Recombinant human growth hormone (rhGH) was introduced in 1985, replacing the previous, pituitary-derived option in many countries when the risk of contamination with the CJD prion was identified.
At that time, specialists desired a patient registry to assess the safety and efficacy of the new treatment provided to patients in Europe and many other parts of the world by the Swedish pharmaceutical company KabiVitrum. The original, modest ambition was to follow 500 patients for five years. During that period, the value of a comprehensive observational survey was noted and the Kabi International Growth Study, KIGS, continued beyond five years to become the largest database of children treatment with rhGH, having enrolled over 83,000 patients and the cohort followed for over 322,000 patient years.
KIGS has now been concluded. A final report on patient outcomes has now been published (https://doi.org/10.1210/clinem/dgac517). Based on the unrivalled dataset within KIGS, the authors of the report conclude that rhGH is safe and effective in children with GH deficiency and also in many other short stature conditions.
Within the KIGS software system, clinicians had access to a valuable patient management tool – KGS – including electronic growth charts. Jani Soderhall, developer of the software, comments: ’my motivation then and subsequently when I established PC PAL, was to provide paediatric specialists not simply with a convenient way enter data into a registry, but to give them an effective patient management tool’.
PC PAL further developed and maintained KGS over a period of 15 years and it set a new standard for patient management systems. Features included instant calculations for growth monitoring based upon an extensive reference library, with growth charts from around the world made available to the KIGS investigators. The KIGS patient registry inspired many other GH companies to develop registries. Following the EU Orphan legislation of 1999, patient registries became the norm for following the real-world use of new drugs for rare diseases.