My European roles and
NHS-Citizen; Nicola Whitehill
Scleroderma, Raynaud's,
Autoimmune Rare Disease.
In the autumn of 1997, I was diagnosed with
diffuse systemic sclerosis (ssc) and Raynaud’s, aged 24, and given a 15month
prognosis.
Now, at the age of 41, having stabalised my
initially very aggressive disease to a manageable level, I feel well enough, to
use some of my experience to get involved in any way that I can, to help in the
understanding of this rare, life threatening diagnosis.
In the summer of 2014 I attended my first
meeting as a patient expert with the European Medicines Agency (EMA).
This was a very exciting day for me, as not
only was it the hottest day of the year (thank goodness that I didn’t have to
wear my Eskimo outfit, and I was back in my suit), the offices are located at
Canary Wharf overlooking the River Thames.
And so, it was quite an adventure to just get
there, with me living in the seaside town of Southport, on the north west
coast.
However, the location more than compensated for
my physical efforts that day. Entry into the building as well as to the meeting
room, I can only liken to something similar to that which I have seen in a
James Bond movie.
My fellow colleagues made me feel very welcome
and at all times, they ensured that I was included in the discussion, often
asking me for my view.
The EMA is a decentralised agency of the
European Union, responsible for the scientific evaluation of medicines developed
by pharmaceutical companies for use in the European Union.
It began operating in 1995.
It is the hub of a European medicines network
comprising: over 40 national regulatory authorities; the European Commission;
the European Parliament; and the World Health Organisation (WHO).
Its aim is to build the best possible
regulatory system for medicines for Europe and protect the health of its
citizens.
These activities aim to foster the timely
exchange of regulatory and scientific expertise, and development of best
practices in the regulatory field across the world.
The EMA’s main responsibility is the protection
and promotion of public and animal health, though the evaluation and
supervision of medicines for human and veterinary use.
The EMA has several further roles which it is
responsible for, including Marketing Authorisations, Safety Monitoring,
Referrals, Inspections and The Telematics systems exchange information, helping
to provide high quality information on medicines to the general public and
support the monitoring of the post authorization benefit-risk balance of
medicines in the EU.
Most of the EMA’s scientific evaluation work is
carried out by its scientific committees, which are made up of members from EEA
countries, as well as representatives of patient, consumer and
healthcare-professional organisations.
These committees have various tasks related to
the development, assessment and supervision of medicines in the EU.
The EMA is also responsible for coordinating
the EU’s safety monitoring or ‘pharmacovigilance’ system for medicines.
It constantly monitors the safety of medicines
through the EU network and can take action if information indicates that the
benefit-risk balance of a medicine has changed since it was authorised.
The EMA’s committees are involved in referral
procedures to resolve issues such as concerns over the safety or benefit-risk
balance of a medicine or a class of medicines.
In a referral, the EMA is requested to conduct
a scientific assessment of a particular medicine or class of medicines on
behalf of the EU.
The matter is the ‘referred’ to the EMA so that
it can male a recommendation for a harmonized position across the EU.
The Agency also plays a role in stimulating
innovation and research in the pharmaceutical sector:
- it gives scientific advice to companies on
the development of new medicines;
- it publishes guidelines on the requirements
for the quality, safety and efficacy testing of medicines;
- it provides special assistance to micro,
small and medium sized enterprises (SMEs) through its SME office;
- it issues opinions on orphan designation for
medicines for rare diseases;
- it manages the Innovation Task Force, a group
that provides a forum for early dialogue with applicants.
Scientific Evaluation:
The Agency’s scientific committees are made up
of independent professionals nominated by Member States from a pool of over
4,500 European experts.
The committees are responsible for the
scientific evaluation of marketing authorisation application dossiers submitted
by pharmaceutical companies, as well as for providing opinions on referrals and
other issues impacting on public health, at the request of the Member States,
the European Commission or the European Parliament.
All committee members are required to make an
annual declaration of any direct or indirect interests they have in the
pharmaceutical industry.
The Agency publishes these declarations of
interest online.
