Module 6

02/10/2024

Reach More Patients and Extend the Clinical Indication Maximise the potential of the approved new medicinal product

Treat more patients • Completely new indication* e.g. RA, MS, CD • New population; paediatrics, elderly • Different “stages” of the disease e.g. Relapsing -remitting MS, Primary-progressive MS, mod-severe

Treat earlier • Treatment line of clinical use e.g. 2nd to 1st

Improved or new therapy • Monotherapy or combination therapy, • e.g. use with SoC i.e. anti-TNF + methotrexate in RA • New target therapy: e.g. Xalkori NSCLC, ALK+ve to ROS1+ve Maximise treatment effects • Treatment will make a difference to patients • Sub-populations (e.g. new age ranges, neonatal) • Optimise B/R considerations • Support effectiveness (HTA, pricing &reimbursement)

How is the licence extended? • Need supportive data: new direct clinical study data evaluating efficacy and safety, extrapolation (PK, PD, popPK), literature review • Submission of Type II complex variation (90 day agency review for changes to or addition of a new indication)

Extend Intellectual Protection (IP) • e.g. extra time, + 1 year EU Regulatory Data Protection to avoid

generic applications from being filed • Added “significant benefit” if orphan

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 2 – Maintain and Extend the Brand

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Alzheimer’s Disease Targeting different clinical stages of the disease spectrum over time

Dementia stages

Pre-dementia stage

MCI due to AD

Pre-Clinical

Mild

Moderate

Severe

• Clinically treat different stages of the Alzheimer’s disease spectrum • Presentation of the diseases changes over time • Early onset predementia stage, measured using the ‘Alzheimer’s Disease assessment scale’ (ADAS). • Mild cognitive impairment (MCI), mild to moderate to severe disease measured by using the ‘clinical dementia rating’ (CDR) • Magnitude of the biomarker increases as the disease worsens.

Source: Jack, et al. Lancet Neurol (2013)

The Organisation for Professionals in Regulatory Affairs

Mild cognitive impairment (MCI) due to AD = Prodromal AD, A β = Beta-amyloid

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