Module 6

02/10/2024

Improved Convenience and New Dosage Forms Examples: extending the treatment and improved or alternative formulation Reformulation: • Valproate Epilepsy Tablets originally modified to oral solution/syrup; appropriate for paediatrics • Racemic mixture to single enantiomer Celexa vs Lexapro (Major Depressive Disorder) • Standard release to quick release; Parodel, Cycloset (Type II Diabetes Mellitus ) New dosage strengths: • Gilenya paediatrics: a lower dosage strength to treat age-appropriate paediatrics • Elocta: additional higher dosage strengths, weight-based formulations to provide further flexibility for patients • Viagra for erectile dysfunction 50-100 mg tablets, reformulated (and re-branded) as Revatio 20 mg lower dose effective to treat peripheral arterial hypertension (PAH). New combination products: • Symbyax treatment of BP disorder: combined with Zyprexa (schizophrenia) + Prozac (depression) • GSK’s HIV products: Retrovir, Epivir, and Ziagen individual products, re-packaged as Combivir and Trizivir

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 2 – Maintain and Extend the Brand

59

Paediatric Regulation (EC) No.1901/2006 Enforces industry to develop better medicines for the paediatric population Paediatric Investigational Plan (PIP): • A regulatory requirement to develop and submit a clinical study development program for all subsets of the paediatric population from birth to <18 years of age or request a partial or complete waiver. PIP should consist of clear clinical study designs, with target indication(s), treatment, statistical plan and timelines , which needs to be agreed by EMA Paediatric Committee. • Under the obligations of the paediatric regulation, companies are required to submit the PIP after adult PK studies are completed . However, in some cases can be submitted later upon agreement with the EMA Paediatric Committee. • Throughout the life-cycle of the product the PIP can be modified due to changes in the paediatric clinical study designs. • Regulatory obligation to submit results compliant with agreed PIP OR submit the granted waiver/deferral, at time of MAA or line extension . • Compliance check required to be submitted (partial or complete) prior to submitting line extensions or new indications. • Another obligation is Article 46 ; submission of paediatric study results within 6 months of study completion. • Incentives /rewards to the company : 6 months patent extension. PUMA: 8+2+1 data protection rule. Paediatric orphan drug: extra 2 years market exclusivity.

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 2 – Maintain and Extend the Brand

60

30