International Pompe Association

Mr. Y.S. Poortman
Dutch Association Neuromuscular Diseases
The Netherlands

Introduction

Condition bound patient organisations (CBPO) are receiving growing recognition as an emerging partner for healthcare officials, scientists and industry.

Differences and possibilities of patient organisations

Limitations in time, energy and financial means of patient associations require efficiency and setting of priorities. There are several differences between the background conditions in which different national patient associations have to function: the socio-economic and political situation, healthcare provisions and moreover the geographical spreading of the patient population show great diversity. Due to these differences good communication is of vital importance. Electronic communication (E- mail, Internet) offers new possibilities.

Necessary ingredients

Clear, realistic, transparent, measurable and well agreed objectives are the necessary starting points for the realisation of these objectives. In addition, a clear mission, an efficient structure and a good business plan provide the basis of successful fundraising for the International Pompe Association. Of course, the responsibilities of the national and international association(s) have to be well agreed upon. Furthermore, the realisation of activities of CBPO needs the executive support of national associations.

Functions and contacts

Some examples of the many functions of a CBPO are social support, advocacy, information-provision, networking (gathering patients/families), stimulation and facilitation of research.

For researchers a CBPO could be of great value because it represents the contacts with patients who are highly motivated to participate in therapeutic trials. Moreover, industry will welcome the support of CBPO for means of marketing.

Affiliation to umbrella organisations on the continental level (EAMDA, EAGS, EURORDIS) and the global level (WANDA, IAGS, AGSD, WAOPBD, WHO) deserves consideration.

Conclusion

A CBPO will be for many families a haven, a wharf, a beacon and a lighthouse. The executive committee of a CBPO should pilot the ship of the association through storms and misfortune to the fulfilment of their aims.