The Global Leaders Meeting

IPOPI hosted a group of leading international experts in the fields of diagnosis and treatment of primary immunodeficiencies in London over the weekend of October 31 2009.

We were impressed by the global representation achieved – people from Latin America, South Africa, Egypt, India, the USA, Europe, China and Japan – a total group of 48 people representing:

• American Academy of Allergy Asthma and Immunology (AAAAI)
• Asiatic Group
• African Society for Immune Deficiency (ASID)
• European Federation of Immunological Societies (EFIS)
• European Society for Immune Deficiencies (ESID)
• Federal Drug Agency (FDA)
• International Nurses Group for Immune Deficiency (INGID)
• International Protein Fractionators Association (IPFA)
• IPOPI Board
• International Union of Immunological Societies (IUIS)
• Jeffrey Modell Foundation (JMF)
• J-Project Group
• Latin American Society for Immune Deficiency (LASID)
• Plasma Protein Therapeutics Association (PPTA)
• World Health Organisation (WHO)

On the Friday evening we assembled over pre-dinner drinks and were welcomed by Mrs Drabwell, the Chair of IPOPI. After dinner Professor Helen Chapel outlined the purposes of the weekend – the principle purposes being that of understanding what we were doing to achieve an improved quality of life for people with primary immunodeficiencies through improved diagnosis and treatment; concerns we share in common and areas where we can work together to achieve bigger, brighter and better things for our community.

Saturday was a day of presentations, discussions and, finally, some very stimulating work in breakout groups. The day got off to a good start when we all enjoyed a short video clip from Morocco, showing the ‘Dance Hajar’ – a short ballet demonstrating the anxiety of a sick child and the relief of diagnosis and treatment – which ‘set the scene’ for the day. A weary group met later for dinner – mindful of the fact that we started very early on Sunday morning so that we could round off the weekend’s proceedings before10 am so that those travelling long distancescould get back to their desks for Monday morning! We enjoyed two entertaining after dinner speeches from Bob Perry and Charles Waller, representing the two industry umbrella groups.

The outcomes document, as it stands right now, appears at the end of this item. A great deal was achieved and none of it would have been possible without the great willingness to work collaboratively as expressed by our speakers – Marcia Boyle, Gaby Strotman, Roberta Pena, Fred Modell, Dr Ana Padilla, Professor Helen Chapel, Dr Mark Ballow, Dr Klaus Warnatz, Dr Jackie Kerr; Dr Basil Golding; Professor Laszlo Marodi; Dr Monika Esser; Dr Jose Franco; Professor Shigeaki Nonoyama; Professor Surjit Singh; Professor Isil Barlan; Professor Xiao-Ming Gao, Sven Fandrup, Vicki Modell, Martine Pergent, Bob Perry, Charles Waller – and of course the IPOPI staff.

In our day to day work at IPOPI we already feel the advantages of having met and shared experiences and values. We look forward to reviewing this in 2011. In the meantime, here are the main findings of our weekend together...

Agreed Statements and Actions

1. DIAGNOSIS – 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.

• Develop Guidelines to enable news of diagnosis to be delivered sensitively and appropriately with due regard to cultural circumstances
• Develop Guidelines for adult care and diagnosis on the same basis as paediatric care and diagnosis
• Develop Diagnostic Guidelines for cost effective front line testing for PID in emerging countries

2. TREATMENT – 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.

• Campaign to ensure that effective treatments are made available as a basic human right
• Obtain a global consensus on viable Ig in emerging countries (refer to the process for distribution of vaccines in emerging countries)
• Track the progress of ICD11 (International Classification of Diseases due to publish in 2011) and work with the expert Committee at IUIS as necessary
• Encourage the work of the WHO Achilles Project
• Campaign globally for adult care and diagnosis on the same basis as paediatric care and diagnosis

3. COMMUNICATION – Proposed Champions: Sven Fandrup, Joy Rosario & 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.

• Consider the use of language and the use of prevalence statistics
• Consider the instigation of a worldwide Primary Immunodeficiency Awareness Day (or Week)
• Be aware of all awareness raising campaigns and launch them simultaneously when possible
• Campaign globally for adult care and diagnosis on the same basis as paediatric care and diagnosis

4. DATA COLLECTION – Proposed Champions: Marcia Boyle, Stephen Baxter & 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.

• Collect data from various countries regarding the efficacy of treatment and the health and experience of patients, as well as the ability to collect plasma for fractionation
• Provide the data to convince payers of the appropriateness of treatment

5. GUIDELINES – Proposed Champions: Professor Helen Chapel & 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.

• Develop Guidelines to enable news of diagnosis to be delivered sensitively and appropriately with due regard to cultural circumstances
• Develop Guidelines on the infrastructure for the transition from paediatric to adult treatment
• Obtain global support for the development of a Guideline for the use and prioritization of Ig

6. SUMMARY:

• 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.