Background

One of the most important areas in drug development is the design of experiments. The better these are, the more efficient the trials. Such efficiencies lead to fewer patients, shorter timeframes and reduced costs. There is therefore a need for more seamless and complex adaptive designs in drug development and mathematical and statistical methodologies and models are key to such improvements.

A major EU funded programme - Innovative Medicine Initiative 2 (IMI2), seeks to improve health by speeding up the development of, and patient access to, innovative medicines. A key component of its research strategy is "Innovative clinical trials paradigms" and this workshop aimed to help facilitate the articulation of clear objectives and opportunities for partnering between industry, academic and regulatory bodies in order to achieve this.

Aims and Objectives

This Open for Business event was part of an Isaac Newton Institute research workshop which brought together leading expertise in the multiple disciplines involved in the design and analysis of experiments in healthcare, which was a follow-on to a Programme held at the INI in 2011. The event was structured to enable the formation of new public-private partnerships, collaborative research and to assist in identifying the common challenges that have greatest potential for research, knowledge transfer, public policy and commercial impact.

A key aim of the workshop was to highlight state-of-the-art design of experiments methodologies and how these can impact the wider societal health objectives, such as those of IMI2. The programme focused on:

• Innovative methodologies to evaluate treatment effect: use and development of appropriate methodology for patient population stratification and treatment prediction, explore potential biomarkers predictive of treatment effects or validate proposed candidate markers
• Adoption of innovative clinical trial designs: build the adaptive designs on the trial, program and portfolio level and needed statistical methodology.

These adaptive designs have a great potential in delivering substantially higher value, especially in dose-ranging studies through improving late stage dose selection and increasing the probability of success. The more complex adaptive designs like population finder, indication finder, and platform trial design, were also discussed along with their potential benefits as well as the new operational challenges in their implementation. Such designs also allow for better assessments of outcomes in patient populations, whilst balancing the efficacy/safety needs of regulators and helping mitigate risks/costs for industry.

This event was of interest to individuals from a number of areas including relevant research establishments, life sciences, the pharmaceutical and biotech companies, innovative small and medium-sized enterprises (SMEs), as well as the Regulatory Authorities and Health Technology Assessment Agencies.

Venue

The workshop took place at the Isaac Newton Institute for Mathematical Sciences in Cambridge. Please see the Isaac Newton Institute A-Z for further information about the venue.