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PM360: How COVID Has Reshaped the Clinical Trial Regulatory Landscape

The concept of patient centricity is hardly new, but in recent months it has assumed new urgency as industry and regulators have had to adapt to an evolving landscape. The evolution is spurring substantial changes in how clinical trials are developed and implemented, as regulators revise their processes and issue updated guidance documents to encourage speed and efficiency while still ensuring patient safety.

The U.S. Food and Drug Administration (FDA) has been at the forefront of recent changes, notably through its issuance of Emergency Use Authorizations (EUAs) to facilitate the availability of testing kits, treatments, and vaccines; the establishment of its Coronavirus Treatment Acceleration Program; and the authorization of reference panel comparative data to enhance diagnostic capabilities. Over the past year and a half, the FDA has also issued 13 guidance documents, some of which have been updated to address various aspects of product development and clinical trials, reflecting a renewed focus on the patient journey in the wake of the pandemic.

Read more at PM360

PharmaTimes: Keeping Pace

In recent years, the gene therapy regulatory landscape has evolved from a broad, general guidance to a narrower focus on testing and manufacturing considerations in specific disease states. This article explores how factors such as environmental risk assessment and biosafety can impact gene therapy clinical study start-up as well as the continuing evolution of guidances from the European Medicines Agency (EMA) and US Food and Drug Administration (FDA).

Tracking the complexity in the global regulatory landscape

The regulatory landscape in the United Kingdom (UK) and European Union (EU) is increasingly complex, reflecting the advancement of gene therapy research. Aside from the EMA, a gene therapy product will have to undergo review by individual member states’ competent authorities (CAs), ethics committees (ECs), and/or genetically modified organism (GMO) authorities. To ease the complexity, the EMA has created the Committee of Advanced Therapies (CAT), whose responsibilities include providing scientific recommendations on the classification of advanced therapy medicinal products (ATMPs) as gene therapy medicinal products (GTMPs), somatic cell-therapy medicinal products (sCTMPs), tissue-engineered medicines (TEPs), or combinations thereof.

Read more at PharmaTimes.com

PharmaLive: Overcoming the Placebo Roadblock on the Path to Novel Analgesic Drug Development

Placebo response is one of the most significant challenges faced by drug developers who are investigating new pain medications. According to a review of published chronic neuropathic pain trials, placebo responses have increased in magnitude over time, making it even more difficult to definitively demonstrate treatment advantage.1 Research has also shown that up to 60 percent of study participants may experience placebo-related analgesic responses and that these responses may be persistent.2

As we know from our own work and the research of others, the placebo response is a recognized psychobiological phenomenon that must be factored into any chronic pain clinical trial with a symptom-based approach. Generally, chronic pain studies are designed under the assumption that the placebo effect accounts for approximately 30 percent of the analgesic response.3In this article, we provide an introduction to the latest placebo considerations for sponsors of chronic pain studies, including strategies for developing robust placebo-controlled analgesia clinical trials and tips for reducing response bias and variability.

Read more at PharmaLive.com

Applied Clinical Trials: The Rise of Gene Therapy: Advanced Regulatory, Site, and Enrollment Considerations

Gene therapy development is gaining momentum. In 2020 alone, gene therapy attracted $12 billion in global financing through just the first three quarters with more than 370 gene therapy clinical trials underway worldwide.1 The U.S. Food and Drug Administration (FDA) itself predicted it may be approving 10 to 20 gene therapies per year by the year 2025.2As gene therapy research expands across modalities and therapeutic areas, sponsors are tasked with designing and executing safe, ethical trials in a fast-changing regulatory environment.

In this article, we explore key regulatory, site, and enrollment considerations for gene therapy trials, offering tips on successfully navigating the development process based on evolving best practices and our own experience in this area.

Read more at AppliedClinicalTrials.com

Applied Clinical Trials: Navigating the New World of Dermatology Trials

Dermatology clinical research is evolving rapidly in response to a regulatory landscape that favors clinical trials that are safer, more justifiable, and less burdensome to study participants. Putting the patient at the center of every phase of the research process — from discovery and preclinical testing to clinical trials and post-marketing studies — can help accelerate the path to successful commercialization.

