Development of a Minimum Dataset for the Monitoring of Recombinant Human Growth Hormone Therapy in Children with Growth Hormone Deficiency: A GloBE-Reg Initiative.

A total of 246 data items were initially compiled for consideration, with 27 removed due to redundancies, leaving 219 items subjected to the grading system.

• Each member graded items on (1) importance of the data field and (2) ease of data collection.
• Data fields achieving 70% consensus in terms of importance qualified for the minimum dataset (MDS), provided fewer than 50% deemed the item difficult to collect.
• The 219 items were compiled from comprehensive lists routinely collected by each clinical and industry expert for children with growth hormone deficiency.

Of the 219 items assessed, 111 achieved at least 70% consensus as important data to collect when monitoring children with GHD on rhGH treatment.

• 69 of the 219 items were deemed easy to collect.
• Combining the criteria of importance and ease of data collection, 63 items met the criteria for the MDS.
• Several anomalies to the MDS rule were identified and highlighted for discussion, including clinical trial participation, HbA1c monitoring, other past medical history, and adherence.

The final MDS recommendation comprised 43 items after discussion of identified anomalies reduced the initial 63-item list.

• 20 items are to be completed once (at baseline).
• 14 items are to be completed every 6 months.
• 9 items are to be completed every 12 months.
• Anomalies considered for exclusion or special handling included current or previous clinical trial participation, HbA1c monitoring, other past medical history, and treatment adherence.

The study was developed through the GloBE-Reg initiative, a new project that has developed a common registry platform to support long-term safety and effectiveness studies of drugs.

• Twelve clinical experts from 7 international endocrine organisations were identified by the GloBE-Reg Steering Committee.
• 2 patient representatives and representatives from 2 pharmaceutical companies with previous GH registry expertise also collaborated.
• The GH scientific study group within GloBE-Reg undertook this study.

The MDS is intended to apply to both current daily rhGH preparations and newer long-acting growth hormone formulations.

• The objective was to identify the minimum dataset that could be measured in a routine clinical setting across the world.
• The exercise aimed to minimise burden on clinicians and improve quality of data collection.
• The MDS is designed for monitoring safety and effectiveness of rhGH in children with GHD through real-world data.