Patient Influence on Decision Making Creates Win-Win Situation
This year's Rare Disease Day EURORDIS Policy Event in Brussels was an unprecedented success with over 150 participants. Speakers at the 'Rare but Real: Talking Rare Diseases' event - including patients, EU Commissioner for Health Vytenis Andriukaitis and Belgian Member of the European Parliament Philippe De Backer - discussed how patients' influence on decision making results in a positive impact on rare disease policies.
The event's new discussion format included four talks, each of which presented successful examples of interactions between patients and various stakeholders. The event was also open to participants across the world via a live stream video shown on the EURORDIS website (now available on EURORDIS TV). Viewers from over 30 countries tuned in to watch live.
Commissioner Andriukaitis opened the event by saying, "I believe European action can make a difference in improving the lives of people with rare diseases; people who struggle to find the rare expertise to diagnose and treat their disease. I am committed to working with EURORDIS and all stakeholders to maximise our work towards delivering European solutions to address rare diseases".
Kathy Redmond, Editor of Cancer World magazine and event moderator, started the discussion by emphasising that the inclusion of the patient perspective in the decision-making process benefits all stakeholders, and not just patients, therefore creating a win-win situation for all involved.
Bojana Mirosavljevic, the mother of a Batten disease patient, gave an emotional account of her tireless campaign to influence change in the prenatal screening law in Serbia. She told of her struggle to get a diagnosis and insurance for her daughter Zoya, who passed away from Batten disease in 2006. The disease did not have a code within the Serbian healthcare system, making it impossible to access treatment and reimbursement for therapies or other healthcare services. Bojana and her husband did not want any other family to live through their experience; they succeeded in changing Serbian legislation (Zoya's Law) that now requires doctors to send samples abroad if a diagnosis has not been reached within six months. Families with one child living with a genetic disease are given access to prenatal testing at no cost and genetic testing is offered to other family members. Hajrija Mujovic Zornic, a legal consultant who is responsible for parts of Zoya's Law,commented, "When Bojana approached me it was clear that the presence of the patient voice would not only help Bojana's case but also a larger number of rare disease families."
MEP Philippe De Backer spoke boldly on the changes needed in Europe, "Access to healthcare and treatment still differs considerably across Member States. Setting up a common European structure is the only way to make access possible for all. The result would be a fairer and more transparent system, and ultimately one that results in better access for patients."
Philip Watt, CEO of patient group Cystic Fibrosis Ireland, discussed the instrumental role that the organisation played in reversing the reimbursement decision of a cystic fibrosis drug in Ireland. As a result of the organisation's actions and a public awareness campaign, over 100 patients were able to access the drug, which has a substantial impact on quality of life and life expectancy. Ri De Ridder, Director General of RIZIV-INAMI, took part in the discussion and emphasised that it is important to recognise that when carrying out a drug reimbursement evaluation quality of life is often a much higher priority for patients than life expectancy. He explained that this has been done in Belgium by creating a multi-criteria decision-making framework.
Carla Fladrowski, the mother of a tuberous sclerosis patient, discussed her experience of collaborating with a pharma company on the design of a clinical trial protocol. She told the audience that at the time of her son's diagnosis she did not realise how important it is to be involved in this process, commenting, "Parents of rare disease patients are the glue between different healthcare professionals involved in the treatment of a patient. My empowerment comes from the fact that I am an expert on my disease".
Speaking alongside Carla, Veronica Foote of Novartis commented, "Not only do patients need to be trained in order to be appropriately engaged, but researchers and clinicians need to understand the importance of such dialogue with patients. There is nothing more rewarding to scientists than to meet the patients they are ultimately helping. The EURORDIS Charter for Clinical Trials in Rare Diseases has been instrumental to the process."
Helma Gusseck, a retinitis pigmentosa patient and Nathalie Bere of the European Medicines Agency(EMA) discussed Helma's contribution to a scientific advice procedure at the EMA. Helma emphasised that good cooperation between patient experts, the EMA and a pharma company provides an excellent opportunity for the patient to promote the last step of bringing new therapies to the market. Through initiatives such as the EURORDIS Summer School, patients are able to learn how to become advocates. Helma said, "The EURORDIS Summer School was an important learning process; I saw a key difference from my first to fourth visit at the EMA and I feel that my words are now more important than the first time I participated." Nathalie told the audience that 50% of patient contributions to protocol assistance result in advice being given to a company.
Terkel Andersen, EURORDIS President, concluded the event by saying "Today we have seen moving examples of not only the challenges patients and parents live with day-to-day, but of the extra courage they need and effort they make to influence policy."
Eva Bearryman, Junior Communications Manager, EURORDIS
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