In this article, we explore how a strategic patient-focused approach to planning and implementing dermatology clinical trials can help to bring new therapies to market.

Read more at AppliedClinicalTrials.com

TrialSite News: Joining the Revolution in Dermatology Drug Development: Tips for Successful Clinical Trials

Skin diseases are the fourth leading cause of health afflictions, affecting nearly 900 million people worldwide. It’s not surprising, then, that there has recently been a noticeable increase in dermatology drug development. There are currently thousands of dermatology clinical trials planned or already launched, including a variety of programs investigating innovative, targeted therapies.

Much of the development progress in dermatology, however, has been made in a small subset of diseases, such as melanoma, psoriasis, and atopic dermatitis. There is still a significant unmet need for safe and effective treatments for the vast majority of the more than 3,000 known skin disorders.

Read more at TrialSiteNews.com

TrialSite News: Going Virtual: How to Implement mHealth and Remote Monitoring in Your Clinical Trials

In the year since COVID-19 brought all normal activity to a halt, clinical trial professionals around the world have been forced to re-examine their strategies. What happens when neither patients nor clinical research associates can visit trial sites? How can data collection and data monitoring occur — and critically, how can researchers deliver clean, concise results that meet regulatory approval standards?

One simple solution: Maximize the usage of mobile health (mHealth) devices and remote monitoring to collect and manage data. 

Read more at TrialSiteNews.com

Clinical Data Trends: Transforming Data Access/Analysis

The clinical trial industry has been steadily undergoing a data evolution. Data from remote capture devices, such as wearables and other novel sources (e.g., increasingly large health data repositories and electronic healthcare data), have gained importance. Proper management and utilization of such data allows sponsors to untether from a set roster of trial sites, focus directly on the patient, and create an ecosystem that relies more prominently on remote patient monitoring. Furthermore, with increasing accessibility to these new and emerging data sets, sponsors now have the opportunity to design nontraditional studies.

Read the full article at Life Science Leader March 2021.

Long-Term Gene Therapy Follow-Up: Patient Considerations

When developing a long-term follow-up strategy for gene therapy trials, safety is the main concern, closely followed by the patient perspective. Digital technologies and monitoring have proven essential to this, easing the burden on the patient, and providing accurate, time-saving methods of data management.

Momentum is increasing in the field of gene therapy, with over 20 products approved and more than 2,000 clinical trials underway worldwide (1). By the year 2025, it is expected that the FDA will be approving 10 to 20 gene therapies per year (2). While gene therapy offers the potential for a cure, it also affects long-acting or permanent changes in the human body. This prolonged period of exposure can increase the risk of delayed adverse events. To understand and mitigate this risk, sponsors may be required to monitor participants of gene therapy trials for a long-term follow-up (LTFU) period of up to 15 years. During this extended period, sponsors will be challenged with not only navigating regulatory requirements and covering the cost of ongoing monitoring but also keeping patients engaged with their studies.

This article is taken from International Clinical Trials February 2021, pages 65-67. © Samedan Ltd

Outsourcing-Pharma.com: Holistic Approach Benefits Drug Development

Professionals focused on drug development are charged with making the most of their resources, maximizing the value of their work, and bringing effective products to market as quickly as they can without compromising quality or safety. It is a long list of responsibilities, and development professionals struggle to balance all of them.

Sameena Sharif, senior vice president of product strategy for Regulatory Professionals, a division of contract research organization (CRO) Premier Research, recently spoke with Outsourcing-Pharma about the benefits of taking a holistic approach to building drug development programs. Adopting such a strategy and philosophy can streamline the process and lead to better outcomes, she said.

“There are a couple of approaches that I think people are leveraging today, or have been heading toward,” ​she told us. “One such approach is leveraging the one drug, one target, and the second is utilizing a multi-target drug discovery strategy. Traditional drug discovery platforms are based on the backbone of the disease targeting a particular aspect of the disease, a receptor, upstream or downstream​.”

Read more at Outsourcing-Pharma.com