A global client wished to develop a Target Product Profile (TPP) for a range of rare disease syndromes with a novel product and unprecedented mechanism of action. We were part of the UK-based team tasked with accelerating the development and securing the initial scientific advice meeting with the EMA and subsequent PRIME designation. We undertook a detailed review of the existing product information and provided gap analysis and guidance on critical aspects essential for EU clinical investigations. In particular, we were also able to maximize the use of published data and demonstrate its applicability to the novel mechanism of action and the selected sub-populations. We focused on the conflicting requirements of stakeholders and arrived at a balanced strategy that was right for the company, the customers and the patients. This enabled earlier engagement with HTA providers within the EU. In addition, we worked with senior managers to produce an overall project plan which detailed preclinical, clinical, CMC, regulatory and commercial activities for the durationof the project. The plan also included resource & budget planning specifications.

We assisted in the Phase 2 study design for a UK-based SME which had invested significant resources in new product clinical development. Our aim was to maximize the application of existing data to ensure a streamlined development process whilst complying with regulatory expectations. The company had sought FDA and EMA advice and needed interpretation assistance together with the choice of study endpoint. Conflicting recommendations had introduced challenges for the company in making a decision. We analysed the data sources and worked with lead patient groups adopting a phased approach. We proposed risk mitigations to optimize early development initiatives. Our suggestion of a translational medicine study to gather additional data on existing biomarkers was undertaken. We worked with patients to design a small-scale non-interventional study aimed at exploring additional endpoints meaningful to patients and which complemented the existing regulatory requirements. The successful completion of the preliminary programme built confidence for progression to a larger study. Time and cost-effectiveness were achieved by using an adaptive design that combined Phase 2a (proof-of-concept) and Phase 2b (dose-ranging) which reduced start-up times and dose response estimates.

We provided a detailed assessment of the data produced by a novel compound used to treat neurological disorders. As the product was under US development, it was important to understand how the data would be assessed in Europe and to develop a strategy for initiating studies in the EU. We provided critical assessment together with next step recommendations to optimize development in the EU. We prepared procedural Scientific Advice documentation for the EMA and project managed through to successful completion enabling the company to implement future EU-based programs

We assisted a company in developing a clinical strategy for combining its digital health product with a third party medicine. The combination product was to be developed in the US. Following our thorough product analysis on its intended use, we reviewed competitor data which revealed the existence of a predicate device. We initiated a filing through a 510 (k) pathway. Our clinical strategy included utilisation of an umbrella study approach, saving time by combining a sequential series of studies into a single protocol. Our approach offered a competitive advantage leading to a product license application which was both cost and time-effective.