SclerodermaRF: My European roles and NHS-Citizen; Nicola Whitehill. Scleroderma, Raynaud's, Autoimmune Rare Disease.

My European roles and NHS-Citizen; Nicola Whitehill

Scleroderma, Raynaud's, Autoimmune Rare Disease.

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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.

www.ema.europa.eu/ema

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.

www.eurordis.org

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.

www.nhscitizen.org.uk/

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

www.eurordis.org/news/living-with-scleroderma-and-raynauds-disease

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

25^th 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

Raise awareness and donate to medical research, order your #SclerodermaFreeWorld #RaynaudsFreeWorld tshirt here

Rare Disease Day 2020: NIHR 'Research gives hope to Rare Disease patients', Click 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

2017 Rare Disease Day Medical Research

2017 Rare Disease Day unmet clinical needs to the scleroderma patient

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.

To read about medical research at the scleroderma unit, Click here

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:

Early diagnosis

Expert specialist centers

Access to innovative medicines

supported by a medical ‘dream team’

(expert specialist as the clinical lead).

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

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

'All you need to know', 2016 Awareness Video, Click here

Global Patient Profiles 2018 Video, Click here

Unmet Medical Needs, Click here

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.