Improving Design, Evaluation and Analysis of early drug development Studies

Project Information

IDEAS

Grant agreement ID: 633567

DOI

10.3030/633567

Project closed

EC signature date 16 December 2014

Start date 1 January 2015

End date 31 December 2018

Funded under

• EXCELLENT SCIENCE - Marie Skłodowska-Curie Actions

Total cost

€ 3 108 939,48

EU contribution

€ 3 108 939,48

Coordinated by

UNIVERSITY OF LANCASTER
United Kingdom

Periodic Reporting for period 2 - IDEAS (Improving Design, Evaluation and Analysis of early drug development Studies)

Reporting period: 2017-01-01 to 2018-12-31

Summary of the context and overall objectives of the project

Background

Drug development is a long and costly process which suffers frequently failure that is often only determined during the final stage. It has been recognised that more care needs to be taken during the early stages of development to avoid lengthy and costly confirmatory studies with ineffective or harmful treatments. Furthermore, the number of new drugs approved has decreased drastically in the last 50 years. Early drug development is crucial to identify promising treatments that have the potential to transform patient's lives. To achieve this goal it is essential to implement efficient methods for the design and analysis of such early development studies. The expertise in this area is, however, limited and adequate methodology is only partially available. The main goals of the IDEAS network were to

(a) train early-stage researchers in state of the art methods for designing, evaluating and analysing early phase studies;
(b) develop novel methodology for early phase studies;
(c) provide an international, collaborative environment;
(d) raise awareness about cutting edge methods.

Scientific achievements

The network has successfully trained 14 early stage researchers in methods for early drug development with 11 having completed a doctoral degree and the remaining three on track to complete a doctorate in the next months. The research undertaken has led to novel methods for pre-clinical studies as well as early clinical trials. In the area of dose-finding, novel methods have been developed that allow pre-clinical data to be formally incorporated, methods for dose-schedule trials have been developed and novel approaches for dose-combination studies have been devised. Moreover, IDEAS has developed methodology to advance precision medicine. Novel designs accounting for subgroups in both Phase I and Phase II studies have been created, visualisation techniques explored and approaches to identify biomarkers developed. Additionally, novel statistical methods for biosimilar development have been proposed, advanced pharmacological models developed and estimation problems after adaptive designs have been explored.

Dissemination

The findings been published in high impact methods journals as well as applied journals and the novel methodology is already being started to be used in real studies which has been facilitated through the provision of open source software, training of end-users via bespoke publications and active engagement in studies. The network is now embarking on establishing a dedicated training route for quantitative drug developers.

Outreach

IDEAS has regularly engaged in outreach activities such as community days, a blog series and writing popular science articles. Additionally, the main research results were presented in short YouTube videos using animations and explaining the relevance of the methodological research in lay terms. To date, the videos have been viewed over 4,000 times. With over 200 different activities, we have reached over 66,000 people.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

A core focus of the activities has been to ensure the network develops to its full potential. Crucial to this has been the development of a sense of belonging to the network which has been achieved by formal team building activities, in-depth discussion sessions around commonalities and synergies between the individual researchers’ projects and regular communication between participants.

Scientific achievements

The early stage researchers (ESRs) have undertaken substantial research work and the research undertaken has led to novel methods for pre-clinical studies as well as early clinical trials and the findings been published in high impact methods journals more applied journals. Contributions to dose-finding (incorporation of preclinical data, dose-schedule trials, combination studies,...) precision medicine (biomarker identification, visualisation of subgroup analysis, …) and topics such as estimation after an adaptive trials and biosimilar development, have been made.
As of 31. April, 11 ESRs have successfully completed their respective PhD studies while the remaining ESRs are on track to obtain their PhD in the first half of 2019.

Training activities

A full training programme for the early stage researchers in the fundamentals of drug development with a focus on quantitative skills and training on transferable skills has been implemented and expanded on the basis of the expressed needs of the researchers. The training comprised of a combination of network-wide training and individual training activities. Four, week-long network-wide summer schools have been organised alongside of two shorter spring meetings and a three day Think-tank meeting. The objectives of these meetings were to train the researchers in methods for drug development, provide transferable skills training, foster networking/collaborations and share their research progress.

Dissemination

• 33 peer-reviewed papers accepted or published as per 28th February 2019 and numerous further publications are currently submitted or in preparation for publication.
• Results were presented by ESRs at numerous international conferences (> 150 talks and posters).
• Short YouTube videos to promote the main results
• Social Media including Twitter (with over 500 entries and >200 followers to the Ideas Account) and LinkedIn
• Participation an popular science events (such as EU researcher´s night or University for children)
• Joint workshops with Learned Socities and partner institutions and dedicated Ideas Sessions at Statistical Conferences
• A final IDEAS dissemination workshop with around 70 participants took place.
• The IDEAS website http://www.ideas-itn.eu/ is maintained and frequently updated.

Outreach

IDEAS has undertaken over 200 outreach activities reaching an estimated 66,000 people with activities ranging from participation in community days, writing a blog-series, non-technical summaries and articles in popular science magazines.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

The IDEAS network is impacting both the researchers’ career prospective and training in quantitative methods for drug development more broadly.

The extensive course program ensured cutting edge statistical expertise in designing, conducting, analysing and assessing innovative early phase trials. Statistical courses provided a well-founded basis in traditional topics relevant for early development to ensure that the graduates have a broad and in-depth competence on efficient methods considering the various aspects of early drug development. Through this training programme the researchers are uniquely trained for a career as research scientists in academia, the pharmaceutical industry, contract research organisations, statistical software developers, regulatory bodies and for entrepreneurs embarking on statistical start-up companies. The network provides all its researchers access to European key opinion leaders and stakeholders in the public and private sector. The researchers are therefore highly trained in the field and equipped with an invaluable network of experts which will make the researchers highly desirable future employees.

The IDEAS network showcases how dedicated training in quantitative methods for drug development can look like and illustrates the value of close collaboration of academic, industry and regulatory agencies which has the potential to transform training in the field for the future.

IDEAS Logo