Friday, 2 March 2018

Rare Disease Day 2018 – Research, Taking Part in Clinical Trials. Scleroderma, Raynaud's, Autoimmune Rare Disease. Nicola Whitehill.


Rare Disease Day 2018
Research
Scleroderma, Raynaud's, Autoimmune Disease



Today, February 28th, is Rare Disease Day.

This year’s theme is Research.   



2017 Vlog 'Research'

Living with a rare disease is daunting in every way imaginable. Having been a diffuse scleroderma, rare disease patient for 21 years, in my view, Research provides immense hope.   

Hope that the desperately needed understanding as to cause and cure will be discovered. With an improvement in medicines, being a soother in the meantime. 

Rare Disease Day provides a spotlight for the rare disease community to come together. 

 

So, what is a rare disease?

A disease or disorder is defined as rare in Europe when it affects fewer than 1 in 2000.  
A disease or disorder is defined as rare in the USA when it affects fewer than 200,000 Americans at any given time.  


There are 2.5 million scleroderma patients worldwide, as by The World Scleroderma Foundation

The Role of Medical Research to the Rare Disease Patient, plays a pivotal role in improving the rare disease patient experience.  


 
This year’s theme of Research focusses on how important the patient is, to the entire medical research arena. 

Including, clinical trials, from the design to the actual live participation.   

During the course of the last few decades, I have taken part in several clinical research trials.   

I am not the most willing of participants for taking any kind of drug, especially not licensed drugs. However, for medical research clinical trial purposes, all ‘mystery’ medications have undergone rigorous testing, prior to reaching the clinical trial stage of testing on humans. Although, this does not eliminate every or any potential risk of taking the drug.   

For a new medication to reach the market place, strict criteria must be met for the medication to become licensed for the indications which it represents. This is where the design of the clinical trial is imperative with its detail.   

In 2015 I became a patient expert adviser with The European Medicines Agency.      

I very much enjoy this role   
 
I have taken part in various  Scientific Advice Working Party SAWP and Committee for Orphan Medicinal Products COMP meetings.    


It is very exciting for me to see in this setting, the increase in pharmaceutical interest for scleroderma patients.    

I have been involved with many clinical trial design set ups with chemical substances which only have a number, and have not even been named yet.    

In relation to the clinical trial design for scleroderma patients- ALL clinical trial assessment criteria now includes digital ulcer involvement, further to my insistence!  

I am delighted to have recently become a Patient Research Ambassador for the Dermatology and Musculoskeletal departments at The Royal Free Hospital.

 
There are many clinical trials currently taking place at the Scleroderma Unit.  


The Christmas 2017 newsletter gives a summary of Research progress so far at the Scleroderma Unit. 

All of the trials which I have participated in, have been conducted at the Unit.  



Sadly, there remains many unmet clinical needs to the scleroderma patient.    

The Pandora’s Box continues to present challenging surprises, making my symptom management a full time job.   


 
This year’s Scleroderma Family Day is to be held on Saturday 19th May in the atrium at the Royal Free Hospital. To view the program, click here 



I am very much hoping to attend this year's family day, as I am celebrating my 20th year anniversary of being a patient at the Unit. 

This Scleroderma Olympian is already in training, currently in full time hibernation.     


Unfortunately, I will not be attending the Rare Disease UK Parliamentary Reception, this year. 

However the 2016 parliamentary reception still remains one of my few patient highlights


  
The UK has a Strategy for Rare Disease. 


2017 Rare Disease Day Flashback       

I discussed other areas of medical research interest within Week3 of my Patient Profiles Campaign  for Scleroderma Awareness Month 2017, Flashback 

As well as highlighting the need for RESEARCH investment.   



The 'rare disease' label can often feel like a bleak and lonely existence.

Although rare disease patients are few in number, eg. 2.5 million scleroderma patients worldwide (as by the World Scleroderma Foundation), the commonalities and golden hallmark for each rare disease patient are the same:


Early Diagnosis
Expert Specialist Centres
Access to Innovative Medicines 


Where MEDICAL RESEARCH investment is VITAL 
to improving the entire rare disease landscape, including, the rare disease patient's day to day reality. 

Thereby, unlocking the rare disease enigma.
Living the dream, scleroderma style.  

 

Please DONATE to help fund medical research at The Scleroderma Unit,The Royal Free Hospital, London where 100% of all of your monies will be used for medical research purposes only. Thank You. 

