The purpose of this assignment is to research the CFTR chaperone lumacaftor and the CFTR potentiator ivacaftor as case studies of drug development. You will then work individually to produce a one-page information sheet designed to inform either people with CF, or policymakers, about these drugs. You will need to consider the science behind these molecules and their discovery, their clinical impact, the policy environment around their translation into clinical use, and the role of patient groups in affecting policy.
Learning outcome: • To help achieve an understanding of how changes in host biology can alter defences against infection.
• To understand the mechanisms of action of key drugs.
• To be aware of a variety of synthetic and natural products currently being researched / developed for use in infection prevention and treatment, and the scientific, financial and ethical considerations involved in product R&D.
• To reflect on the methods and styles of communication required to successfully interact with different stakeholders in pharmaceutical development (scientists, policymakers and patients).
This assignment will be graded on your ability to research, synthesise and communicate information about how two examples of recently-developed drugs work, and their impact on a specific patient group. You will need to take into account the priorities of your chosen audience (patient groups or polymakers), the key information they will be looking for (economic, social, health etc) and communicate in an appropriate professional register. Marks will also be given for the visual layout (graph, tables) and design of your information sheet, in so far as it aids clear communication.