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, Taking Part In Clinical Trials.
Scleroderma, Raynaud's, Autoimmune Disease.


Today, February 28th, is Rare Disease Day.  

 
 
This year’s theme is 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 



 
2019 theme: Bridging Health and Social Care. 
 
2018: Research.   
 
Rare Disease Day 2018 – Research, Taking Part in Clinical Trials. Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here     

2017 Rare Disease Day Flashback  
 
2016 Rare Disease Day Patient Voice  
2016 Rare Disease UK Parliamentary Reception    
 
 
 
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.
 
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 overall: 
 
For optimum patient care, 3 hallmarks preside:



supported by a medicaldream team’ 
(expert specialist as the clinical lead).

Where, investment in medical research is crucial, to improve the day to day reality, as well as, the long term outlook for the entire global scleroderma community. 
 
To read my articles:
 
Becoming a Patient Research Ambassador for the NIHR, Click here   
 
NIHR Video: 'My Experience of Clinical Trials', Click here 

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

The Importance of Medical Research and Awareness to the Scleroderma, Raynaud's, Autoimmune Rare Disease patient, Click here  

2018 Scleroderma Awareness Raising and Medical Research, Click here    

 

SCLERODERMA:

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- How to Diagnose, Click here      


Sept 2017

Prof Chris Denton and I, Sept 2017
 
This year, 2019, I am celebrating 21 years of being a patient at the Scleroderma Unit, The Royal Free Hospital - a world leading expert specialist, research centre. 
Read more, here
 
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. Along with the Raynaud's world trail blazer, Dr Kevin Howell. 
I am truly humbled and inspired by their work ethic and commitment to their patients. 
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. Read more, here     
 
 
The annual Family Day at the Scleroderma Unit, The Royal Free Hospital, is taking place on the 18th May 2019. Browse the program, here


March 2018 Autoimmune Disease Awareness Month, Click here    

 
World Scleroderma Day 2018, Click here 
World Scleroderma Day 2017, 29th June, Click here   
World Scleroderma Day 2016, 29th June. Origins of the date, Click here  
World Scleroderma Day 2015, 29th June. 

James Carver, myself, Prof Chris Denton
I had the pleasure of being a presenter and part of the European co-hort who gave a presentation at the European Parliament, Brussels, in honour of World Scleroderma Day 2015.  

I am immensely grateful to James Carver MEP for organizing this event in memory of his late wife Carmen, who sadly passed from Scleroderma.  

To view the presentation, Click here 

Raynaud's
October: 
 
Raynaud's, Click here  
 
To view the UK treatment guidelines for Raynaud’s Phenomenon, Click here   

To view the EULAR treatment guidelines, Click here    
To view Thermograph Video, Click here   
To view Thermograph image, Click here   
   
My Raynaud’s reality, Click here   
 
The global Raynaud's trailblazer - Dr Howell and I, Sept 2017
 
To view Nailfold Capillaroscopy equipment (used to identify possible blood vessel damage), Click here  
 
To read My ‘Invisible Disability’ experience, Click here  

  


Facebook Page: Raynauds Unit Royal Free Hospital
  
Twitter: @RaynaudsRf 

 

 
 

 
For latest updates follow: 
  
Facebook Page:
Twitter: @SclerodermaRF  @RaynaudsRf  
#SclerodermaFreeWorld           #RaynaudsFreeWorld    #Research
#ADAM #Scleroderma #Raynauds #RareDisease  #RareReality #ShowYourRare 
#RareDiseaseDay   

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. Thank You. 

  
My article for Rare Disease Day 2016 was published on the Rare Disease Day website and the Rare Disease Day social media platforms.

  

 
Last Update: February 2019.

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