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…

Read more
  • 0

FDA releases draft guidance to protect children in clinical trials

FDA has published draft guidance to clarify its perspective on including children in clinical trials.

The agency notes that it wrote the draft guidance to help industry, sponsors and institutional review boards (IRBs) protect children in clinical studies testing drugs, biological products and medical devices.

The draft guidance is titled “Ethical Considerations for Clinical Investigations of Medical Products Involving Children.”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

Read more
  • 0

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…

Read more
  • 0

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…

Read more
  • 0

What biopharmas should know about RBM and RBQM

[Image courtesy of iStock]

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)? 

Read more

  • 0

Startup vies to get blockchain adoption in pharma off the ground

[Image courtesy of Pixabay]

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…

Read more
  • 0

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

[Image courtesy of Pixabay]

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…

Read more
  • 0

Data-driven diversity: Making clinical trials equitable for all

Image courtesy of Pixabay

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…

Read more
  • 0

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 …

Read more
  • 0

9 predictions for pharma in 2022

Image courtesy of Pixabay

The pharma industry has been slower to embrace technologies such as AI and digital technology than many less-regulated sectors. The COVID-19 pandemic has changed the equation, forcing pharma companies to become more agile and open-minded in approaching drug discovery and development, including managing evolving clinical trials.

In this article, several experts offered their predictions of the trends that will be most meaningful in 2022.

1. Digital components drive new pharma value

Jaydev Thakkar

In 2022, a growing number of pharmaceutical companies will recognize how their products could deliver significantly improved outcomes with a digital component, predicted Jaydev Thakkar, chief operating officer of Biofourmis, which offers digital therapeutics and care-at-home solutions powered by AI. “This digital component c…

Read more
  • 0

RedHill Biopharma makes progress in RHB-107 COVID-19 study

RedHill Biopharma Ltd. (NSDQ:RDHL) has randomized the last patient in Part A of an ongoing Phase 2/3 study focused on RHB-107 (upamostat), an investigational antiviral.

The Tel Aviv–based company anticipates that RHB-107, a serine protease inhibitor, would be broadly effective against emerging SARS-CoV-2 variants.

RedHill Biopharma expects top-line data for Part A of the trial, which intends to evaluate the safety and tolerability and dose selection, to be available in the first quarter of 2022.

The trial is testing once-daily oral RHB-107 for non-hospitalized patients with symptomatic COVID-19.

By contrast, Merck (NYSE:MRK) and Ridgeback Biotherapeutics tested a twice-daily regimen for the COVID-19 antiviral molnupiravir. The companies tested molnupiravir in patients with mild-to-moderate COVID-19.

“Once again, we see a rise in COVID-19 infections in many countries across the world – and the need for effective, simple and safe oral t…

Read more
  • 0

U.S. to buy 1.4 million additional courses of molnupiravir

Merck (NYSE:MRK) and Ridgeback Biotherapeutics appear to be gaining growing support for their oral COVID-19 therapy molnupiravir.

One week after Great Britain granted conditional marketing authorization for the drug, the companies announced that the U.S. government intends to purchase 1.4 million additional medicine courses for approximately $1 billion.

The U.S. has committed to buy approximately 3.1 million molnupiravir courses for $2.2 billion, which equates to about $710 per course.

For the sake of comparison, the U.S. pays about $40 per dose of COVID-19 vaccines.

Molnupiravir photo courtesy of Merck.

The U.S. has the option of buying an additional 2 million courses of molnupiravir.

Merck and Ridgeback are awaiting news from FDA regarding their submission for emergency use authorization of molnupiravir. The application would cover adults with mild-to-moderate COVID-19 who have …

Read more
  • 0