Module 6

02/10/2024

Target Product Profile Define and shape the brand

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 1 – Develop and Shape the Brand

15

EXAMPLE: Creating a Target Product Profile

TPP Handout

PRODUCT ATTRIBUTES

TARGET CHARACTERISTICS

Product Description / Type Mechanism of Action

Type of agent / molecule, e.g. ATMP, biological drug, small molecule, monoclonal antibody; first in class; The target the treatment is believed to act upon; how does the drug molecule exert its effects on the target Intended clinical need which the product will address. E.g. Treatment for..; prevention of..; disease; condition; specific severity with specific symptoms; relief of symptoms; first-line or adjunctive therapy. Adult; paediatric; specific age; subpopulation target (e.g. positive identification of genetic markers; antigen or antibodies; patients who failed on other treatments) Dosage form, regimen and level required for therapeutic effect (dictated by pharmacology and potency); Recommended administration route e.g. oral, IV, SC, inhaled (dictated by bioavailability and stability data); Primary and secondary endpoints for phase III clinical study; including variables (e.g. disease state) and specific measures, e.g. all-cause mortality, or cardiac function measured; validation of endpoints

Indication and Usage Unmet Medical Need

Patient Population

Dosage Form, Strength and Administration Route

Clinical Efficacy: Primary and Secondary Endpoints

Clinical Assessment of Success

Percentage of improvement in primary endpoint (specify absolute or relative to baseline) compared to reference or control group required for marketing success; study design options; treatment duration / frequency

Contraindications

Situation(s) in which the drug should not be used because the known risk outweighs any possible benefits; known hazards from clinical studies (not theoretical possibilities) Potency of product; specific values, e.g. NOEL and NOAEL (extrapolated from preclinical testing data); Data from studies; any advantages over other existing treatments; identified risks; special care to be exercised by practitioner for safe and effective use Any serious drug-related adverse reactions, reactions anticipated from preclinical studies; reactions dictated from experience from clinical studies

Safety Profile Warnings and Precautions

Pertinent Adverse Reactions

Storage and Handling

Storage requirements e.g. temperature, refrigeration, freeze; any specific instructions. Drug product preparation before administration; reconstitution requirements

Regulatory Pathway

Regulatory considerations; data package content; orphan status; PIP requirements; engagement with regulatory agencies; use of any early access tools; intended MAA filing strategy; concessions for any eventual approval; post approval concessions Any involvement of partners for development or marketing; market jurisdictions; intended market exclusivity targets; patent protection status; cost of treatment to bring to market; market size

IP and commercial development considerations

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 1 – Develop and Shape the Brand

16

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