The EMA has seven scientific committees that
carry out its scientific assessments :
- Committee for Medicinal Products for Human
Use (CHMP)
- Pharmacovigilance Risk Assessment Committee
(PRAC)
- Committee for Medicinal Products for
Veterinary Use (CVMP)
- Committee for Orphan Medicinal Products
(COMP)
- Committee on Herbal Medicinal Products (HMPC)
- Committee for Advanced Therapies (CAT)
- Paediatric Committee (PDCO)
The work of these committees is supported by
Working Parties and other groups.
I am a member of The Committee for Orphan
Medicinal Products (COMP).
This is the committee at the EMA that is
responsible for reviewing applications from people or companies seeking
'orphan-medicinal-product designation'.
The COMP is for medicines to be developed for
the diagnosis, prevention or treatment of rare diseases that are
life-threatening or very serious.
In the EU, a disease is defined as rare if –
it affects fewer than 5
in 10,000 people across the EU.
The COMP is also responsible for advising the
European Commission on the establishment and development of a policy on orphan
medicinal products in the EU, and assists the Commission in drawing up detailed
guidelines and liaising internationally on matters relating to orphan medicinal
products.
So far I have been involved with several COMP
meetings- the majority of which, I have been able to participate in, without
having the physical exertion of going to London.
All of the relevant paperwork and documents are
forwarded via a secure electronic system network (James Bond style, again), and
the meetings can be accessed by teleconference.
This participation arrangement has worked
perfectly for me, as my energy, pain and stamina levels are easily exhausted.
This arrangement allows me to be respectful of
my symptoms whilst being able to provide an active contribution.
I have found the content of the meetings to be
most stimulating, and I am extremely hopeful for the future for newly diagnosed
ssc and Raynaud’s patients.
I take huge comfort and encouragement in seeing
for myself that the pharmaceutical companies, as well as the leading world ssc
experts, are relentless with their research for improving the medicines and
treatments for this disease, with the ultimate accolade being to discover the,
so far, unknown, cure.
I have also become an expert with EURORDIS – a
non governmental patient driven alliance of patient organisations and
individuals active in the field of rare disease, dedicated to improving the
quality of life of all people living with rare diseases in Europe.
It is the voice of 30 million people affected
by rare diseases throughout Europe.
The mission of EURORDIS is to build a strong
European community of patient organisations and people living with rare
diseases, to be their voice at the European level, and – directly or indirectly
– to fight against the impact of rare diseases on their lives.
So far, I have been able to fulfill my role by
engaging from home, using the internet.
All in all - I would urge ANYone to get
involved in any way you can, with anything which will help the experts / new
patients, understand this cruel, life- hijacking diagnosis.
There has never been a better time for patients
who are practically housebound, to get involved with being active about their
disease, and taking some responsibility for their own health, due to the power
of the internet.
And here in the UK we have The Equality Act
2010, which stipulates equality for all.
So, all public meetings, legally, should be
made available to participants on-line in the event that a person is unable to
attend due to their disability.
I am also an active on-line participant with
NHS–Citizen, an advisory partner to NHS-England.
I was delighted to have the pleasure of
chatting with Simon Stevens (CEO NHS-England) in a chat room, discussing the
transfer of medical records between departments, at a recent public meeting.
This was very uplifting for me, as I had been
quite upset that I was unable to attend the meeting in person due to my
disability.
NHS-Citizen is a forum, which if you have
internet access, I would thoroughly recommend you become involved with - to
help improve the amazing service which we have here in the UK, our National
Health Service (NHS).
I am eternally grateful to the NHS, along with
my dedicated medical team, to have kept, and continue to keep, me alive.
In the meantime, I continue to share some of my
survival tips and daily coping mechanisms on my blog www.cosmicfairy444.blogspot.co.uk
This article was written for the Raynaud’s and
Scleroderma Association’s Spring Newsletter Jan 2015.
In 2016, Eurordis published my story on their
website here
PLEASE DONATE to the ROYAL FREE CHARITY, to
help fund desperately needed MEDICAL RESEARCH at the SCLERODERMA UNIT, THE
ROYAL FREE NHS HOSPITAL.
