irector Peter Marks explains the benefits of Accelerated Approval and CBER’s START (Support for clinical Trials Advancing Rare disease Therapeutics) Pilot Program in this DIA 2024 interview with Lori Ellis, Head of Insights, BioSpace. “We want to lean into accelerated approval whenever we can, particularly in the rare disease space. Because if you have patients in need, getting something to them a year or two sooner can make the difference, for instance, in a child with Duchenne muscular dystrophy, from the ability to walk or not walk,” Marks said. “Getting there fast is important. We want to get there with good accuracy because we want the confirmatory trials to confirm, but if we do our scientific work well, that will happen.

he life sciences industry faces a constant challenge: balancing rapid innovation with stringent regulatory requirements. In this high-stakes drug development landscape, traditional digital technologies often fall short, as they can be rigid and slow to adapt, making rapid protocol and technology changes a major hurdle. “Headless technology” offers a solution that is both innovative and extremely effective at reducing risks.

atients with cutaneous T-cell lymphomas (CTCLs) may go months or even decades without a proper diagnosis and treatment, experiencing debilitating itch and progression to fungating weeping wounds as they cycle through ineffective therapies. Diagnosis is just one of the many clinical challenges patients face. Fortunately, dynamic partnerships between patients, physicians, and researchers have led to significant advances in the diagnosis and management of this life-threatening disease.

uch has been studied and written about the recruitment funnel in clinical trials—from patient identification to enrollment through completion—and stages where high percentages of patients fall out or discontinue in the process. A recent pilot study helps explain why a very high percentage of identified and interested patients drop out of the funnel early on, prior to completing pre-screening. This article summarizes the results of the pilot study, examines the implications of the “last mile” leak, and highlights the urgent need for further research quantifying the magnitude of the problem and identifying opportunities for new solutions.

n healthcare advancement, ensuring that medical innovations address patients’ needs is paramount. Target Product Profiles (TPPs) are critical tools in product development, aligning regulatory requirements, market needs, and commercial objectives. However, a crucial voice is often missing: that of the patients themselves. By integrating direct insights from people with lived experience and expertise throughout the TPP development process, we aim to catalyze the creation of new diagnostics that directly respond to patient need.

he advent of artificial intelligence (AI) and its application in healthcare has opened new frontiers in medical practice and research. Large Language Models (LLMs) like GPT-4 are at the forefront of this revolution, offering unprecedented capabilities in processing and generating human-like text. When integrated with Digital Health Technologies (DHTs), LLMs can significantly enhance clinical decision-making, patient engagement, and overall healthcare delivery. There are numerous multifaceted applications of LLMs in healthcare, including their integration with DHTs and the implications for clinical and patient care.

f automation is a bullet train, culture and processes are the tracks that will keep pharmacovigilance on course.

he Certificate of Pharmaceutical Product (CPP) is an essential part of almost every dossier submission for most regulators around the globe. No other regulatory document has influenced regulatory discussions and connected regulators globally as profoundly as the CPP. But despite the WHO Scheme’s original goal in 1969 of establishing the CPP as a tool to enable reliance practices and reduce/replace (parts of) the assessment by CPP-requesting National Regulatory Authorities (NRAs), regulatory procedures and CPP acceptance have varied significantly among countries.

ack in 1969, the World Health Organization (WHO) Certificate of Pharmaceutical Product (CPP) was implemented to accelerate the availability of new drugs in developing countries by providing evidence of the quality of products. Nonetheless, there are still differences in the usage of CPPs by regulatory agencies in Growth and Emerging Markets (GEMs) that may lead to challenges relating to today’s more innovative, complex, globalized products.

This article presents an updated overview of previous research developed by the Centre for Innovation in Regulatory Science (CIRS) on how CPPs have been used for the marketing authorization of New Active Substances (NASs) in 18 GEM countries between 2021 and 2022 with a special focus on three aspects: CPP submission timing, CPP legalization, and adoption of eCPP format, considering both guidelines-based agency requirements and current company practices.

he need for regulators to ensure business continuity, make fast and informed decisions, and adapt to a digital environment has driven the adoption of various digital technologies and solutions to enhance regulatory processes. This rapid adaptation and use of digital technologies by regulators was accelerated during the unparalleled challenges posed by the COVID-19 pandemic. In this context, the Certificate of Pharmaceutical Product (CPP) was no exception, leading to the advent of the electronic CPP (eCPP). However, an industry survey shows that many regulators are not currently in the position to use this eCPP, a problem that causes unnecessary delays in the regulatory assessment process.

he recent series of sessions on pharmaceutical regulations and industry practices at DIA’s Southeastern Europe (SEE) Region Regulatory Conference highlighted key areas of development, challenges, and strategic directions for the future of the SEE region. Discussions ranged from the new EU pharmaceutical legislation to reliance practices, harmonization, pharmacovigilance, supply chain security, and digital transformation. This conference underscored the importance of collaboration, harmonization, reliance, and digitalization in enhancing pharmaceutical regulations and practices in the region. By addressing these key areas, the region can improve healthcare outcomes, accelerate EU accession, and ensure the efficient and secure supply of medicines to its patients.

n India, effective risk communication and minimization in the healthcare sector faces unique and multifaceted challenges. Unlike many other regions, India’s vast linguistic diversity, varying literacy rates, and varying accessibility of reliable drug information create significant barriers to ensuring that patients and healthcare providers fully understand the risks associated with medications. This creates a problematic gap where patients may be unaware of potential adverse effects, leading to medication errors, nonadherence, and underreporting of drug safety issues.

n a departure from their approach to previous PMDA Town Halls held at DIA Global Annual Meetings, at DIA 2024 this past June, leaders from the Pharmaceuticals and Medical Devices Agency (PMDA) of Japan invited sponsors and researchers to discover the benefits of developing innovative therapeutic products in Japan, based on Japanese market information and pharmaceutical regulation.

he need to optimize drug development and facilitate faster access for patients has focused discussions on the importance of improving interactions between health technology assessment (HTA) bodies and regulatory authorities. In most cases, once the medicine is authorized, market access is determined by the healthcare system’s financing mechanisms, and payers usually rely on the assistance of HTA bodies who decide whether to reimburse a product based on its relative value under current clinical practice scenarios. This is usually a lengthy process that occurs after regulatory review. Though regulators have been working to reduce review timelines through regulatory reliance and work-sharing procedures, HTA timelines have remained unchanged, and thus the overall benefit of these regulatory reforms to patients is far less significant than it might be. In recent years, however, international collaborative cross-border regulatory and HTA body frameworks have been established to optimize decision making.