Scientific due diligence: A handbook for investigators and investors

Many a blockbuster drug began in the mind of a single entrepreneur. From start-ups to spin-offs, there are no lack of options for a company or venture capitalist to license or invest into. But how can you know that this is the correct investment? The correct option to choose? The one which will lead to financial success and a nice end-of-year bonus? This is where scientific due diligence comes in, the independent, realistic and critical review of a potential technology. From early development data through to clinical study results, patents to competitor analysis, the due diligence process is essential for any investment decision. We have developed this handbook to guide investors and due diligence investigators through the minefield of scientific due diligence in the pharmaceutical world. It covers best-practice approaches, traps to avoid, and the most important areas to focus your limited time on. Investing in pharma? Then this is the book for you. --- Book contents --- The book has been divided into sections which cover the entire due diligence process. The first section covers the basics of due diligence: - Chapter 1 introduces due diligence investigations, including the attributes of good due diligence investigators, the basic rules to follow, and commonly-seen licensing approaches. - Chapter 2 covers the initial steps of assessment, including the preliminary screening for potential licenses and the secondary screen to identify true opportunities. - Chapter 3 shows the preparation for the on-site scientific due diligence investigation, including typical organisational tasks and team set-up. Next, the specific requirements for each area of expertise are covered in more depth: - Chapter 4 covers the investigation from the regulatory affairs perspective, including factors such as approval risk, regulatory planning, and useful special pathways. - Chapter 5 deals with quality, the assurance that the technology has been developed and manufactured to the required quality levels. The chapter covers typical GMP documents and important GxP requirements which will need to be verified. - Chapter 6 covers chemistry, manufacturing and control, the details of the product and the production process. This includes manufacturing-site specific documents and the process development and validation requirements. - Chapter 7 describes preclinical trials, the preliminary work prior to human testing. This includes approaches for evaluating preclinical studies as well as more specific information for toxicology and pharmacology work. - Chapter 8 involves clinical trials, the most important test of any new drug. This section covers both general trial requirements as well as those specific to individual clinical phases. - Chapter 9 deals with marketing, the ability to sell the new product. This includes determining market position, analysing potential competitors, and determining reimbursement options. - Chapter 10 describes the intellectual property factors which may be involved, covering both patenting and data exclusivity approaches to IP protection. - Chapter 11 finishes the scientific due diligence process by providing the final set of questions to ask prior to making the final recommendation. Finally the five appendices provide reference information which will help when conducting a due diligence investigation, from example checklists to work from through to advice for when you are being audited.