clinical trials Archives - Page 5 of 11

AI in drug development: Janssen exploring potential of AI in everything from target discovery to clinical trials

As enterprise AI adoption surges, the life science industry stands at a pivotal juncture. “We are truly at a tipping point,” remarked Najat Khan, chief data science officer at Janssen, during the 2023 Stanford Drug Discovery Symposium. “It’s amazing how quickly we’ve been able to make progress across the whole value chain in terms of drug development,” she said. “The reality is that there’s a lot of hype, but there is already a lot that’s happening.”

While most Big Pharma companies are exploring AI applications, as a 2022 Clinical Trials Arena article noted. It’s easy to understand why. While developing a given drug might take more than a decade, AI could potentially enable the development of “drugs in one-tenth of the time, from being discovered to being able to treat patients,” wrote McKinsey partner Alex Deverson in late 2022.

Janssen is aiming to distinguish itself by building a team of “bilingual”…

Accelerating Alzheimer’s research: ADDF’s chief scientific officer reflects on the Lauder Foundation’s $200M gift

Beta-amyloid plaques and tau in the brain. [Image from National Institute of Aging]

The Estée Lauder family has donated $200 million to the Alzheimer’s Drug Discovery Foundation (ADDF), a nonprofit they founded in 1998 to support Alzheimer’s research. The gift is the largest ADDF has received.

“We’ve deployed about $270 million so far for about 700 programs in 19 countries of drug discovery and development over the past 25 years,” said Dr. Howard Fillit, co-founder and chief science officer of the nonprofit.

The Lauder gift will sustain ADDF’s philanthropic model for the next 10 or 15 years, Fillit said. “We want to use our donors’ money solely for the advancement of the development of new drugs for Alzheimer’s disease,” he explained. The organization ensures that every cent of each dollar donated goes towards drug research, with no deductions for administrative costs, salaries, rent…

AbbVie and Janssen voluntarily withdraw Imbruvica from accelerated approvals for MCL and MZL

AbbVie (NYSE:ABBV) and Janssen Pharmaceutical (NYSE:JNJ) have revealed their intent to voluntarily withdraw the accelerated approvals for Imbruvica (ibrutinib) for patients with mantle cell lymphoma (MCL) and marginal zone lymphoma (MZL) in the U.S.

Ibrutinib is a selective Bruton’s tyrosine kinase (BTK).

The main reasons for the move relate to FDA’s request for additional studies to confirm clinical benefits that relate to the accelerated approval status granted by the FDA for MCL and MZL. Initially, FDA authorized the accelerated approval program for the two indications following the publication of positive overall response rate data in phase 2 trials.

While the phase 3 shine study in previously untreated MCL met its primary endpoint of progression-free survival, there was an increased rate of adverse reactions when combining Imbruvica and chemotherapy compared to the placebo-controlled arm.

Conversely, the phase 3 SELENE study in re…

Three strikes in pharma: Exploring recent drug withdrawals and clinical trial challenges

[Gorodenkoff/Adobe Stock]

Pharmaceutical companies face a long list of regulatory challenges ranging from patent expiry to bioequivalence and international harmonization. It’s not uncommon for drug makers to withdraw or discontinue drugs after failing to meet clinical requirements or endpoints, resulting in drug withdrawals. On average, life science companies pull roughly 4,500 drugs and devices from the market, many of which are widely used. In recent years, drug companies, for instance, have pulled a growing number of products for nitrosamine contamination. While the FDA can identify safety concerns, it often falls upon the manufacturer to initiate and execute a recall.

Here, we round up three recent examples of recent drug withdrawals where pharma companies either pulled drugs from the market or gave up on developing a drug for a given indication.

Covis Pharma withdraws Makena

The Zug, Switzerland–base…

Breaking down barriers: Prioritizing diversity in clinical trials

[Image courtesy of FDA]

Diversity in clinical trials is crucial for ensuring that new medical treatments are safe and effective for all populations. Last year, the FDA released draft guidance that aims to improve the enrollment of historically underrepresented populations in clinical trials.

Demand is growing for more transparency and diversity in clinical trials, according to Erin Leckrone, senior director of clinical trials at Be The Match BioTherapies. Additionally, a growing number of stakeholders are stepping forward to promote diversity. “The COVID pandemic really amplified a lot of people’s voices in this space,” she said.

A growing number of stakeholders have stepped forward to demand better diversity in clinical trials and to understand “what data is actually collected as part of the clinical trials,” Leckron said. Those players range from regulatory agencies like the FDA to clinical trial …

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…

How EEG and machine learning are transforming epilepsy clinical trials

[Image courtesy of peterschreiber.media/Adobe Stock]

Epilepsy is a brain disorder that triggers recurring seizures. It is the fourth the most common neurological disorders in the world, according to the Epilepsy Foundation. The Centers for Disease Control and Prevention estimates that 65 million people worldwide have active epilepsy. In 2015, 1.2% of the total U.S. population — 3 million adults and nearly 500,000 children — had active epilepsy.

