ROHHAD Association Research Strategy - ROHHAD Association

ROHHAD Research Strategy
Why Medical Research into ROHHAD Syndrome is so important

ROHHAD Syndrome is a cruel, complex disease that is extremely rare. Sadly, for many of the children diagnosed, the condition is life limiting and often means that few will reach adulthood. Monitoring and treatment will require of a lifetime of hospital stays, appointments and invasive procedures. The risks to life are serious; both the quality and quantity of life is significantly reduced for those affected and without medical research into ROHHAD this will never improve.

The primary aim of the ROHHAD Association is to fund and support medical research into ROHHAD to gain a better understanding of the disease, improve treatments and work towards developing a cure. We will continue to do all that we can to raise awareness and funds so that we may achieve all of this.

To date there has been limited progress in ROHHAD Research. We would like to drive change and with hard work from our charity trustees, the families affected and our supporters around the world we hope to advance research.

Our research priorities are set out in the section directly below. These priorities are reviewed intermittently and annually to reflect advances in technology, medical care standards, and our increasing knowledge of ROHHAD Syndrome.

We publish all of the latest information in the ROHHAD Reader, which is a bi-annual newsletter (click here to read). We also publish updates twice a year on all research projects we fund and partly fund. Further information is available on our website or alternatively you can contact us directly. 

If you are a researcher wishing to apply for funding please contact the charity directly via rohhadassociation@gmail.com and we will share with you the details of the type of research we will support, deadlines for submission of grant applications and the criteria for applying for research grants.

Alternatively click here to view this information for the funding process for 2019/2020 (please note proposals are now being accepted for 2019/2020 and are open from Friday the 16th of November 2018 until the closing date of 1st of February 2019) .

Our Research Priorities for funding during 2019/2020

In order to decide our priorities for ROHHAD Research this year we consulted those it affects the most. We contacted all of the families affected by ROHHAD that are on our mailing list and asked them what is most important to them when it comes to ROHHAD Research.  The families voted, by majority, for researchers to find the cause, improve treatments and have clinicians and scientists work collaboratively in the hope that they will one day find a cure.

The following priorities have been agreed:-

Those applying for funding must fit at least one of our priorities for 2019/2020.  (All applications must also meet the criteria for funding which you can view here).

1. Understanding ROHHAD better by focusing on finding the cause or trigger for the disease.  Thus far no research has led to a definitive cause for ROHHAD Syndrome. If you can find the cause of the disease, be it autoimmune, genetic or something else entirely, knowing a cause will help physicians understand ROHHAD better and could lead to an earlier diagnosis and even a cure therefore any research that focuses on understanding ROHHAD better and finding the cause of the syndrome/disease is our main priority.
2. Improving treatments for ROHHAD Syndrome are very important to the the quality and quantity of life for those directly affected by ROHHAD.  Therefore research that focuses on one particular area of the ROHHAD syndrome acronym will be considered, so long as it is trying to improve treatments relating to that particular area of the syndrome.
3. Shaping or devising a care pathway for ROHHAD Children/Adults.  Due to how rare ROHHAD Syndrome is and how little is understood about the disease there is not a clear care pathway or indeed any care pathway for those affected.  Clear Care Pathways for other rare diseases and more common disease have been proven to help improve the quality of life for the patient affected.  If your research proposal could potentially lead to a care pathway or if your research proposal includes developing a care pathway then it will be considered.
4. Collaboration between doctors and researchers who are actively working on ROHHAD Research or who wish to actively work on ROHHAD Research.  Working together is something the families and we at the charity can see will hugely benefit ROHHAD Research.  Therefore we will consider all proposals from those who are working together collaboratively with other doctors or scientists from anywhere in the world.  That is not to say that we will not consider individual applications however collaboration proposals will be given a priority when being considered provided those involved are qualified to be working on ROHHAD Research.
5. Finding  A Cure, this is a priority for all families affected however we recognise at the charity that this would be difficult to ascertain and find without extensive research and improved understanding of ROHHAD Syndrome first therefore this is not a requirement for funding to be approved during this round of funding applications.  Finding A Cure though will remain a priority for the ROHHAD Association and the ROHHAD families and any research applications that are exploring a cure will be considered for funding. 

What happens after funding is approved?

Any research proposal approved for funding will have set time-scales and a contractual agreement in place. Researchers will agree to update the ROHHAD Association Trustees on a regular basis as set out within their contract, the minimum being once every 6 months.  We will update those supporting the charity via our website and other publications.

We will work closely with the researchers, before, during and after all research is being carried out and support them fully as per the agreed contract.

Once the grant and award period is complete.  Researchers will publish their ROHHAD Research findings and the ROHHAD Association charity will help them disseminate those findings.  We will also share the published papers on our website.

