ince at least the birth of ICH in 1990, regulatory harmonization and trade globalization have followed parallel trajectories, synergizing successfully, exceeding expectations, and surprising those who may have initially been skeptical of its prospects. These trajectories are now diverging. This is not good news for patients.

here is no single solution to solve the challenge of recruiting and retaining participants in clinical research, and especially for those who have been underrepresented or historically excluded from participation. The need is great because such underrepresentation can affect drug utilization and patient experience in terms of product safety, efficacy, and quality. It can also materially impact the applicability of results to broader patient populations and the cost of research innovation. This challenge will not be effectively addressed or solved by incremental adjustments to recruitment approaches, particularly for individuals or groups most impacted by life-threatening health conditions in the real world. More diverse and inclusive representation would benefit everyone in the clinical research and product development process, including patients themselves.

he absence of labeling related to Patient-Reported Outcome Measures (PROM) has been striking: Between 2010 and 2020, out of the 108 oncology drugs approved by the US Food and Drug Administration (FDA), only nine (8.3%) included PROM-related labeling. In June 2021, the FDA released a draft guidance document titled “Core Patient-Reported Outcomes in Cancer Clinical Trials.” This guidance aims to enhance the quality and consistency of data collection related to PROMs in cancer trials. However, despite its potential benefits, pharmaceutical companies have been slow to fully embrace these recommendations.

one-size-fits-all approach to biopharma R&D no longer fits the technology-savvy, diversified world of drug development. Limited resources, patient recruitment challenges, and other barriers have forced the biopharma R&D industry to explore alternative trial designs, which this article illustrates along with some recently developed tools to enable their implementation.

lear, effective communication and close collaborations between statisticians and other functions at all stages of the clinical trial pipeline are key to ensuring that clinical trials are informative (regardless of the outcome). Unfortunately, clinical and statistical colleagues within the industry sometimes find this challenging. Statistical jargon often results in inefficient, confusing discussions between statisticians and the team at large, and study teams, particularly those from smaller organizations, often seek statistical support too late in the process. Both can compromise the study design and the data and their analysis. These challenges can lead to inefficiency and inconsistency in messaging to senior leaders who are keen to see how assets are progressing so that they can determine the next steps in terms of resourcing, prioritization, and regulatory discussions.

Overview

he electronic Common Technical Document (eCTD) is 16 years old, approved by the International Council on Harmonisation (ICH) to Step 4 in 2008. It’s old enough to drive a car in the US. It’s had some small changes over the years but is still pretty much the same as it was in the George W. Bush era. It’s had a huge impact on the speed and accuracy of regulatory submissions for pharma … but it could be better. That same year, the US Food and Drug Administration (FDA) started looking at a next-generation submission format, which would become eCTD 4.0. That standard was approved to Step 4 at ICH in 2015, and nine years later, FDA is still not using it. Let’s look at how we got to this point, and what’s holding us back.

edical product companies are entrusted to engage with healthcare providers and payers in a compliant manner. However, the emerging laws, the FDA’s evolving guidance landscape, and interpreting court decisions make compliance a complex process. This article discusses the new laws and FDA guidance documents that enable off-label communications with payers and providers and the differences between them. Specifically, focus will be placed on the Pre-Approval Information Exchange (PIE) Act, the Scientific Information on Unapproved Uses (SIUU) guidance, and the FDA’s guidance on medical product communications that are consistent with FDA-required labeling.

hile it is widely recognized that Reliance should be a fundamental part of a modern 21st century regulatory framework to efficiently use resources and avoid duplication of efforts, many open questions on how to operationalize its use on a global scale still remain. This is especially relevant for handling Post-Approval Changes (PAC), which represent the majority of regulatory submissions. Considering this, senior regulatory experts representing Health Authorities from all around the world joined industry representatives at a DIA Europe 2024 Pre-Conference Workshop to discuss the practical aspects of applying reliance approaches to PACs more widely.

he African Medicines Agency (AMA) is paving the way for Africa to achieve regulatory self-sufficiency and reliance. By aligning with global standards, this continent-wide regulatory ecosystem is expected to revolutionize patient care and access to medicinal products across Africa in new and unprecedented ways.

n mid-2023, Professor Tony Lawler succeeded Professor John Skerrit as Deputy Secretary, Health Products Regulation Group (HPRG), Australian Government Department of Health and Aged Care, leaving his position of Chief Medical Officer of the State of Tasmania, Australia.

Tony’s responsibilities include the Therapeutic Goods Administration (TGA), the Office of Drug Control, the Office of the Gene Technology Regulator, and the Australian Industrial Chemicals Introduction Scheme.

n recent years, real-world evidence (RWE) developed from real-world data (RWD) has become one of the hottest topics worldwide in the practice of drug and medical device registration and supervision. China’s regulatory authorities have also shown positive attitudes toward the role of RWE in drug R&D and registration review.

n February 24, 2022, many aspects of life in Ukraine were profoundly changed or altogether stopped. This included the clinical research industry: On this very day, the approximately 600 clinical trials in Ukraine were stopped. Subsequently, the State Expert Center of the Ministry of Health of Ukraine, the primary regulator of clinical research in the country, received 132 letters of premature clinical trial termination: 108 due to the invasion and 24 due to financial, safety, and efficacy issues. The State Expert Center also received 223 letters from sponsors and CROs regarding the transfer of clinical trial patients to other Ukrainian clinical trial sites or sites in other countries. Most of these were oncologic, neurologic, rheumatologic, gastroenterologic, or cardiologic patients.

Here is the story of one of those patients.

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