Agreed Statements and Actions
Proposed Champions: Martine Pergent, Vicki Modell & Dr Jose Franco
Primary immunodeficiencies (PID) are often misdiagnosed, leading to unnecessary illness and suffering. To increase the rate of diagnosis of these disorders there must be well orchestrated awareness programmes to match the needs of each individual country at a political and social level. Those campaigns must be easily understood both by family doctors and specialists.
Proposed Champions: Dr Monika Esser, Dragana Koruga, Roberta Pena
Once diagnosed, most PID patients need access to a number of therapies, depending on the condition. The majority of patients have an antibody deficiency and require life-long immunoglobulin (Ig) replacement therapy, which is available from human blood and plasma donations. Ig is a proven treatment throughout the world and enables most people receiving it to lead full and productive lives. Failure to receive this treatment results in unnecessary hospitalisation and major reduction of quality of life and life expectancy. In addition each country needs to provide infrastructure support for bone marrow transplant and stem cell therapy to save the lives of severely affected patients.
Proposed Champions: Sven Fandrup, Stephen Baxter & Bob Perry
There needs to be sustained, regular and effective communication between all those committed to patient care including patients, caregivers, clinicians and researchers, patient group representatives, regulators, manufacturers and political decision makers.
4. DATA COLLECTION
Proposed Champions: Marcia Boyle, Joy Rosario & Charles Waller
Data from patient registries and patient and physician surveys is invaluable in providing the information needed to demonstrate to those who make policy decisions the best strategies for success and the consequences of non-action.
Proposed Champions: Prof Helen Chapel, Dr Espanol & Dr Klaus Warnatz
Diagnosis and management need to be directed by frequently updated Guidelines, as well as Guidelines that emphasise priority usage of Ig and other valuable plasma-derived products.
- Primary immunodeficiency diseases are under-diagnosed, life threatening, but treatable disorders and patients globally face similar issues of diagnosis and access to appropriate specialists and optimal treatment.
- Because the needs of the patients are great and resources are limited, it is imperative that we work even more closely together by having open, honest, productive, relationships as stakeholders in our efforts to educate physicians and health policy makers. Only then, by sharing and understanding goals, will we achieve the optimum for each other.
Registration No. 1058005