Medical Research and Animals 

We understand that using animals in medical research is a very sensitive subject, but we also recognise that there is a real and urgent need to find a cure for ROHHAD Syndrome.

Where possible we favour the use of non-animal experimental approaches to find a cure for ROHHAD such as cultured cells or human tissue equivalents, however we recognise that there are instances where animal test subjects are necessary.
The use of animals in medical research is strictly controlled. Legislation typically only allows animals to be used in medical research where the potential benefits the work is likely to bring outweigh the effects on the animals concerned.
All new medicines in the UK are required by law to use animals during development and safety testing, however as a charity we would like any research funded by the ROHHAD Association to commit to the following:
Replace where possible the use of animals with other lab techniques
Refine the use of animals to minimise their use and the severity of procedures carried out
Reduce the number of animals used to the minimum

The research funding process and how we plan to fund research in 2019/2020

This year the ROHHAD Association will put out a 'call' to ROHHAD researchers around the world. This call asks them to submit proposals for funding, to support their ROHHAD research, in areas that the ROHHAD Association has determined to be a priority. Researchers must apply by submitting a proposal.  The proposal must include all of the stipulated requirements and meet the criteria. 

We are delighted to now be in a position to offer financial grants for ROHHAD Research and begin the process of funding research for the year 2019/2020.

The funding we have available for 2019/2020 is £275k which will be awarded as a grant(s). We will consider requests for full or part funding.

ROHHAD is extremely rare and there are a limited number of Professors, Doctors and Scientists who will be in a position to research it.  Therefore how we release our funding will be developed in response to the applications for funding and the amounts required.  

Please note, researchers must be qualified either in medicine or science.  Those applying for funding should have experience or at the very least a keen interest in ROHHAD Syndrome.  Since we are based in the United Kingdom the professional who is heading the research must be at the very least a consultant in one of the specialities that helps to treat ROHHAD children for example an endocrinologist. The equivalent qualifications elsewhere in the world are also required.  

We may consider funding for research from student doctors; so long as it is supported by a consultant with experience in treating ROHHAD Syndrome.     

We will accept proposals from anywhere in the world.  We do not have a United Kingdom or European restriction. 

Some information for ROHHAD families regarding current ROHHAD Research

We have recently been asked by the team at the Ann & Robert H. Lurie Children's Hospital of Chicago to include the following information on our website.  We are more than happy to share this with you all and ask that any family affected by ROHHAD who isreading this for the first time to please either contact us here at the charity for further information or click on the links below.

Lurie Childrens Hospital are currently undertaking some Research into ROHHAD, they have started an international registry for children diagnosed with ROHHAD and they are actively seeking participation in this Registry

An international Registry will help any team researching ROHHAD and allows a collaboration of the number of children diagnosed around the world as well as a collaboration of key information about the children affected.

Please click the following links for further information about the registry and details of how you may be able to participate.

https://www.luriechildrens.org/en-us/care-services/specialties-services/center-for-autonomic-medicine/research/Pages/international-rohhad-redcap-registry.aspx

https://redcap.nubic.northwestern.edu/redcap/surveys/?s=THHEHWHRX9

The hospital at Chicago is also currently updating their website to include some other ROHHAD studies that they are currently doing and are preparing to do.  It may also be that you would be interested in participating with these studies.  

Participation in the International Registry requires the Online interest form to be completed. Participation in the other studies requires participation in the International Registry.

If you have any questions relating to the International Registry you can contact us here at the charity and we will happily try to help you.  

What happens after the researchers submit their proposals ?
If the proposal meets the criteria set out by the charity it will be sent for peer review where it will be assessed by other medical and scientific experts in the area. The remit if peer review is to provide feedback and advice for the trustees(the applicants are not told the identity of the peer reviewers) on each proposal.
Once all proposals have been reviewed, the trustees will make a decision on the most appropriate proposal that it will help support.  All applicants will be notified of the decision and outcome within one week of the decision being made.

Time-scales for 2019/2020 Grant applications

The ROHHAD Association will be accepting proposals for funding for medical Research into ROHHAD Syndrome for the year 2019/2020 from Friday the 16thof November 2018.  The closing date for proposals for funding is Friday the 1st of February  2019.

The ROHHAD Association will endeavour to be ready to release funding by the 30th of April 2019. (these dates are subject to change and funding may be released sooner or slightly later dependent upon the peer review process).  

Each applicant will be notified in writing if the process is likely to take longer than planned.

Each applicant will be notified in writing of the outcome of their application for funding and if the proposal has been accepted within one week of the decision on funding being made.

Supporters of the charity will be notified as soon as possible after funding has been approved; of whom we have agreed to support financially in ROHHAD Research. We will also release regular updates on how the research is progressing via our website and social media pages. 

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