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!
- 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 public data disaggregation:
Sponsors make age-specific data more accessible.
The FDA mandates public reporting of disaggregated data by age.
At least five-year increments disaggregate trial data.
Recent research proposes a standardized set of age groupings for health data, including more nuanced groupings for younger ages followed by 5-year age groupings for adults (see sources here and here).
Improving Adverse Event Reporting
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. Disaggregating data by narrower age brackets is crucial for publicly capturing the nuances of disease epidemiology and treatment effects in young adults.
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.