Moving the needle on diversity in clinical trials: Where do we go from here?

[Image courtesy of Adobe Stock]

Enhancing patient diversity in clinical trials has become a key priority in drug development. The main concern is that critical data that includes underrepresented patient populations is being left out as many clinical trials do not reflect all populations that may eventually take a therapy. These underrepresented groups consist of women, including those who are pregnant and lactating, pediatric and elderly patients, people with disabilities, LGBTQ+ individuals, and racial/ethnic groups specifically, Black/African American, American Indian/Alaska Natives, Hispanics/Latinos, Asians, Native Hawaiian, and other Pacific Islanders.

As a result, the U.S. government has taken increased measures with the passage of the Food and Drug Omnibus Report Act of 2022 (FDORA), which will require sponsors to submit diversity plans to the U.S. Food and Drug Administration (FDA) for all late-stage st…

Read more
  • 0

One-time gene therapy Zolgensma from Novartis shows lasting benefits for SMA patients

[Zolgensma image from Novartis]

A gene therapy for spinal muscular atrophy (SMA) patients is making waves. Novartis (NYSE:NVS) revealed new long-term data highlighting the durability of Zolgensma (onasemnogene abeparvovec) up to 7.5 years after a single treatment. The data comes from two long-term follow-up studies, LT-001 and LT-002, which examined a range of patient populations and demonstrated a positive overall benefit-risk profile. LT-001 is an ongoing 15-year long-term follow-up study of patients who completed the phase 1  START study. More than 3,000 children have been treated with Zolgensma in clinical trials, managed access programs and commercial settings. 

Novartis expects Zolgensma to eventually generate $1.5–2 billion, according to BioPharma Dive. NVS shares were up 1 percent to $83.01 today after announcing the new data. 

FDA approved Zolgensma (onasemnogene abeparvovec-xioi) in 2019 for chi…

Read more
  • 0