The pandemic has caused a lot of suffering in the last two years, but it has also brought science closer to society. Among the concepts that citizens have become familiar with is the clinical trial. It is the last step in the development of new drugs to demonstrate their efficacy and safety before authorization by regulatory agencies, and one of the most complex and expensive.
According to Farmaindustria (the National Business Association of the Pharmaceutical Industry), and under normal circumstances, the discovery of a new drug involves between 10 and 12 years of research and development, and an investment of around 2,400 million euros. This high figure is the result of adding the cost of research and development not only of each treatment that reaches the market but also of all those drugs that remain by the wayside. It is estimated that only 10% of candidate drugs that succeed in being tested in clinical trials receive the green light from health authorities. That is, only 1 in 10,000 compounds manages to become a new drug.
With the aim of optimizing clinical development times and reducing the cost of new drugs, the design of clinical trials is evolving from a fixed model to a more flexible one. The result of this new methodology is adaptive clinical trials in which it is possible to modify the protocol to integrate the knowledge acquired during the course of the trial.
“Clinical development is the most important category of R&D investment in the pharmaceutical industry. And the vast majority of the money invested in clinical trials goes to molecules that fail, ”says Miquel Balcells, Medical Director of the biotech pharmaceutical company Amgen in Spain. “For this reason, we need to increase the amount we invest in drugs that reach patients and decrease the amount we spend on those that do not get it,” he reflects.
Greater chances of success
In conventional clinical trials, a study protocol is firmly established before the first patient is recruited. However, the newer methodologies use adaptive clinical trials, which allow not only to accelerate drug development but also to increase the probability of success.
“The new adaptive design of clinical trials allows the study protocol to be modified to integrate and introduce the findings as soon as possible and accelerate the drug development process. In this way, it is easier to apply the results ‘live’ and refine the eligibility criteria more precisely, adjust the sample size and the proportion of randomization, as well as consider adding new treatments ”, explains Dr. Elena Garralda , researcher at the Early Clinical Development Group of Drugs of the Vall d’Hebron Institute of Oncology (VHIO).
By providing an interim evaluation of the performance of a clinical trial, adaptive designs allow for corrections that can lead to better results.
Faster and less cost
In the opinion of Dr. Garralda, “this type of adaptive study has multiple advantages over conventional studies since they are faster, less expensive and better show the risks and benefits of a drug. In addition, they are useful to correct or stop the clinical trial in progress if the drug does not provide satisfactory results, allowing the most promising drugs to advance faster.
Adaptive clinical trials allow you to change the study thanks to the data collected by expanding, for example, the sample or the duration to better show the real impact of the drug without compromising the validity or its integrity.
“The sooner we know whether a drug or dose works or not, the sooner we can decide whether to move it to the next phase of development or to stop testing it on patients. Also, by reducing spending on molecules that will ultimately fail, we can invest in more promising drugs in a much more efficient way than would be possible with the traditional clinical research model, ”Balcells emphasizes.
In addition to generating greater clarity about the potential of a drug being tested, adaptive designs are also an opportunity for patients to access new treatments in their earliest research phases.
“Thanks to the new adaptive trials, it will be possible for patients to have available before the new drugs that are being developed with the benefit that this affects them, without the need to carry out independent studies for each of the applications, as was necessary until now. ”, Says Dr. Garralda.
While the concept of adaptive clinical trial design has been around for a long time, the adoption of this new methodology is beginning to be used by major biotech companies. “At Amgen, we have opted to apply adaptive trial designs as the default setting for our clinical research in order to be able to perform clinical development as quickly and as inexpensively as possible and with a higher success rate,” concludes Balcells.