Part of a white paper that I wrote about patient enrollment trends in 2015. For full article :Clinerion
Patients in Clinical Trials
The increases in life expectancy and quality of life in the last century are emphasized in all health statistics. Although some authors point to an “increased female literacy rate” as the most important reason for this progress, the consensus suggests that clinical trial development is the most compelling reason (the impact rate is 40%). We would like to remark on the importance of clinical research, while accepting this exchange as an open discussion.
Clinical trials play a pivotal role in medical advances and in the drug development life cycle. It is quite possible to trace the beginning of clinical trial history to ancient times. Over the centuries, changes in scientific perspective have affected the doctrine of clinical research. Active participation by patients/human subjects in clinical research is one of the most critical factors driving successful drug development. Although preclinical laboratory research has been well defined through animal models, many regulations worldwide will not accept a drug’s claim of health benefits without human subject experience. The competence of any clinical trial is bounded with many conditions, most importantly, sufficient volume and desired quality of patient data. Consequently, throughout history scientists and patients have developed an interdependent relationship.
Today, we see that research procedures are more sophisticated; for example, the required study sample size is increasing and the duration of the studies is often longer. However, patient recruitment and retention rates have declined steadily.
With the development of evidence-based medicine, the evaluation of scientific publications is changing. Research designs are classified according to the evidence value; the sample size and the selection criteria of the subjects are more scrutinized. Comprehensive meta-analyses with the mission of identifying “universal truths” are more frequently published.
While these shifts help bring about a barrier to scientific fraud, the cost of proving added health benefits and/or improved outcomes with innovative drugs becomes much more challenging. In order to strengthen the evidence of the research, researchers need to study the drug’s effects in a larger sample population.
A study conducted in Italy reveals that within six years, there were 65% more clinical trial applications and the average duration increased more than 70%; however, the patient participation rate reduced by 21% and the retention reduced by 30%.
In another study, more than 80% of the international trials failed to reach a sufficient number of patients in the planned recruitment period. Indeed, 26.5% of investigator-initiated trials and 8.4% of sponsor-initiated trials faced early termination due to an insufficient number of patients. Even if the mentioned researchers published the evidence, its medical value would be considered as
Along with the above trends, the drug development costs have increased: in 1979 the cost of a drug was $100 million, in 2005 $1.3 billion, today it is over $1.7 billion.
Analysis of Trends
Today, our expectations of drug safety, understanding of benefits and risks, and regulations have increased remarkably compared to previous generations. Negative experiences, such as the thalidomide disaster in the early 1960s, show how drug development affects those at the community level. The necessity for more stringent regulations for the pharmaceutical industry has largely increased the cost of research and development activities.
The challenges facing clinical trials are not only growing costs (thereby increasing risk), they are also threatened by falling patient participation and retention trends. An insufficient number of patients will generally reduce the reliability of clinical research.
Along with a rapidly growing number of international research centers, more investigators and more sites are involved in the process. Relationships between investigators and patients are changing often and this can further complicate the process planning.
In a survey conducted in the USA, 32% of the drug development budget was related to patient activities such as participation, retention, incentives, etc. An extension in an international trial due to insufficient patient numbers resulted in a $ 1.3 million additional cost per day.