Young adults with chronic medical conditions 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 trials often overlook the unique needs and characteristics of young adults (>18 years of age), leading to significant gaps in our understanding of how treatments affect this population.

Current clinical trials often group a wide range of ages (e.g., 18-64) when 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.

Young adult patients also need robust safeguards for pharmaceutical and medical device products we rely on to ensure their safety and efficacy.

Young Adult Representation in FDA User Fee Reauthorizations

Generation Patient is committed to ensuring young adult patients are represented in the reauthorization of the Prescription Drug User Fee Act (PDUFA), the Medical Device User Fee Act (MDUFA), and other User Fee Reauthorizations. We are working hard to ensure the following priorities listed below are included in PDUFA VIII and MDUFA VI.

Click here to learn more about the importance of FDA User Fees!

White Paper: Promoting Transparency in Clinical Research and Post-Market Data Reporting for Young Adults and Other Adult Age Populations: Policy Recommendations

Gaps remain in understanding the clinical outcomes related to young adults, due in part to the underrepresentation of young adults in clinical and post-market data reporting for drugs, biologics, and medical devices. Without standardized reporting of data specific to young adults and other adult age groups, clinical researchers, regulators, health providers, and patients remain largely in the dark about how these physiological differences are impacting therapeutic safety and efficacy for specific age subgroups.

Read Generation Patient’s white paper on supporting young adult representation.

Check out our latest work:
  • Comment to FDA - On the Reauthorization of the Prescription Drug User Fee Amendments VIII
  • Comment to FDA - Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies; Guidance for Industry
  • Abstract at UEGW - United European Gastroenterology Week: Exploring the age spectrum of real-world patients with Inflammatory Bowel Disease: a machine learning approach
  • STAT News Article: If Pharma Can Market to Youths by TikTok, it Should Include Them in Clinical Trials
  • Generation Patient Letter: Prescription Drug User Fee Act Feedback
  • Presentation at University of Pennsylvania’s Research Ethics and Policy Series (REPS): Representation of Adolescents and Young Adults in Clinical Trials

There are biological impacts of age, including: 

  • Young adults experience unique physiological changes, including hormonal shifts, rapid metabolic adaptations, and varying immune responses, which may affect how they react to medications compared to older adults. 

  • For example, hormonal fluctuations can influence drug metabolism, potentially altering therapeutic effects or risk profiles.

To better understand age-related differences, we recommend standardized data disaggregation:

  • Standardize adult clinical and post-market data reporting across Health and Human Services, utilizing the following model: 

    18 - 24 years; 25 - 34 years; 35 - 44 years; 45 - 54 years; 55 - 64 years; 65 - 74 years; 75 - 84 years; 85 - 94 years; 95 + years

  • Integrate age analysis into FDA multidisciplinary reviews

  • Hold public meetings on young adult health trends, clinical research, and adverse event reporting

 

Improving Clinical Trial Data Reporting

Where data is reported for young adults, it is often aggregated with other adult age groups, clinical trials may report participants in a single age grouping of 18-49, 18-64, or 18-65 with little consistency, despite the known changes in drug safety and efficacy that occur as humans age. Requiring clinical trial sponsors to disaggregate adult data by narrower age brackets is crucial for patients, providers, and regulators to make more informed decisions regarding the treatment options for specific age subgroups.

FDA Drug Trial Snapshot: TZIELD Delay Onset of Stage 3 Type 1 Diabetes for High Risk Individual. Figure 3. Baseline Demographics by Age, Efficacy Population. 28%: 18 to 65 (21 patients). 72%: <18 (55 patients)
FDA Drug Trial Snapshot: ALYFTREK Treatment of Select Variants of Cystic Fibrosis. Figure 3. Baseline Demographics by Age, Efficacy POpulation. <18 (136 participants)14%. 18 to 65 (831 participants) 85.6%, >65 (4 participants) 0.4%
 

Updating Adverse Event Reporting Systems

The lack of focus on age-specific data extends to adverse event reporting systems. Currently, the FDA groups individuals aged 18-64 as a single demographic.  The current broad age groupings make it exceedingly difficult to track drug safety signals impacting specific adult age subgroups, emphasizing the need for disaggregated adult age groupings.

FDA Adverse Event Monitoring System (AEMS) Public Dashboard for Drugs and Biologics
 

Ensuring Our Medical Devices are Safe and Effective

There is a major regulatory gap in ensuring the safety and efficacy of the medical devices young adult patients rely on for their survival. Especially, as FDA reports that annually, they receive over two million suspected device-associated malfunctions, serious injuries, or deaths. Medical Device post-market surveillance and adverse event reporting systems must be strengthened and need to include age-specific data that include young adult patients. In addition, the long term impact of implanted medical devices needs to be reviewed, especially during the transition from pediatrics to young adulthood.


In 2026, we will continue developing evidence and working with legislators to determine the next steps for more representative and transparent clinical trials. For more information or to collaborate, email admin@generationpatient.org.