here are few things that a parent dreads more than having a sick child. But the journey to diagnosis and recovery for what could be a child’s fatal illness can be even more difficult.

When her 8-year-old daughter Autumn was diagnosed with Stage 2 T-cell acute lymphoblastic leukemia (T-ALL), Ebony Dashiell-Aje knew that their family was about to venture down a challenging road. But she didn’t anticipate how Autumn’s outlook would help lead their family through their darkest days, or help encourage others through a documentary of their experience titled Autumn’s Story.

In the following interview, Ebony shares three perspectives on Autumn’s road to diagnosis, treatment regimen, and recovery: as a pharmaceutical industry professional leading patient-centered outcomes research, as a pediatric oncology patient caregiver, and as Autumn’s mom. The video documentary and our audio interview with Autumn are embedded within this interview.

very authorized medicine has potential benefits and potential risks; its benefits must outweigh its risks for it to be licensed and remain in the market. We can improve the balance between benefit and risk by minimizing the risks, especially those that are serious and may substantially impact patient safety. Risk mitigation can be substantially improved by engaging patients in co-creating educational materials on the medicines they use.

here is increasing agreement that aiming for health equity through inclusive clinical research is in the best interest of patients. This renewed focus from research sponsors on diversifying their clinical trials has also been driven by new laws (i.e., the December 2022 Food and Drug Omnibus Reform Act – FDORA, sections 3601-3604) and regulatory guidance (i.e., the April 2022 FDA Draft Guidance to Industry on Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials) that require submission of diversity plans to FDA prior to the start of pivotal studies.

he major truism accepted by all is that biology is complex. Yet, since the 19th century, the major experimental focus in biology has been reductionist, which is to isolate component parts, divine their individual function, and then use this knowledge to extrapolate the behavior of the larger system. With the advent of comprehensive genomic, proteomic, and metabolomic technologies powered by advanced computational approaches, all possible genes have been mined, and each resultant mutant molecule or pathway can be inferred.

efining intelligence, whether natural or artificial, remains a realm shaped by subjective perceptions. Intelligence is often measured by the yardstick of reasoning, with mathematics serving as the benchmark for such logical processes. While deep learning artificial intelligence (AI) language models excel at pattern recognition, they currently struggle with reliably solving even eighth-grade mathematical problems. Two quotes worth considering from a human utility standpoint are George Box’s “All models are wrong, but some are useful” and Lord Kelvin’s “To measure is to know. If you cannot measure it, you cannot improve it.”

hat does the path to eradicating all human disease look like?

Almost two years ago, I asked this question in a blog titled “Accelerating the path from possibility to proof in the development of new medicines.” I wrote that leveraging our most advanced tools would drive new synergies at the intersection of safety, science, and speed and usher in a new era of drug development to save and improve countless lives. Today, we are here. Today, we stand at the threshold of a new era of scientific development fueled by artificial intelligence and machine learning (AI and ML).

rtificial intelligence/machine learning (AI/ML) has been leveraged in various industries for decades, but generative AI (GenAI) has recently emerged to offer new use cases in life sciences. Ultimately, GenAI—or any other form of AI—is not going to meet today’s lofty expectations if it does not provide a tangible return on investment (ROI).

ecent years have seen a dramatic increase in new clinical trial technologies. These tools, layered onto sites’ daily workflows, are meant to capture more high-quality data as well as alleviate the traditional burdens of trial operations.

t’s not just about survival, it’s about increasing or maintaining function as a patient. While you’re still able to, you know, give a hug to a child, or take a sip of water, … the focus seems to be on survival. And I agree, that is the end game. But functionality until you get to the cure is equally important.” —Steven Kowalski, ALS patient and patient advocate.

he range of treatment modalities now available is promising for amyotrophic lateral sclerosis (ALS) patients, but frameworks to guide prioritization of development are lacking. The development of efficient solutions for patients is impeded by a multitude of challenges that require collective efforts from researchers, policymakers, and other stakeholders.

eal-world evidence (RWE) has taken center stage in the biopharma landscape, offering insights into drug applications and therapeutic impacts in real-world settings. As the field evolves, understanding the present use of RWE and anticipating its future trajectory is key. At DIA’s recent RWE Conference, stakeholders and experts from the pharmaceutical industry, regulatory bodies, and academia convened. Various questions surrounding RWE were broached, addressing concerns about data quality and access, compliance, and the application of emerging technologies.

thical behavior in clinical research and therapeutic product development can help demonstrate the trust that transforms patients from research participants into research partners. In the following interview, DIA Bioethics Community Co-Chairs Karla Childers and Lindsay McNair look back at their foundational educational experiences, explain the founding of the Bioethics Community, and outline topics they anticipate this community will focus on in the future. “It’s a real privilege to think about not just what we’re doing but the people we’re doing it for and how we’re doing it,” Karla explains.

s the number of decentralized clinical trials (DCTs) continues to grow, the US FDA, the US Office for Human Research Protections, Harvard’s Multi-Regional Clinical Trials (MRCT) Center, Medable, and other industry stakeholders have collaboratively developed a new toolkit to streamline and accelerate IRB/Ethics Committee review of DCT applications. Medable Chief Science Officer Pam Tenaerts and Harvard MRCT Director Barbara Bierer explore the strategic and practical benefits of this new toolkit with DIA Head of Science and Scientific Strategy Courtney Granville in this DIA Global Annual Meeting 2023 interview.

he US Food and Drug Administration has increasingly recognized the importance of diversity and representation in clinical trials over the past few decades. This evolution reflects growing awareness across medicine that differences in age, gender, race, and ethnicity can impact disease risk, treatment response, and health outcomes. A review of the relevant legal and regulatory history helps document this growing awareness and points the way to further necessary advances to make clinical research truly available to all and representative of all.

uravit Clinical Research Business Development Manager Natalia Husby explains the skillset and shares advice that has helped her discover her role as a woman in the life sciences industry. “It’s exciting to see more and more women moving into leadership positions in science. Thankfully, I had professors who were women who were mentors to me,” she says. “But on the business side, you’re seeing women CEOs of biotech companies and the opportunity of going into different avenues within science. It doesn’t have to just be in the lab as a research scientist.”