There are many different causes of epilepsy, including genetics, head trauma, brain abnormalities, infection, prenatal injury and developmental disorders, such as autism.

Seizure symptoms vary greatly and can manifest in a person as uncontrollable limb movements, staring, muscle stiffness, confusion and loss of consciousness or awareness.

These symptoms are not mutually exclusive. Some patients with epilepsy experience multiple types of seizures.

Epil…

Harnessing the untapped potential of legacy data in pharma R&D

[Image courtesy of James Thew/Adobe Stock]

Clinical trials for a new therapy cost a median of $41,117 per patient. Costs like this are no surprise to pharma leaders. But during an age of increasing budgetary pressures, drug developers are under pressure to do more with less money and staff. While there are no “simple” answers to this challenge, there is one strategy that offers research and development (R&D) teams a very powerful approach: better leveraging existing legacy data.

Pharmaceutical companies own petabytes of imaging data, generated by in-house research, investigator-initiated studies or clinical trials. This data is valuable and can yield insights that can help researchers better understand disease mechanisms and inform therapeutic approaches. But in many cases, researchers cannot access this important data, as it remains in silos with CROs, investigator labs, or within a specific research group…

Janssen sees Blood Cancer Awareness Month as an opportunity to close the clinical trial diversity gap

Janssen (NYSE:JNJ) is working to highlight the importance of cancers such as leukemias, lymphomas and multiple myeloma for Blood Cancer Awareness Month in September.

Some 1.5 million people in the U.S. are currently living with or in remission from blood cancers, according to the Leukemia & Lymphoma Society. Roughly 35,000 people are diagnosed with multiple myeloma each year, according to the American Cancer Society.

In addition to highlighting the incidence of hematologic malignancies, Blood Cancer Awareness Month offers an opportunity to provide education about the need for improved clinical trial diversity. While the industry has made strides recently, people of color continue to be underrepresented in clinical trials. According to FDA data, in 2020, three-quarters of trial participants were white, while only 8% were Black. That same year, 11% of clinical trial participants were Hispanic.

According to the U.S. Census Bureau, 13.6% of the U.S. popu…

FDA releases draft guidance to protect children in clinical trials

FDA has published draft guidance to clarify its perspective on including children in clinical trials.

The agency notes that it wrote the draft guidance to help industry, sponsors and institutional review boards (IRBs) protect children in clinical studies testing drugs, biological products and medical devices.

The draft guidance is titled “Ethical Considerations for Clinical Investigations of Medical Products Involving Children.”

It includes an ethical framework to safeguard children in clinical research. It provides recommendations for ethical frameworks for IRBs, sponsors and industry. In particular, the document includes information on evaluating whether an investigational therapy is likely to offer a prospect of direct benefit to a child. It also provides an assessment of risk for interventions with the potential of direct benefit and protocols for obtaining permission from a parent or guardian and consent from a child.

FDA’s Office of Pediatri…

Using real-world evidence in clinical trials: Challenges and opportunities

Photo by Joshua Sortino on Unsplash

The healthcare industry traditionally has used site-based clinical trials to assess the efficacy and safety of drugs and medical devices. Data collected from participants in these trials would be evaluated to determine whether the product in question should be approved for commercial use.

In recent years, however, the exponential increase in patient data recorded by digital devices has raised the possibility that real-world data (RWD) and real-world evidence (RWE) may be used to augment clinical trial data.

But what are RWD and RWE? Are they the same thing? And how could they effectively be integrated into clinical trials to produce better outcomes for consumers?

Here’s how the FDA defines RWD and RWE:

Real-world data relate to patient health status and/or the delivery of healthcare routinely collected from a variety of sources. RWD can come from mul…

Defining complexity: A framework to identify complex clinical trials and set them up for success

It’s easy to feel like clinical trials are complex. They are experiments involving human beings — so things change. But, despite feeling like complexity is everywhere, there is little clarity on what constitutes a complex trial and what sponsors need to do to prepare these trials for success. Both the FDA and EMA, for example, offer somewhat vague definitions of what makes trials complex; the FDA says that designs “intended to advance and modernize drug development” are complex.1 The EMA defines complex trials as having “non-conventional … elements, features, methods … that confer complexity of their designs, conduct, analyses or reporting.” Neither of these definitions provides a practical framework to help sponsors identify what makes trials complex, whether a particular trial meets the criteria, or how to set up complex trials for the greatest chance of success.

This article aims to fill that gap by providing a framework for complexity in clinical trials. Over Suvo…