Patient group input requested for a new Joint Assessment on a medicinal product for cerebral adrenoleukodystrophy (CALD).
EUnetHTA deems patient involvement very important in the production of Joint Assessment reports. We recognise that patients and those who support them have unique knowledge about what it is like to live with a specific disease or medical condition. We believe patient groups can help us understand patients’ unique perspectives by collecting and presenting patients’ and carers/care-givers’ views and experiences by engaging with a wide range of patients. They can describe advantages and disadvantages of medicinal treatments based on patients’ experiences and what patients value from a new treatment.
EUnetHTA recently started a new Joint Assessment on a medicinal product for the treatment of patients less than 18 years of age with an ABCD1 genetic mutation and early cerebral adrenoleukodystrophy for whom a human leukocyte antigen (HLA)-matched sibling haematopoietic stem cell (HSC) donor is not available.
For this specific assessment on a medicinal product for cerebral adrenoleukodystrophy (CALD), an ‘open call for patient group submission‘ is used.(1) Since this is a rare disease among patients less than 18 years of age we are also open to receive input from parents/caregivers. If you are an individual patient representative and would like to complete our open call, please contact us on WP4_pharmaceuticals@zinl.nl by the 15th of December.
We are seeking European and national patient organisations to provide an organisational perspective on the questions in English. The questionnaire has been translated to national languages, so that it is easier for patient organisations to gather input from their members. You can find the EUnetHTA Patient Input Template in the EU official languages here.
In all parts of this form the term ‘patient’ refers to anyone living with, or who has lived with, the condition for which the new medicine is indicated. The information provided will inform the scope of the assessment, e.g. on patient relevant outcomes or possible neglected outcomes, as well as on the burden of disease.
EUnetHTA strives to be transparent with any information collected, while at the same time we also recognise that completing this form requires substantial resources. Therefore, we are committed to making the key messages of the patient submission publically available via our webpage by including them, verbatim, in the appendices of the assessment report. In due course, the report can be found here.
It should be noted that the remainder of questions can be included in the report by the authors as background material. Since any such questions are not the key messages, these should be included at summary level, rather than quoting patient organisations by name.
As a matter of policy, individual patient or patient organisation data will be anonymised prior to publication. Nevertheless, it should be noted that participating organisations will be mentioned in the project plan, and possibly within the report. Thus, while such data is not directly linked to any particular organisation, in the instance of low numbers of responding organisations (or certainly if there is only a single respondent), the inferred link between any given respondent and such data could therefore increase.
Finally, our Joint Assessment report will document how the information from patients has been considered in developing the scope of our EUnetHTA Joint Assessment.
If you are interested in submitting information, please go to the questionnaire below and submit your input before 15th of December, 16:00PM.
Patient input requested for a new Joint Assessment on a medicinal product for cerebral adrenoleukodystrophy (CALD)
Please see below for further guidance on how to complete this questionnaire. If you have any questions when completing this form, please contact EUnetHTA pharmaceutical Joint Assessment Co-Lead Partner, Zorginstituut Nederland at WP4_Pharmaceuticals@zinl.nl
Many thanks for your valuable input!
(1) The questions in this call are taken from the HTAi questionnaire template. For more information on the development of the HTAi questionnaire template please see their website.
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