Monday, 5 January 2015

My European roles and NHS-Citizen; Nicola Whitehill

In the autumn of 1997, I was diagnosed with diffuse systemic sclerosis (ssc) and raynauds, 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 raynauds 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 Nikki Whitehill Jan 2015