# Understanding effectiveness of new drugs in older adults shortly after market entry

> **NIH NIH R01** · BRIGHAM AND WOMEN'S HOSPITAL · 2020 · $364,106

## Abstract

When a new drug enters the market, comparative effectiveness evidence is often comprised solely of evidence
from the randomized clinical trials (RCTs) which led to regulatory agency approval. Older adults are major
consumers of drugs and other therapeutics and underrepresented in randomized clinical trials. During the
critical early period after a new drug enters the market, evidence from RCTs may not reflect the average
experience for the types of patients actually treated and there is insufficient accrual of ‘real world’ experience in
longitudinal healthcare databases for robust evidence generation.
At this critical juncture, combining evidence can enhance understanding of net benefit of new drugs in the
older, sicker populations who are actually treated in practice. However, appropriate methods for integrating
pre-market RCT and early post-market comparative effectiveness evidence to guide clinical practice have not
yet been identified. We will explore these issues in three case studies. For each case study, we will obtain
individual level data from pre-market RCTs and create observational cohorts comprised of initiators of the new
drug and comparator using Medicare data. We will evaluate different methods which combine evidence
generated from RCT with ‘real world’ evidence from data observed in routine care of older adults in Medicare
during early as well as later post-market experience with the new drugs. These methods include complex
weighting as well as cross-design synthesis methods that combine information from RCT and ‘real world’ data.
Specifically we will research: (1) How can innovative application of methods to combine individual-level data
from pre-market RCTs and early post-market observational data accelerate understanding of effectiveness and
safety of new to market medications in older patients who are typically underrepresented in RCTs? (2) How
can we evaluate and understand reasons for differences in treatment effect estimates from pre-market RCTs
and observational data in the early post-market time period?
This project will produce a framework for combining pre-market RCT and early post-market evidence as a
means to accelerate understanding of treatment effectiveness in older adults with multiple comorbidities. Use
of this framework will provide early insights and clinical guidance to geriatricians on use of new drugs in their
patients shortly after market entry. Because the methods used in this project are designed to provide early
evidence that reflect average effectiveness in the types (and subtypes) of patients actually treated as part of
routine care as opposed to average effectiveness in participants of a trial, the impact of this project will be
particularly profound for new medications that target older, sicker patients typically underrepresented in trials.

## Key facts

- **NIH application ID:** 9908033
- **Project number:** 5R01AG053302-03
- **Recipient organization:** BRIGHAM AND WOMEN'S HOSPITAL
- **Principal Investigator:** Shirley Wang
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $364,106
- **Award type:** 5
- **Project period:** 2018-08-01 → 2022-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9908033

## Citation

> US National Institutes of Health, RePORTER application 9908033, Understanding effectiveness of new drugs in older adults shortly after market entry (5R01AG053302-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9908033. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*