aHUS Expert Centre- what makes it so

 

Within the six pillars of support for those with rare diseases is a  need for disease expert centres  (aka- centres of excellence/expertise). The alliance has featured several articles about such centres for aHUS. It even visited four European centres in 2018 (reports of those visits can be seen starting with the visit to Paris

During the visits the alliance made a record of examples of how each Centre addressed  EURORDIS’ list of attributes for determining  expert centres (plus other attributes offered from research by Rare Disease UK and the aHUS alliance). Using those attributes, the alliance was able to compare and contrast those centres to understand better the roles of aHUS Expert Centres.

The result of that assessment is not an evaluation of the competence of the Centres, but an observation of the strength of focus exhibited by them on each of the attributes in the mix.

The table below gives the alliance’s opinion of the strength of focus derived from those observations. There is no priority in the order in which attributes are listed.

Using a scale of SF ,F ,f ,where SF is a stronger focus, F is expected focus and where there is little or no focus evidence, the activities of each Centre are “scored” for comparison.

The overall results show  aHUS  expert centres vary in focus.  The scope of each centre in terms of its geographic remit ( local/regional, national, international) and its specific role in the healthcare for that location varies. Such scope inevitably  impacts on the number of direct patient beneficiaries.

Centres may have a hands on or shared/indirect responsiblity for patient treatment ; including different powers for prescription of drugs like eculizumab. They were found , however,  to be  influential in the drafting of national aHUS  diagnostic and treatment protocols

Even though the level of direct patient contact varies it does not diminish an  overall sense that their ultimate focus is  on patients ,however remote the contact. This is seen particularly  in their delivery of diagnostic services to speed up diagnosis and quicken patients’ advancement to treatment.

Centres also provide information to empower patients in their personal decision making about their treatment and to improve the quality of their lives.

However little evidence was found of expert centres  focussing on the transition of young patients to adult care. Care transition of children to adult services  is a universal problem in health care. Whilst the centres recognised it is an issue it was disappointing to find  no pro-active solutions designed specifically for aHUS patients.

Generally the centres displayed a strong  focus on genetic counselling to help clinicians and patients fully understand the implications of their genetic diagnosis, prognosis and susceptibilities. There was a reticence observed about  the important topic that matters to many aHUS patients i.e. that of counselling on aHUS family planning decisions.

The centres also have different relationships with patient organisations from being fully embedded in the operation of the centre through to an increasing arms length and more distant approach.

It is  the carrying  out of aHUS Research, sometimes for decades, to increase an understanding of aHUS ,and complement , that has made these centres expert and stand out. That expertise is then built on through a strong focus on further research. Frequently by working in collaboration with other expert centres ,and, also by running a global patient registry  or contributing to international patient registries run by others.

There is also an evident willingness to share this understanding among their peers and others in healthcare  through networking, e.g. within ERKNet or their work being published in professional journals as well as them being invited to give authoritative and insightful talks at professional conferences. Some centres explicitly display these achievements as a kind of “ references letter” to give evidence of their expertise , which adds to further acknowledgement of their expert status.

Some attention is given to economic issues not only for patients but in the value added by their centres. Each has to not only to find funds for their work , but also to control the centre’s finance and budgets to remain sustainable.

The leader of each of the centres visited is a well recognised and eminent individual in the field of aHUS , but it is also important to them ,again for sustainability , that successive generations of researchers  are developed within strong teams.

Better patient outcomes are achieved because of the work of these centres.

Attribute Centre A Centre B Centre C Centre D
Patient orientated approach SF F SF F
Help & encouragement of patient knowledge and empowerment in care SF  F SF  f
Information resources  provided to patients/families F SF SF f
Engages with patients and families SF F SF F
Cooperates with patient organisations SF F F f
Improves care atmosphere/collective morale of patient cohort SF F F f
Develops harmonisation of diagnosis and treatment protocols SF SF SF F
Better times to diagnosis SF SF SF F
Provides coordinated care through multi-disciplinary approach- areas integrated SF F F f
Ensures child to adult arrangements F f f f
Establishes care guidelines and recommendations F SF SF F
Undertakes Research and supports international aHUS Registries SF SF SF SF
International Collaborations F SF F SF
Undertakes international and national networking SF SF SF SF
Presents at conferences SF SF SF SF
Displays record of published work/talks  f   f SF SF
Shares knowledge with other aHUS Expert Centres F F SF F
Awareness and knowledge dissemination  to clinicians F F F F
Educates and trains health care professionals F F SF f
Interfaces with sufficient number of patients F SF SF SF
Provides Genetic Consultancy SF SF SF SF
Family planning /lifestyle with aHUS counselling F f f f
Improves quality of life SF F SF F
Patient /Family socio-economic  cost/benefits f f F f
Teamworking not dependent on sole individual SF SF SF SF
Does economic assessment of centre activities F F F F
Improves patient outcomes F F F F

 

The aHUS alliance appreciates the welcome and time given by the Centres and their willingness to share  information to the aHUS patient community.