Our Strategy (2020-2023)
Introduction
The diagnosis of a rare condition, which has a life-long negative impact on the quality of life of the patient, family and other people directly involved, drastically changes their lives. Dreams and expectations need to be adjusted. A lifelong search for the best possible upbringing, treatment and guidance must be initiated. Medical knowledge is still often inadequate or difficult to access.
Fortunately, there are fellow sufferers groups for these patients, families and other people directly involved (patient organizations, hereinafter referred to as PGOs). These PGOs all have people on the same search tour, but often in a different phase. They can support each other with information and a listening ear. Although information can be shared via social media, these media are unsuitable for reference and interaction with experts and do not have a good reputation with regard to privacy, information security and the reliability of the information provided.
Idea and history
WaihonaPedia Four years ago, eight PGOs - made possible by a grant from the Ministry of Health, Welfare and Sport - started developing an online platform where patients, family and other people directly involved, together with care providers and researchers, can share knowledge and experiences in a safe and loyal environment: WaihonaPedia (www.WaihonaPedia.org). This evolving platform is unique in offering two-way traffic: users can not only place or update information in it when it suits them, but also extract information from it. From the information obtained, one can get clues for optimal support and development of their child or loved one. Healthcare providers and researchers can find information to adapt their treatment and research to the needs of patients and parents, which in turn will lead to improved care and guidelines for treatment. It has already been successfully used by two WHOs, both nationally and internationally.
WaihonaPedia offers the opportunity to involve people from other countries. For example, the number of patients familiar with Marshall-Smith syndrome in the Netherlands is only three (around 80 worldwide) and the exchange of data with people from other countries is essential to gain insight into this condition.
In 2018, the questions and answers collected through Waihonapedia "Ask the Expert" contributed to a scientific publication in the authoritative Nature Review Genetics, a pri- meur for a rare disorder. Information in WaihonaPedia is currently available in eight languages and can be expanded as needed.
Objectives
For the period covered by the plan, the following objective shall be pursued: To develop the platform in such a way as to enable PGO members:
- share experiences with other members and experts, both nationally and internationally,
- gain insight into the course of a disorder in their child,
- increase their knowledge about a disorder,
- take control of their lives and those of their child as much as possible
The best care, the highest possible quality of life, empowerment and social inclusion are achieved, which suits an individual family and is based on all the experiences and knowledge available through WaihonaPedia.
Activities
Activities to achieve the goal set, made possible by joined contributions from participating PGOs and a subsidy from ZonMw (on behalf on the Dutch ministry of Health) of € 100,000.
During the planning period, the aim is to bind the participating PGOs more closely together and to allow them to participate in the platform by introducing them further in the four sub-areas below.
- Theme rooms First of all, 'theme rooms' will be set up in the platform for various aspects of rare diseases at the request of PGOs. In a theme room, parents, supervisors, care providers and specialists will share information and process it into a clear overview for one specific aspect of the disorder. This information is then made accessible to all members of the PGO and can also be modified by members. By adapting it over time to the latest developments and insights, the overview remains up to date and of great value to all those involved, and where possible also to several PGOs.
- Questionnaires In order to allow more people to gain insight into the course of a disorder, questionnaires have been developed and are adapted if necessary. If parents fill in these lists periodically, they get a good insight into the course of the disorder. "By filling in the questionnaires, I actually learned about my own son" is an impressive quote from one of the parents. These forms can also serve as a starting point for an interview with a doctor. In the AMC there is already some experience with this.
- Development of knowledge Due to the rarity of the disorders, the combined knowledge and experience of parents is of inestimable value to learn more about a disorder, both for themselves and for specialists. The knowledge in the theme rooms will be bundled into a valuable and reliable encyclopaedia about a disorder.
Direction and empowerment With the acquired knowledge and experiences of others, parents will be:
- be better able to take control of their own life and that of their child or loved one.
- and be a well-informed interlocutor for caregivers and - improve their child's development, thereby enhancing their social inclusion.
progressed.
Further development
The planning period is also intended to create financial continuity to be able to stand on its own two feet with the help of the participating PGOs. Financial resources are needed to maintain and expand an easily accessible, user-friendly and safe platform for all the diseases mentioned. Financial resources are needed to maintain and expand an easily accessible, user-friendly and safe platform for all the disorders mentioned. In doing so, strong consideration is given to people who are less digitally literate by improving user-friendliness. Participants of PGOs will be supported as much as possible in the use of the system with tools, scripts, courses and training sessions.
Cooperating PGOs - Cornelia de Lange Syndrome Association (NL) - Pitt-Hopkins Syndrome Foundation (NL) - MSS Research Foundation (Marshall-Smith) (International) - Williams-Beuren Syndrome Association (NL) - Angelman Syndrome Association Netherlands (NL) - Tuberous Sclerosis Foundation Netherlands - Rubinstein-Taybi Syndrome Foundation (NL)
The AECO Expertise Centre of the AMC for the Pitt-Hopkins, Marshall-Smith-, Rubinstein-Taybi- and Cornelia de Lange syndrome, among others, provides support for setting up the platform and drawing up various questionnaires.
In order to make the platform easily accessible in all aspects to the participating PGOs, each PGO has taken out a subscription of € 5000 per year. For the period September 2019 - March 2021 a subsidy of € 100,000 has been obtained from ZonMw. During the planning period we aim to increase the number of participating PGOs by at least three. In addition, we want to increase our working capital by obtaining subsidies and donations.
The funds for the project are managed by the board of the foundation, which consists exclusively of volunteers who receive no remuneration for their work. The funds are spent on costs to keep the platform functional and, where necessary, to expand it in accordance with the wishes of the users.
Results in the longer term
Completion of the platform will result in the translation facilities of the platform, both nationally and internationally:
- PGO participants will gain insight into various aspects of a rare disorder, in the development of the platform and in the use of the platform.
walk with their child and in the best developmental opportunities and stimulation of their child, - PGO participants can ask questions to health care providers, researchers and experts and vice versa.
The anonymised answers remain accessible to others. - the 'empowerment' of PGO participants will be increased and they will be able to take more control of their own lives. Through WaihonaPedia they experience that they are not alone and that they can learn from others (support) and help others again with their own knowledge and experience.
- Healthcare providers and researchers can gear their research to the needs of PGOs. The system may in the future be able to send notifications on the basis of a unique profile if new information on a preferred topic becomes available, thereby not only achieving but also proactively 'bringing' information.
- new PGOs can join quickly and easily.
