For orphan drug development, clinical trials can pose an insurmountable barrier to getting a product to market. This is especially true for substances looking to treat rare diseases for which comparatively little is know. In these instances, setting out clinical endpoints, objectives and biomarkers can be difficult, and if they do not take into account some as of yet unknown aspect of a condition, they can strangle the clinical development of a drug.
This is where adaptive clinical trials come into their own for orphan drug industry. By improving the flexibility of trials developers can maximise the efficacy of their treatment for patients, producing usful information, without invalidating the trial.
Using adaptive designs in clinical trials is a way forward in making the process more efficient in terms of saving costs and time. In this white paper, we discuss the different types of adaptive trials, the challenges in implementing these and the opportunities they open up for the IT industry.
Types of adaptive designs:
- Adaptive randomization design
- Group sequential design
- Flexible sample size re-estimation design
- Drop-the-losers design
- Adaptive dose finding design
- Biomarker-adaptive design
- Adaptive treatment-switching design
- Adaptive-hypotheses design
- Seamless adaptive trial design
- Multiple adaptive design
Advantages of adaptive designs
- They allow midway changes, decreasing the time of trial periods, leading to major cost and time savings.
- A trial can be stopped early if the accumulating data suggests that the drug is not efficacious or has a major safety concern.
- A trial can be stopped if the accumulating data suggests that based on the number of patients recruited, the efficacy of the drug can be statistically demonstrated.
- Some ineffective arms can be dropped early, the best dose can be identified early, and recruitment of patients in that arm can be carried forward by seamlessly integrating Phase II and Phase III trials.
- Adaptive trials help select the most promising option early and allow the correction of any wrong assumptions made at the beginning of a trial.
- Information from even outside the trial can be used in the interim analysis, which can have an impact on the trial.
Opportunities for IT
Adaptive clinical trials rely on the timely collection of data readily available for interim analysis. Systems for electronic data collection and data made available for analysis will be possible with the development of integrated technologies especially designed to support adaptive designs. Modeling and simulations (M&S) is going to be used more and more in predicting clinical supplies and trial results at the interim stages.
M&S is computationally intensive and needs a lot of research before implementation. Developing the M&S software and providing computational capabilities through cloud can be a major opportunity for the IT industry. The development of special statistical analysis software that can support a wide range of adaptations is also required.
TCS is a Platinum Sponsor at the ‘Disruptive Innovation in Clinical Trials’ and ‘Clinical Outsourcing & Partnering World’. To find out more about the conference, click here.