The number of elderly patients is rapidly increasing, but there is a lack of evidence to support treatment; it is urgent to design clinical trials that reflect the actual condition of elderly patients.
Background
With the development of medical technology and changes in population structure, elderly patients have become the fastest-growing group in modern medical practice. However, the scientific evidence to determine their treatment methods is significantly lacking. Existing clinical trials for new drug development have primarily focused on demonstrating the efficacy of drugs by including young patients with no comorbidities or good physical function. This selection method does not reflect the health status of elderly patients who often have multiple diseases and take multiple medications simultaneously. As a result, when the drug efficacy derived from clinical trials is applied to elderly patients in actual clinical practice, unexpected side effects or reduced efficacy may occur.
Key Findings
An international research team led by Professor Hans Wildiers of the University of Leuven in Belgium presented specific improvements in the design of clinical trials for elderly patients in a review article published in the medical journal The Lancet. The researchers identified three key areas to improve the usefulness of clinical trials, considering the high heterogeneity of elderly patient populations. The first area is to lower the threshold for recruiting participants to better represent the actual patient population. By relaxing the strict exclusion criteria, patients with comorbidities or slightly impaired physical function should be allowed to participate in clinical trials to obtain data that is more consistent with actual clinical practice. The second area aims to optimize therapeutic interventions by considering the physiological characteristics of elderly patients. This includes adjusting treatment doses to reflect polypharmacy or geriatric syndromes such as frailty, or incorporating de-escalation or non-treatment strategies into the clinical trial design instead of aggressive drug therapy. The third area involves establishing new evaluation indicators to measure clinically meaningful outcomes in elderly patients. Instead of focusing solely on indicators such as overall survival (OS) or progression-free survival (PFS), the study suggests that quality of life (QOL), maintenance of physical function, and the ability to live independently should be considered as major evaluation factors to assess the actual therapeutic value. The researchers pointed out that, although there have been increasing calls for these improvements over the past 10 years, the actual changes in research practice have been slow. Recently, however, there have been encouraging examples of successful trials that have been designed to better reflect actual clinical situations, thereby encouraging the participation of elderly patients and obtaining meaningful comparative data. In addition, the researchers added that combining clinical trial results with observational studies using real-world data (RWD) can provide a valuable complementary source of evidence to supplement the limitations of clinical trials.
Significance and Prospects
This study is expected to provide guidelines to the pharmaceutical and biotechnology industries and regulatory agencies for verifying treatments tailored to elderly patients. As regulatory agencies increasingly scrutinize the inclusion of data from elderly patients in new drug approval reviews, they are also demanding stricter representation of the clinical trial population. As a result, global pharmaceutical companies have begun to incorporate the comorbidities and physical frailty of elderly patients into the design of clinical trials from the initial stages. However, there are still many challenges to be addressed before these changes can be fully implemented. Relaxing the criteria for clinical trial participation may increase the risk of unexpected side effects and failure to demonstrate the efficacy of the drug, and it will also increase the cost and complexity of clinical operations. Ultimately, the key challenge is to find an appropriate balance between the precision of clinical trials and the representativeness of real-world clinical settings. If elderly patients are at the center of decision-making through this process, the industry expects that it will pave the way for reducing unnecessary overtreatment and providing the most appropriate medical services for individual needs.
Older patients represent the fastest growing patient group in clinical care. They are a heterogeneous group, for whom evidence for making treatment decisions is often scarce. Important domains for advancing the relevance of clinical trials for older patients are selection and inclusion of representative patients, choosing appropriate therapeutic interventions, and studying relevant outcomes. In the last decade, a myriad of publications has recommended multiple solutions that improve the relevance of trials for older people.
A practical application scenario can be clearly observed in the development of anticancer drugs in the field of oncology. In the clinical trial phase of new anticancer drugs, elderly patients aged 75 years or older with slightly impaired renal function or diabetes are included, and their survival period is extended, as well as factors such as maintaining physical activity and the ability to perform daily activities independently, are designed as the primary evaluation indicators. This helps clinicians to initiate drug treatment with less concern about side effects and with greater confidence. From the perspective of the pharmaceutical industry, the introduction of clinical designs tailored to the elderly is also a factor that increases market share. As society enters an ultra-aged era, products that have proven efficacy and safety in elderly patients, who are the actual consumers of drugs, will gain a competitive advantage in the market. As a result, drugs that increase their participation and provide appropriate dosage adjustment guidelines from the clinical trial stage are expected to gain an advantage in terms of adoption and prescription expansion.