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The impact of real practice inappropriateness and devices’ inefficiency to variability in growth hormone consumption

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Abstract

Introduction

Growth hormone (GH) consumption is the object of a particular attention by regulatory bodies, due to its financial impact; nevertheless, GH treatment has been demonstrated to be cost-effective and is, therefore, usually reimbursed by public health service systems. In Italy, significant differences in GH consumption between regions have been recorded. Different appropriateness in real practice is a possible explanation, but the proportion of drug wasted due to different combinations of therapeutic regimes and types of devices used in drug administration is a complementary explanation. Aim of the study is, therefore, to determine how much of the variability in GH consumption is actually due to differences in clinical practice, and how much to waste.

Materials and methods

A model was settled to estimate the population with indication for GH administration, separately for children, transition subjects and adults, based on both the scientific evidence available and directly collected clinical evaluations. A systematic literature search was conducted using Cochrane Library (HTA and NHSEE) databases, Medline via Ovid, Econlit via Ovid, Embase.

Conclusion

The model applied to the Italian population showed that there was apparently unexplainable over-prescription and potential under-prescription in various regions, ranging from 20 to 40 % less than the estimated theoretical consumption to over 200 %. Wastage, at level of single device, could amount to as much as 15 % of the consumption, demonstrating that price per mg is not in general a good proxy of the cost per mg of therapy. Our estimates of the wastage shows a significant potential gap in the model assessment of the HTA bodies, as far as they do not explicitly take into account the issue of wastage and, consequently, the actual variability in local clinical practice.

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Notes

  1. SHOX has not been included in following analysis because till 2011, in Italy, GH wasn’t authorized for the specific indication.

  2. Adult onset: patients who have growth hormone deficiency, either alone or associated with multiple hormone deficiencies (hypopituitarism), as a result of pituitary disease, hypothalamic disease, surgery, radiation therapy, or trauma; childhood onset: patients who were growth hormone deficient during childhood as a result of congenital, genetic, acquired, or idiopathic causes.

  3. It’s worth noting that since 2006 a biosimilar GH formulation is available (EMEA/H/C/000607).

  4. To date, devices available differ in technical aspects (for example, they may or not have a needle) and in capacity; the amount of device-related waste is consequent to the actual “divisibility” of the capacity to the dosage, as well as to the possibility of using all the GH before it expires. No other potential sources of inefficiency, as malfunctioning, have been considered. More details in [10].

  5. In Italy public reimbursement rely on the predisposition of a therapeutic plan by Regional specialized centres.

Abbreviations

AIFA:

Agenzia Italiana del Farmaco (Italian Drugs Agency)

AOGHD:

Adult onset growth hormone deficiency

CRI:

Chronic renal insufficiency

GHD:

Growth hormone deficiency

HAS:

Haute Autorité de Santé (French National Health Authority)

Istat:

Italian National Institute of Statistics

NICE:

National Institute for Health and Care Excellence

OSMED:

Osservatorio nazionale sull’impiego dei Medicinali

PWS:

Prader–Willi syndrome

SHOX:

Short stature HOmeoboX containing

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Acknowledgments

This Project was supported by an unrestricted grant from Pfizer Italia s.r.l. Pfizer had no role in development of the project, data analysis, preparation of the manuscript, and decisions about submission.

Conflict of interest

The authors declare that they have no competing interest.

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Authors and Affiliations

  1. CREA Sanità (Consortium for Applied Economic Research in Health), University of Rome Tor Vergata, Via Columbia 2, 00133, Rome, Italy

    F. Spandonaro

  2. Unit of Pediatric Endocrinology, Children’s Hospital Bambino Gesù, Rome, Italy

    M. Cappa

  3. Division of Endocrinology and Metabolism, University and Azienda Ospedaliera Universitaria Integrata of Verona, Verona, Italy

    R. Castello

  4. Department of Pediatrics, University of Chieti, Chieti, Italy

    F. Chiarelli

  5. Division of Endocrinology, Diabetology and Metabolism, Department of Medical Sciences, University of Turin, Turin, Italy

    E. Ghigo

  6. CREA Sanità (Consortium for Applied Economic Research in Health), Rome, Italy

    L. Mancusi

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  1. F. Spandonaro
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  5. E. Ghigo
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Correspondence to F. Spandonaro.

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Spandonaro, F., Cappa, M., Castello, R. et al. The impact of real practice inappropriateness and devices’ inefficiency to variability in growth hormone consumption. J Endocrinol Invest 37, 979–990 (2014). https://doi.org/10.1007/s40618-014-0138-x

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  • DOI: https://doi.org/10.1007/s40618-014-0138-x

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