Module 6

02/10/2024

TPP Handout

Target Product Profile Create early in the development program

A summary of the development : •

TPP provides a summary of the development of a medicinal product in terms of realistic labelling and commercial target concepts and goals (best case and worst case scenarios). A dynamic document, which gets updated as new information unfolds. • Aim is to have one global TPP, but not always possible with divergent regionally health authority opinions.

Ultimate objective: • To develop a drug for an unmet medical need, which physicians want to prescribe, patients want to take, regulators will approve and payors will value. Gain external knowledge, multi-dimensional: • Advisory boards and key opinion leaders • Clinical considerations e.g. review competitors (now and future predictions), key issues in real life setting e.g. managing an emerging safety profile, review of key pivotal clinical data • Commercial considerations: key claims for launch e.g. differentiating factors – what must be achieved to make the drug marketable; Improved efficacy, safety, tolerability…a new treatment option, superior claims, betters gold standard. • Regulatory precedents; clinical guidelines, endpoints, comparators, study designs, learnings from competitors (EPARs).

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 1 – Develop and Shape the Brand

17

Target Product Profile Create early in the development program

Regulatory considerations •

Input into phase III study designs

• Monitor link between TPP, core data sheet and study data • Regulatory filing pathway to approval • Input prior to Scientific Advice

Clinical considerations • Meet the requirements of Regulators, HTABs or both • Types of study / evidence: RCTs, pragmatic study designs, biomarker / surrogate measures, adaptive trial design, indirect comparisons and meta-analyses, observational studies. • Endpoints: Regulators vs HTABs preferred (e.g. clinical endpoint vs QoL, PROs). • Comparators: choice of appropriate comparative (if applicable); head to head, demonstrate absolute or relative efficacy or both. • Analysis: non-inferiority or superiority. Novel statistical analysis models. Extrapolation from adults to paediatrics • Post-approval studies: PAES, PASS - Regulators and HTABs requirements

Proof of Concept (PoC) versus Proof of Value (PoV )

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 1 – Develop and Shape the Brand

18

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