Patients have a unique insight into the reality of living with an illness and are therefore in the best position to lead and inform both policy and research development. Informed patient involvement from initial stages of the planning process can be incredibly beneficial as it ensures proposed projects meet the ‘real life’ needs of the targeted patient population. It results in a more tailored project design which will work in the ‘real world’ for patients. Informed patients’ participation in the development of health policy and research-both nationally and internationally- is something which I support wholeheartedly.
As a person with Cystic Fibrosis (CF) and in working with Cystic Fibrosis Ireland (CFI), I have varied experiences of patient engagement with decision-makers. There has definitely been progress over the past year in Ireland, but there is certainly room for improvement–patient engagement is still happening too late in the policy process. Patients/patient representatives need to be involved in policy development and research design from the very beginning to provide the most value.
Resources also play a significant role for many patient organisations, which are often unable to fund a dedicated patient representative to provide input. To have valuable, informed patient representatives, they need to be recruited and remunerated accordingly; we must move away from the volunteer ethos surrounding patient advocacy. This would ensure secured participation for the duration of policy development and research projects. In my experience, my ability to dedicate time to projects through my role within CF Ireland has allowed me to be engaged at a significant level and have meaningful input.
Research shows that empowered patients have better outcomes than patients who do not engage in their treatments. From a young age, I have had an active interest in learning about CF and actively sought advice on how to improve my health outcomes. This has certainly had a positive impact on my overall health.
Health literacy in CF is extremely important as it is a progressive illness; symptoms and treatment change throughout a patient’s lifetime. One living with CF is required to visit a specialist team a minimum of five times a year, which provides opportunities to ask questions and learn about the illness. CF Ireland also helps in producing publications which detail information and promote literacy surrounding the disease.
Something which needs to be recognised across Europe is the need for access to high-quality healthcare and treatments for patients – in particular for people living with rare diseases. Last year, the drug I need was approved for use. The disease progression model predicted that this treatment has the potential to double the life expectancy of CF patients and clinical trial data reported a 55% decrease in hospital admissions among other promising benefits. However, the National Centre for Pharmacoeconomics (NCPE) deemed that the drug was not ‘cost-effective’ at a cost of €234,000 per patient per year.
It was extremely difficult to hear that a drug which could radically alter my health status was not going to be made available based on financial grounds. In situations like these, there is a responsibility on pharmaceutical companies to offer these life-changing drugs at a fair price, and there is a responsibility on the governing bodies to remember patients and their needs in their Health Technology Assessments.