Wishing you a very happy Rare Disease Day, feeling good.  
#SclerodermaFreeWorld #RaynaudsFreeWorld  #HOPE
#RareDiseaseDay   
Follow Raynauds Scleroderma Awareness Global Patients Facebook Page for latest updates   




An edited version of this article was published here, in my Column with Scleroderma News.  
February 2018


To read my articles: 


Rare Disease Day 2018, Research - Taking Part in Clinical Trials, Click here   

March Autoimmune Disease Awareness Month 2018, Click here   

Global patient video, Click here   

Becoming a Patient Research Ambassador for the NIHR, Click here  

If we only had more RESEARCH investment for Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here   


Scleroderma Awareness Month 2017 Patient Profiles Campaign, Click here     
 
Scleroderma Awareness Month 2017 Patient Profiles Campaign Patient Index, Click here        
The Pandora’s Box of the rare autoimmune disease Scleroderma, Raynaud's and Cancer, Click here


Key Event Dates 2018, Click here     

Importance of an early diagnosis, Click here     

Taking Part in Clinical Research Trials, Click here    


The Importance of a Multi Disciplinary Medical ‘Dream Team’, Click here 

Expert Specialist Centres, Click here      

My Skin is Cured from Scleroderma, Click here      

UK Guidelines for Managing and Treating Scleroderma, Click here      

Fatigue, Click here      

Mobility, Click here    

Diet and nutrition, Click here     


Raynaud's, Click here    



World Scleroderma Day 29th June 2016, Click here

World Scleroderma Day 29th June 2017, Click here   


Why Global Collaboration is important to the Rare Disease Patient, Click here      



RARE DISEASE DAY:
The theme to this years Rare Disease Day is Research

Rare Disease Day 2018 – Research, Taking Part in Clinical Trials. Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here     

2016 Rare Disease Day Patient Voice
2016 Rare Disease UK Parliamentary Reception 

2017 Rare Disease Day Flashback  

More unmet clinical needs  

Rare Disease Day 2016



Rare Disease Day is a fantastic opportunity for the entire rare disease community to shine a spotlight on their reality, combining as one unified voice. Where, at least one commonality presides –  
Medical Research provides the brightest light
for the illumination of the rare disease patients’ plight. 

I highlighted other areas of medical research interest within Week 3 of my Patient Profiles Campaign for Scleroderma Awareness Month 2017. 

VIDEO



Although rare disease patients are few in number, eg. 2.5 million scleroderma patients worldwide, (the World Scleroderma Foundation), the commonalities and golden hallmark for each rare disease patient are the same: 

Early Diagnosis
Expert Specialist Centres
Access to Innovative Medicines

Where MEDICAL RESEARCH investment is VITAL.

Research is the key. Abstracts from 2016 World Congress, Click here


Scleroderma Awareness Month Campaign 2016, Click here

Links

View video, here   

Preamble - here



The Family Day at the Scleroderma Unit, The Royal Free Hospital is taking place on the 19th May 2018. Browse the program, here

This year, I am celebrating 20 years of being a patient at this world leading expert specialist research centre. 

Sept 2017


I am eternally grateful to the global scleroderma trail blazers Dame Prof Black and Prof Chris Denton, whose commitment and dedication to unlocking the scleroderma enigma, is nothing other than, superhuman.
I am truly humbled and inspired by their work ethic. I am wholly appreciative for Prof Denton’s continued medical expertise and support, especially during my barrister qualifying years, 1997 - 2004. 

1st March 2004, I qualified as a self employed practising barrister. Further to having been told in 1997, by my diagnosing doctor, that I was looking at a 15month prognosis. 

I very much hope to utilise my professional skills and qualifications along with my patient experience, to help achieve the #SclerodermaFreeWorld dream, hoping to improve understanding and best practice, in the meantime.
  
For latest updates follow: 

Facebook Page:

Twitter: @SclerodermaRF  @RaynaudsRf  


#SclerodermaFreeWorld           #RaynaudsFreeWorld
#ADAM #Scleroderma
#Raynauds
#RareDisease  

Living the dream, scleroderma style.  


Please DONATE to help fund medical research at The Scleroderma Unit,The Royal Free Hospital, London.  



100% of your monies will be used for medical research purposes only. No wages or admin costs. Thank You.
#HOPE 


 

Last Update: April 2018




No comments:

Post a Comment