, the number of those patients supplying one or more of each PRO and the number of each PROs received.

Following 2012,  the number of new patients enrolled grew until recruitment began to slow in 2017 and then drop sharply after  Alexion’s  recruitment policy change. Naturally similar trends can be seen in the numbers of PROs provided.

In no year do all newly enroled patients complete both PROs and in the eight years  only  1426 out of 1821 enrolled patients complete a fatigue form, and slightly more , 1473 out of 1821 complete the general questionnaire.

The  number of PROs  entered however is higher than the number of patients enrolled, so some patients had provide 4 PROs in the calender year. Overall there were 1910 and 1988 of each PRO  entered from 1821 patients. Slightly more than the average to be expected of one of each PRO per patient in the year of enrollment.

But that is the overall perfomance for the eight years., In Table 2 the percentage of patients complying in each year and the average PROs they complete are shown.

Year of Enrolment % of new patients  completing one Fatigue Form Average Fatigue forms completed  per patient % of new patients completing at least one questionnaire Average general questionnaires completed per patient
2012 95.2 1.10 90.5 1.05
2013 83.0 1.05 86.3 1.10
2014 86.8 1.24 87.4 1.25
2015 78.4 1.06 78.9 1.07
2016 75.8 1.02 78.7 1.06
2017 70.0 0.92 74.4 1.02
2018 62.5 0.90 62.5 0.93
2019 59.4 0.72 68.8 0.81
Total 78.3 1.05 80.9 1.09

Table 2:  Comparison of relative  PRO compliance per calendar year.

Table 2  reveals 2012 was an exemplorary year for PRO compliance. Although not 100%  performance was above 90% for both PRO versions. However as  the number of patients enrolled increased in subsequent years,  the percentage of new patients completing PROs , apart from in 2014, drops significantly, even more so in 2018 and 2019.

By 2017 only 70% of new patients were completing the Facit Fatigue form on enrollment and 74.4% were completing the general questionnaire. In 2018 and 2019 after the change in enrollment policy, non compliance  increased and compliance was in the 59 to 69% range.

£xcluding 2014 , a  similar fall in the average number of PROs completed per patient can be found and by 2017 the number  dropped below an expectation one of each PRO version. Once again there were even bigger reductions  in 2018 and 2019 , when it perhaps could have been expected that there would be an improvement in PRO compliance because  patient numbers enrolled were nearer to the 2012 levels when compliance was at its highest.

The data in Tables 1 and 2  is  restricted to what happens to PROs at the beginning of a patient’s participation in the Registry. It does not reveal what happens as the patient’s participation continues into subsequent years. It cannot be concluded therefore whether there is any improvement with more experience or a greater fall off as the novelty and enthusiam of joining the  Registry wears off, or a habit of non compliance is embeded.

What is revealed however is  that, as the years have gone  by, newer patients are less likley to provide PROs. This deterioration in compliance could impact on the quality of the Registry data . Their PROs at enrollment are doubly important as they  set a benchmark at a time when they may be at their illest.

395 in total did not meet their obligations in their enrollment year, and although they may provide PROs  later, they cannot go back to that important moment. They may even be more likely to not provide PROs in the future.

In Part 3 of “How are you doing?” the implications  of an increase in non compliance with  PRO obligations will be discussed and how the current trend might be reversed.

Click HERE to go to  Part 1 or HERE  to move on to Part 3.