Module 1 2021

30/06/2021

How to demonstrate significant benefit

MoA, and PK and/or PD profile

*Scope has been reduced with Commission notice on the application of Art 3,5 and 7 Reg 141/2000, published 2016. Source: Fregonese, L. et al. Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe, Drug Discov Today (2017)

The Organisation for Professionals in Regulatory Affairs

21

Example – Plenadren (hydrocortisone)

Product facts:

Hydrocortisone (cortisol)

•

modified-release tablet

•

treatment of adrenal insufficiency

•

History: 2006 - OD 2011 - MAA with OD status

• Showed significant benefit compared to immediate release tablets and synthetic glucocorticoids  “once-daily modified release formulation produces levels of active substance in the body which more closely mimic the natural release pattern of the hormone in healthy people compared with existing treatments. This offers a major contribution to patient care.”

2016 - UK challenged orphan designation • No change in the seriousness of the condition • Prevalence increased but still below threshold (4.85 people in 10,000)

• Still significant benefits for patients with adrenal insufficiency because based on clinical data its once-daily modified release formulation produces benefits in terms of body fat, control of blood sugar, and aspects of patients’ quality of life compared with existing treatments. This was considered a major contribution to patient care.

https://www.ema.europa.eu/en/documents/orphan-review/comp-assesses-whether-plenadren-still-meets-orphan-designation-criteria-recommendation- maintain_en.pdf

The Organisation for Professionals in Regulatory Affairs

22

11