Evaluation of a national resource to identify and study rare diseases: The Australian Paediatric Surveillance Unit

Aims:   To evaluate the Australian Paediatric Surveillance Unit (APSU).
Methods:   We used criteria recommended by the Centres for Disease Control and Prevention (CDC) for evaluating surveillance systems and reviewed productivity, response rates, completeness of the mailing list and impacts of APSU studies. Anonymous evaluation questionnaires were sent to 1260 reporting clinicians, 42 researchers and 86 public health professionals to seek their feedback as users of the APSU.
Results:   APSU provides national epidemiological and clinical data about rare childhood conditions that inform public health policy and clinical practice. Between 2000 and 2007, APSU data were disseminated in 106 journal articles, 207 scientific presentations and 85 media items. Of paediatricians and paediatric sub-specialists actively practicing in Australia and listed as Fellows of the Royal Australasian College of Physicians, 92% participate in APSU surveillance. An average 96% of monthly report cards were returned per annum since 2000. Sensitivity of case ascertainment was difficult to calculate for many conditions because alternative sources of ascertainment do not exist. Evaluation questionnaires were returned by 818 (65%) reporting clinicians, who believe APSU is valuable for generating knowledge 665 (81%), identifying research priorities 635 (78%), guiding clinical practice 572 (70%) and informing public health policy 575 (70%). Similar responses were received from researchers and public health professionals.
Conclusions:   The APSU fulfils its objectives and meets relevant CDC criteria for usefulness, simplicity, acceptability and representativeness, sensitivity and timeliness of data quality. However, stability is threatened by lack of continuing core funding. APSU is highly productive and valued by child health clinicians, researchers and public health professionals.