#SclerodermaFreeWorld #RaynaudsFreeWorld #Research
#Autoimmune #RareDisease #LifeChanging
Scleroderma Family Day
2020
25th
Anniversary Meeting
The Atrium, Royal Free
Hospital, London, NW3 2QG
Chair: Professor Chris
Denton
PROVISIONAL PROGRAMME
09.30 – 10.00 Registration
and Coffee
10.00 – 10.20 Welcome Dame
Carol Black
Prof Chris Denton &
David Abraham
10.20 – 10.40 What
is a Biopsy? Dr Kristina Clark
10.40 – 11.05 Dental
aspects of Scleroderma Prof Stephen Porter
11.05 – 11.25 Gastrointestinal
problems – Dr Fiza
Ahmed
shedding
new light on old problems
11.25 – 11.50 Pulmonary
hypertension and the heart Dr Gerry
Coghlan
11.50 – 12.15 Scleroderma
cohort studies – Dr
Francesco
‘Learning
from our patients’ del Galdo
12.15 – 14.15 LUNCH
BREAK – see below
14.15 – 14.45 25
years of progress – Prof
Chris Denton
from
‘black box’ to ‘positive trials’
14.45 – 15.15 International
speaker – Dr
Madelon Vonk
Scleroderma
Management in Netherlands
15.30 Raffle
Lunchtime Discussions Groups /
Demonstrations include:
Clinical Trials Rachel
Ochiel and team
National Institute of Health Research Christine Menzies
Drug Information / monitoring Pharmacy
Massage Keith Hunt MBE
Pulmonary Hypertension Education / nursing Sally
Reddecliffe/Adele Dawson
Rheumatology Laboratory Research
Laboratory Staff
Scleroderma Education / Nursing Louise
Parker/ Joseph Cainap
Thermography Dr Kevin Howell
SRUK Ollie Scott
Sjogren’s syndrome British
Sjogren’s Society
To Read My Articles:
Gift in My Will, Click here
Planning for the Future, Click here
Rare Disease Day:
Rare Disease Day 2020:
Listen to my interview with John Smeeton, (Silver Fox), from the Royal Free Radio, here
Rare Disease Day 2020:
Listen to my interview with John Smeeton, (Silver Fox), from the Royal Free Radio, here
Raise awareness and donate to medical research, order your #SclerodermaFreeWorld #RaynaudsFreeWorld tshirt here
Rare Disease Day 2019: Leaving a Legacy Gift, Click here
Rare Disease Day 2019: 11am 97.4RockFM headlines, NO CURE, Click here
Rare Disease Day 2018 – Research, Taking Part in Clinical Trials. Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here
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.
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: 
INVESTMENT in MEDICAL RESEARCH is CRUCIAL.
To read my articles:
Celebrating 20 years of being a patient at the Scleroderma Unit, Click here
2019 New Challenges, 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
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:
Calcinosis Video, Click here
The scleroderma tooth fairy, Click here
Skin Cancer and scleroderma, Click here
 |
| Sept 2017 |
 |
| Prof Chris Denton and I, Sept 2017 |
 |
| June 2019 |
 |
This year, 2020, I am celebrating 22 years of being a patient at the Scleroderma Unit, The Royal Free Hospital - a world leading expert specialist, research centre.
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 original diagnosing doctor, that I
was looking at a 15month prognosis.
 |
| Chat Magazine May 2019 |
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
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, former 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
 |
| The global Raynaud's trailblazer - Dr Howell and I, Sept 2017 |
For latest updates follow / subscribe:
@SclerodermaRF
@RaynaudsRf
@SclerodermaRF
@RaynaudsRf
Twitter, Instagram, Blogger, YouTube, Facebook Page:
#SclerodermaFreeWorld #RaynaudsFreeWorld #Research
Alternatively, to make a direct donation to fund medical research via the Royal Free Charity, Scleroderma Unit, Click here
100% of your monies will be used for medical research purposes only. NO wages or admin costs. Thank You.
My European roles and
NHS-Citizen; Nicola Whitehill
Scleroderma, Raynaud's,
Autoimmune Rare Disease.
