Innovations in Clinical Trial Designs for a New Drug Development
Dr Md Anawar Hossain
A new drug development and delivering to the patients include a lot of research and many steps including R&D, clinical trials and regulatory approval, which spend millions to billions of dollars. Even after approval, it needs further expenses for market feedback and studies. Sometimes, some medicines do not get regulatory approval even after clinical trials and spending a lot of money. The clinical trials cost a large amount of money out of the total cost for a new drug development. Therefore, the success of a drug development and financial cost depend on designing an effective and careful clinical trial program. As for example, Amgen is making adaptive trial designs for clinical research, which can be less expensive, but faster and more successful clinical trials (Amgen Science, 2018). They said that human genetics could be exploited to pinpoint drug targets and develop a new medicine. Innovative and promising strategy is to be designed which can bring success in clinical trials.
Conventional versus flexible clinical designs
The protocol of a study is pre-set in conventional clinical trials. The criteria for the patients, the doses tested, the outcomes measured, and the study’s duration are pre-planned, whilst these criteria are examined in clinical trials without any change. Amgen suggested to move from fixed to flexible study designs. Their adaptive clinical trials are newer methodologies and far more flexible.
Adaptive clinical trial designs
In adaptive clinical trial designs, the protocol can be changed or modified based on the preliminary data or results coming from study. The following criteria can be changed for adaptions to the clinical trials: dropping or adding doses, increasing the size or duration of a trial, and enriching the study population. The doses, that aren’t effective, differentiated, or safe, should be changed and we need to optimise the dose level which can benefit the patients. If we are not obtaining the anticipated results, we need to increase the size or duration of a trial, which gives the test drug a better opportunity to demonstrate its true impact. The types and number of the study patients can be increased to obtain the more reliable and variable response from them.
Adaptive designs permit for course corrections by incorporating a mid-course assessment of a clinical trial’s performance, which will ultimately provide success, and reduce the time and cost of a drug development. Based on the preliminary results, we should understand a drug or a dose of a drug either works or doesn’t work and then we should quickly decide whether we will further process or not with a clinical trial of a drug. This will reduce the cost and time of a drug development.
Complex Innovative Trial Designs (CID)
The U.S. Food & Drug Administration (FDA) and Drug Information Association (DIA) together organized a conference to provide the global experts, health authorities, clinicians, patient advocates, drug development innovators, regulatory scientists and reviewers with opportunities to share their knowledge and experiences on clinical trial designs, especially Complex Innovative Trial Designs (CID). The conference themes included CID master protocols, complex adaptive design, Bayesian techniques, and also the potential of alternative data sources (DIA/FDA, 2020). Complex Innovative Trial Designs (CID) have the potential to increase the efficiency and lower the cost of drug development which will accelerate patient access to life-altering therapies. The conference explored the suitability of each CID topic and the proposed innovations, their usefulness from a patient perspective, and solutions to the challenges in the clinical trial designs. One of the main objectives of this conference was to promote CID concept. Beckman et al. (2022) published an article which also highlighted the designs and approaches based on the meeting linked to an ongoing FDA pilot program in the field.
References
Amgen Science, A Strategy for Making Clinical Trials More Successful. Science & Innovation, 10/19/2018.
Beckman RA, Natanegara F, Singh P, Cooner F, Antonijevic Z, Liu Y, Mayer C, Price K, Tang R, Xia A, Apostolaros M, Granville C, Michaels D. Advancing innovative clinical trials to efficiently deliver medicines to patients. Nat Rev Drug Discov. 2022 Aug; 21(8):543-544.
DIA/FDA, 2020. Advancing Complex Innovative Clinical Trial Designs to Efficiently Deliver Medicines to Patients, Meeting, March 2-3, 2020, Silver Spring, Maryland, USA.