MODULE 7: TARGET PRODUCT PROFILES

1 MODULE 7: TARGET PRODUCT . PROFILES . DNDi/Scholars & Gentlemen 126. 1. INTRODUCTION. TARGET PRODUCT PROFILES are key strategic The global community, including WHO, UNAIDS, documents used to communicate summary the Global Fund to Fight AIDS, Tuberculosis requirements for new products that fulfil a and Malaria (Global Fund), the united States priority need. The purpose of TARGET PRODUCT President's Emergency Plan for AIDS Relief PROFILES is to guide industry during the drug (PEPFAR) and the united Nations Children's Fund development process and serve as a planning (UNICEF), have a responsibility to define the tool that can facilitate discussions with requirements around paediatric medicines and regulatory agencies and be updated as new have clear, transparent communication to industry information becomes available.

2 On the products that are required to meet the unique needs of children. The importance of TARGET PRODUCT PROFILES resides in their role in identifying the critical attributes of Some organizations, such as WHO, UNICEF and a PRODUCT before development begins, to ensure the Drugs for Neglected Diseases initiative, have that the final PRODUCT is adapted and responds developed TARGET PRODUCT PROFILES for specific to the needs of the end-users (Fig. ). TARGET desired products such as medicines, diagnostics PRODUCT PROFILES can help address issues early in and vaccines that have served to guide industry the PRODUCT development process and prevent in their own PRODUCT development The late-stage development failures.

3 TARGET PRODUCT profile describes how the end- user will use the PRODUCT and is based on such Fig. A TARGET PRODUCT profile as a strategic attributes as indications, targeted population, planning tool clinical efficacy, safety and tolerability, stability, route of administration, dosing frequency and cost, along with development timelines. Research and Table outlines various properties of TARGET development PRODUCT PROFILES and the optimum or ideal characteristics and minimum characteristics. Key properties include the ability to use the PRODUCT across the age spectrum of children and adolescents, ease of administration, heat stability, TARGET Regulatory PRODUCT Market palatability and swallowability and acceptable advice research production costs.

4 Profile Manufacturers and suppliers 2. ; ; pdf;jsessionid=6D9A30 EDDBCC978978CF9928 FAB921AE?sequence=1. 127. Table Properties of TARGET PRODUCT PROFILES Property Optimum or ideal TARGET PRODUCT profile Minimum TARGET PRODUCT profile TARGET One dosage form for ages 0 6 years Ages 0 2, 2 6 and >6 years population >6 years: adult Safety, Excipients selected from already used excipients in the Excipients selected in tolerability new drug application or abbreviated new drug application accordance with regulatory and in accordance with the Inactive Ingredients Guide of guidelines on inactive the united States Food and Drug Administration ingredients Limited use of excipients.

5 Minimum toxicity and drug interactions Drug attributes Accommodates a wide range of doses and drug A set of 3 5 technologies that properties (such as solubility) accommodate 80% or a majority Durability high barrier to resistance of drug types and doses and fixed-dose combinations Weight based Possible to administer the same dosage form across Possible to administer the same dosing multiple weight bands dosage form across multiple 1 formulation for children age <6 years; weight bands 1 formulation for age >6 years: adult (or half a dose).

6 Administration Easy to administer minimum manipulation by the Solid oral dosage forms considerations caregiver preferred Minimal opportunity for child to reject medication If bottle pack, then it should Easy to apply with no irritation (non-oral) have a child-resistant cap Fixed-dose combination, dispersible or small tablet size Administration PRODUCT does not need device or appropriate device Minimum instructions necessary device supplied if needed to use device if needed consideration Intuitive no use instructions necessary Taste and texture Palatable, child-friendly flavour, good mouth feel Palatable, acceptable taste and (oral dosage)

7 Mouth feel Manufacturing Accessible development and manufacturing capability in Low technology easy to low- and middle-income countries manufacture in resource-limited Robust and able to deliver medicines of adequate quality settings at an affordable price Feasibility for technology transfer Cost Acceptable cost to caregivers and funders Low-cost (total cost of goods and landed costs) options Preparation Should not require complex preparation by the end-user Easy to prepare and administer, before before administration such as with water, milk or food administration Include recommendations for extemporaneous Suitable for low-literacy settings compounding in the summary of PRODUCT characteristics Heat stable, Suitable for all climatic zones.

8 Including International Suitable for the supply chain and longer shelf life Council for Harmonisation Zone IVb (30 C and 75% end-user relative humidity) and 24 month total shelf life No special transport and See Annex 2, Stability conditions for WHO Member storage handling requirements States by region (5). or No special transport and storage handling requirements Easy to transport and store 128. Property Optimum or ideal TARGET PRODUCT profile Minimum TARGET PRODUCT profile Packaging Compact, light weight, easy to open and administer, Same inexpensive, easy and low cost to transport, sustainable packaging Disability For example.

9 Braille labelling or talking patient information Due consideration for end-user disabilities Regulatory Clear regulatory approval pathways considered up front Regulatory pathways in end- approval both in source and end-user countries user countries considered up front Patents Full geographical access, open licences, no data exclusivity Equitable geographical access Feasible to have PRODUCT monographs in official pharmacopoeia as soon as possible to produce generics Sources: Special Programme for Research and Training in Tropical Diseases (1), WHO Expert Committee on Specifications for Pharmaceutical Preparations: fifty-first report (2), TARGET PRODUCT profile paediatric HIV (3), Lopez et al.

10 (4) and Annex 2, Stability testing of active pharmaceutical ingredients and finished pharmaceutical products (5). Additional considerations in the drug formulation Table outlines the advantages and development process include TARGET candidate disadvantages of various formulations. Oral PROFILES and critical quality attributes. These include liquid preparations and oral solid preparations various drug characteristics that impact what are the most common formulations used for type of formulations can be manufactured and antiretroviral (ARV) drugs.