Translational Biotechnology: From Intellectual Property to Licensing Product Development Path Classify your product – Drug, biologic, medical device Identify you primary claim/indication – Will guide your assessment of the product. E.g [Product X] is to diagnose, cure, mitigate, treat or prevent a certain disease or condition Determine your target market and understand regulatory requirements (e.g. local regulations and ICH, available regulatory pathways) Develop Regulatory strategy Knowing the regulations and content of relevant guidance documents, the disease or conditions being investigated and the approved therapies and their basis of approvals provides the foundation for your regulatory strategy Regulatory Strategy Document should be balanced, realistic and achievable to support the organization’s mission and vision Product Development Path Establish Product Development Plan – This translates the product requirements into actions. Plan is established collaboratively with input from different functional groups (covering manufacturing, quality, regulatory, non-clinical and clinical). It should include key milestones, critical paths and periodic reviews for “go and no-go” decisions and be updated periodically. Target Product Profile A Good TPP Provides Summary of product under development Products desired characteristics and features The studies that must be completed to demonstrate safety and efficacy The features that provide a competitive advantage TPP Continued Summary of a drug development program described in terms of drug labeling concepts and goals. Developed with the commercial goals of the product in the mind. Balance goals against the pharmacology of the drug and the practicalities of the clinical development program. TPP should be an optimized realistic view of the goals of drug development. Contains a synopsis of what will end up on the drug label, listed for each of three categories: the ideal product description (Optimal or “best-case”), a minimally acceptable product description (Minimal or “worst-case”), and a description that falls in between these best- and worst-case scenarios that will likely resemble the actual commercial product label after approval (Target or “likely-case”). The Optimal version should be the goal: what the sponsor hopes to claim on the final label, which will be used to guide the design, conduct, and analyses of clinical trials to provide maximum efficiency to the overall development program. TPP can and should be updated as the drug development program progresses and knowledge of the drug increases. Target Product Profile Benefits A Good TPP Helps address early issues in development and prevent late stage failures Serves as a good strategic planning tool to be used internally Serves as a good communication tool Will feed into your business plan Informs go/no-go decisions FDA Encourages use of TPP Draft Guidance Document produced in 2007 – ‘Target Product Profile- A Strategic Development Process Tool’, describes TPP as: • “Embodying the notion of beginning with the goal in mind. [As] labelling concepts are the goal of the drug development program... The ideal version of what the sponsor would like to claim in labelling guides the design, conduct, and analysis of clinical trials to maximise the efficiency of the development program” (5) •FDA recommends submitting TPP in the Briefing documents for formal meetings •May help contribute to more productive meetings and faster IND/NDA reviews and approvals TPP Guiding principles Key sections (Use MaRs Template on Blackboard): Indications and usage Dosage and administration Contraindications Adverse reactions Clinical pharmacology Non-clinical toxicology Clinical Studies ***Review the MaRs Emerging Life Sciences Entrepreneur Workbook: Defining your Target Product Profile - Therapeutics Highlighting special populations and incentives TPP should determine which special populations will need to be included study designs, i.e. paediatrics, geriatrics, the renally or hepatically impaired and woman of child-bearing potential Available options for accelerated development should be described Potential for incentives should be described i.e. additional exclusivity from paediatrics or orphan indications Cross-Functional Contribution is Key Creation of target claims requires cross-functional input Regulatory Affairs, Commercial, Clinical, etc. Enhances communication and collaboration to drive program forward Ensures an understanding of key success factors and responsibilities for each stakeholder Sample TPP https://www.ninds.nih.gov/Funding/Apply-Funding/Application-SupportLibrary/CREATE-Bio-Example-Target-Product-Profile-TPP Leveraging Precedent Understand competitive environment and ongoing development https://clinicaltrials.gov Critical to understand what has been done and the opportunities and risks based on precedent FDA Summary Basis for Approvals https://www.fda.gov
