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…

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How EEG and machine learning are transforming epilepsy clinical trials

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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.


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

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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…

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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…

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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…

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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…

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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…

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What biopharmas should know about RBM and RBQM

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As a growing number of emerging biopharma companies seek to develop novel treatments for chronic diseases, the clinical trials market could be worth $78.3 billion by 2030. 

Clinical trials, however, continue to be difficult and expensive to manage. Yet successful clinical trials, however, remain critical for investors. Small biopharmas are thus looking for guidance in navigating clinical trial design to limit risk and bolster their chance of success. 

To help identify what emerging biopharma companies need to know to keep in mind when planning clinical trials, we reached out to two experts at IQVIA. Adrian Kizewski is an associate director of RBQM, digital trial management suite at IQVIA, while Gayle Hamilton is a director in the same division at the company. 

What is the difference between Risk-Based Monitoring (RBM) and Risk-Based Quality Management (RBQM)? 

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Startup vies to get blockchain adoption in pharma off the ground

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Blockchain may be arguably one of the most hyped technologies in recent memory, but the distributed ledger technology better known for its role in cryptocurrency may have significant potential in the pharma supply chain, clinical trials and beyond.

A blockchain-based initiative known as PharmaLedger has won support from prominent companies like Pfizer, Novartis, Merck & Co., Bayer and AstraZeneca.

In 2019, the FDA launched a pilot program supporting the Drug Supply Chain Security Act with the support of IBM, KPMG, Merck & Co. and Walmart.

The pandemic’s disruption of the pharmaceutical supply chain further spurred industry interest in exploring blockchain to improve logistics networks, as the Harvard Business Review observed.

Overall adoption of blockchain in pharma or the enterprise at large, however, remains at a nascent stage.

Blockchain in…

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A real-world data approach for bridging diversity disparities in clinical trials

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The lack of appropriate representation in clinical trials, particularly in terms of ethnicity and race, has been a long-standing issue that directly impacts health equity and treatment efficacy. In a 2020 analysis of the global participation in clinical trials, the Food and Drug Administration (FDA) highlighted the vast difference between enrolled participants and the global population. Of the more than 297,000 participants in clinical trials globally, 76% were white, 11% were Asian, and only 7% were Black. By comparison, 60% of the global population hails from Asia, 16% from Africa, 10% from Europe, 8% from Latin America, and just over 4% from the United States.1

The FDA has focused on addressing this issue, drafting new guidance in April 2022 aimed at increasing clinical trial enrollment from underrepresented racial and ethnic populations. This draft guidance, “Diversity Plans to Im…

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Data-driven diversity: Making clinical trials equitable for all

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Lack of diversity in clinical trials has long been an issue, driven by challenges with recruitment and participation. In recent years, pharmaceutical companies have prioritized recruiting more diverse patient groups for their trials. And in some areas, it is working. In the past ten years, the representation of Black and African American patients in U.S.-based clinical trials has improved. Currently, data from Phesi show that 14.9% of clinical trial participants self-identify as being in this group. That proportion is slightly greater than the 13.4% of the U.S. population that identifies as Black and African American, according to the 2019 U.S. Census.

To develop truly effective therapies for all, clinical trials must be carried out in populations representative of those who will receive the new treatments. However, certain patient subpopulations are significantly and consistently u…

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Radiomics: The present and future of advanced imaging analytics

Radiomics image courtesy of Wikimedia Commons

Radiomics, or the science of advanced imaging analytics, is an emerging field that promises more personalized care, improved clinical decision support and greater efficiency in clinical trials. 

Radiomics are being used to discover new biomarkers, improve the accuracy of diagnosis, predict the risk of disease and likelihood of treatment response, and identify clinical endpoints.

Today, much of the focus in radiomics has been in oncology. However, radiomics applies to other clinical areas and is being used today in several clinical domains. Radiomic data are a key component of a “multi-omics” approach to medicine, in which radiomic data is combined with data sets from other sources such as genomic and clinical data. Adding more robust phenotype data to these data fabric will present clinicians with synergistic information in which the whole is greater than …

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