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

[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)? 

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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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9 predictions for pharma in 2022

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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…

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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…

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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 …

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Merck projects at least $5B in 2022 sales for molnupiravir 

Merck’s (NYSE:MRK) investigational oral COVID-19 drug could net between $5 billion and $7 billion in revenue through the end of 2022 if FDA authorizes its use in December, the company projected.

Sales of the drug could be higher, assuming it finds widespread use in wealthy countries.

Merck expects sales between $500 million and $1 billion for 2021.

Merck’s stock climbed more than 5% per share to $85.70 in mid-day trading.

Several other companies are also developing COVID-19 antivirals, including Pfizer and Novartis. Roche and Atea recently announced disappointing Phase 2 results involving their investigational antiviral AT-527.

Merck also announced that the molnupiravir, which it developed with Ridgeback Biotherapeutics, would be available in developing countries. To that end, Merck agreed to a royalty-free license for molnupiravir with the Medicines Patent Pool. As a result of the arrangement, generic drug manufacturers could sell a five-d…

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Pfizer-BioNTech COVID-19 vaccine was 91% effective in 5- to 11-year-olds in Phase 3 study

Pfizer (NYSE:PFE) and its partner BioNTech (NSDQ:BNTX) have announced that their COVID-19 vaccine was 90.7% effective at protecting against symptomatic COVID-19 in children ages 5 to 11. 

The study concluded that a 10-µg dose of the BNT162b2 vaccine, which is one-third the amount authorized for people 12 and older, had the best benefit-risk profile.

Recipients of 20- and 30-µg doses had significantly higher rates of mild or moderate side effects, including fatigue, headache, chills and muscle pain. 

The independent Vaccines and Related Biological Products Advisory Committee (VRBPAC) will meet next week to discuss the prospect of recommending the vaccine for use in 5- to 11-year-olds to the FDA. 

 The BNT162b2 vaccine is currently FDA approved for people 16 and older. An emergency use authorization currently covers children aged 12 to 15.

One concern regarding the use of the vaccine in younger children is its association with myocarditis an…

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Why the pandemic is a crucible for clinical trial innovation

Image from Sam Moqadam on Unsplash

The rapid pace of development of COVID-19 vaccines and antibodies has redefined expectations for clinical trials. As SARS-CoV-2 variants such as Delta continue to fuel the epidemic, the industry must continue to ensure testing and clinical trials stay ahead of mutations.

To get a clearer sense of what the ramifications of this accelerated development will be for the industry at large, we spoke with Neta Bendelac, senior director of strategy at 4G Clinical, which offers software and services for clinical drug trials.

Drug Discovery & Development: What impact has the pandemic had on trials for COVID-19 therapies?

Neta Bendelac: Speed was the most important factor for the early COVID-19 studies as many sponsors raced to get promising therapies and potential vaccines into the clinic. We all felt the urgency, all day, every day. It made sense for the FDA to sti…

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