Generation Patient is a nonprofit organization created by and for young adult patients—i.e., young adults with chronic medical conditions such as lupus, inflammatory bowel disease, Lyme disease, rheumatoid arthritis, and more. As young adults, we are at a pivotal life stage: navigating the transition to adulthood while simultaneously managing unpredictable health conditions and our personal, educational, professional, and psychosocial needs. To address our community's unique challenges, Generation Patient provides peer-support programming and drives systemic change through policy work, leadership programming, and evidence generation. We aim to amplify young adult patient voices in critical conversations where real change can occur. Through this strategy, we envision a future where young adults with chronic medical conditions can live with greater quality of life, access, dignity, and fulfillment. As an organization, we do not accept funding from private healthcare, including pharmaceutical and insurance industries. We are guided entirely by the lived experiences of young adult patients, including this community-drafted comment.

Below, we have included some considerations from our community of young adults with chronic/rare conditions, many of whom are heavily impacted by the issue of underrepresentation in clinical trials.

The Importance of Including Young Adults in Clinical Trials and Disaggregating Reported Data

Young adult patients represent a distinct and growing demographic, with over half of young adults in the United States managing at least one chronic condition. Despite this, clinical care often categorizes young adults (aged 18-34) similarly to older adults, leading to significant public unknowns of young adults in clinical trials. This oversight is concerning because physiological changes unique to this life stage, such as hormonal shifts and metabolic variations, can significantly impact disease presentation, treatment response, and potential complications. For certain conditions, young adults may experience different drug effects or adverse events compared to older populations, highlighting the need to treat this age group as distinct from "pediatrics," “adolescents,” "adults," or "older adults."

Key Considerations for Clinical Trials:

1. Defining Age Brackets: Clinical trials often group a wide range of ages (e.g., 18-64) when publicly reporting safety and efficacy data. This broad categorization can obscure age-specific responses to treatments. Establishing clearer age brackets in clinical trial design and data reporting is crucial for identifying potential risks or dispositions unique to young adults.

2. Understanding Biological Impacts of Age on Drug Safety and Efficacy: The biological impact of age on drug response and safety is a crucial but under-researched area. While significant progress has been made in understanding how age affects drug responses in cancer treatments, this knowledge is largely absent for other chronic and rare conditions. Young adults who experience unique physiological changes—including hormonal shifts, rapid metabolic adaptations, and varying immune responses—may react differently to medications compared to older adults. Failing to account for these age-specific biological differences can lead to suboptimal treatment protocols, unrecognized adverse events, and overall poorer health outcomes for our demographic. Exploring how hormonal changes, common during young adulthood, affect drug efficacy and safety is crucial. For example, fluctuations in hormones like estrogen, progesterone, and testosterone can influence how medications are metabolized, potentially altering their therapeutic effects or risk profiles. These considerations are important for recognizing and mitigating potential complications that may arise from inappropriate drug dosing or overlooked side effects.

3. Examples of Age-Related Differences:

Hormonal Profiles and Fertility: Studies have shown significant variations in hormonal levels across different age groups, affecting fertility-related outcomes. For instance, younger women (<20 years) and those aged 20-29 have more favorable hormonal profiles than women aged 30-39 and 40-49. These differences indicate that fertility-related adverse events may be underreported or misinterpreted if data from younger and older women are combined. (Bartimaeus et al., 2020)

Impact of Immune Checkpoint Inhibitors (ICIs): Research on the effects of ICIs in young women with melanoma demonstrated a decrease in ovarian reserve markers, highlighting the need to separate younger women in clinical trials to accurately assess the impact of treatments on fertility. (Hickman et al., 2023)
Ovarian Reserve in Hypopituitarism Patients: A study on women with hypopituitarism found that they had lower AMH levels compared to age-matched controls, illustrating how underlying health conditions can influence fertility outcomes. (Fitz et al., 2023).

Proposed Solutions and Considerations:

Sponsors should make age-specific data more accessible, and the FDA should mandate public reporting of disaggregated data by age.
Research on conditions affecting young adults should prioritize exploring the potential for unique disease epidemiology in certain conditions. Trial data should be disaggregated by at least five-year increments to provide a clearer understanding of age-related differences.

Young adult patients are at a unique time period as it pertains to independence. The fear and stigma of sexual and reproductive health is an important consideration in the enrollment of young adults in clinical trials. Ensuring confidentiality and creating a supportive environment can encourage participation from these under-represented groups.
Trust: Sponsors should be required to provide more clarity surrounding post-trial access to meds. This is a real opportunity to increase trust by understanding the long-term opportunity to access medicines as part of a trial.

Recommendations for Improving Adverse Event Reporting:

While not specific to clinical trials, the lack of focus on age-specific data extends to adverse event reporting systems. The FDA currently groups individuals aged 18-64 as a single demographic, while the CDC and other registries use narrower age bands. Disaggregating data by narrower age brackets is crucial for capturing the nuances of disease epidemiology and treatment effects in young adults.

Young adults with chronic and rare conditions must be included in clinical trial design for disease areas impacting our demographic. We are a growing demographic with many specific considerations, as outlined above. We welcome the opportunity to engage further.

Sincerely,

Generation Patient

admin@generationpatient.org