White Papers

  1. Could The Gut Microbiome Revolutionize Medical Care? 10/25/2019

    There is still much to learn about whether a dysregulated gut microbiome causes disease, or whether disease leads to dysbiosis. Despite the scientific questions that remain to be investigated, several biotechnology companies have emerged with a focus on testing various approaches to developing therapies targeting the gut microbiome. This paper provides a summary of the current gut microbiome therapy development landscape and an overview of the commercialization challenges faced.

  2. The Future Of REMS: The Time Is Now For REMS Training And Professional Development 10/25/2019

    Implementing and managing a REMS program takes a distinct set of skills and expertise. Manufacturers often struggle, however, to find an organizational “home” for those responsible for administering these programs, whose roles are often spread across pharmacovigilance, drug safety and regulatory affairs functions. Read how to better prepare associates for their REMS managerial and supporting roles, and ways to bring greater awareness to REMS program design, implementation and assessment as a specialized discipline.

  3. REMS Modernization Can’t Wait: A Call to Action 10/25/2019

    Since the Food and Drug Administration Amendments Act (FDAAA) was signed into law in September 2007 REMS programs have evolved in both scope and complexity but the use of new technologies to relieve the burden a REMS can place on prescribers, pharmacists, other healthcare providers (HCPs) and/or patients has not kept pace. Read more about the current state of REMS implementation today — what’s working and what’s not — and where to go from here to optimize the success of REMS by more effectively balancing risk mitigation with stakeholder burden.

  4. Just-in-Time Site Activation: An Innovative And Efficient Strategy For Studies Of Targeted Oncology Therapies 10/23/2019

    Targeted oncology therapies are found only in a small fraction of patients, making it extremely difficult to identify eligible participants for clinical studies. Sponsors who rely solely on the timeworn method of asking sites that have performed well in the past to find patients in any given new study run the risk of drawing out the recruitment phase of their trials and adding substantially to their development costs. It is critical to identify from the outset the most appropriate strategies for recruiting patients. Just-in-time site activation is an approach that has proven successful in studies of targeted therapies/rare diseases and is worth considering as a solution.

  5. Returning Study Results To Research Participants 10/16/2019

    Studies show that most clinical trial participants want to know what was learned from their involvement. A growing number of sponsors are implementing plans to deliver plain language summaries to trial participants.  Read this white paper to learn about the current best practices for the content and preparation of plain language summaries, and the current guidance for how sponsors should work with Institutional Review Boards (IRBs) which have oversight of the clinical trials for which the summaries are provided.

  6. Virtual Clinical Trials Best Practices 10/16/2019

    In recent years, alongside the increase in patient involvement in the design and conduct of research programs, there has been a notable increase in the idea of “virtual” or “decentralized” clinical trials.  This paper examines the nature of virtual clinical trials, potential benefits and risks of this new paradigm, and best practices for maintaining regulatory and Institutional Review Board (IRB) compliance.

  7. Clinical Trial Recruitment Practices: The Evolution Of Ethical Considerations 10/16/2019

    Recruitment practices for clinical trials have evolved significantly over the last few decades. Read this white paper to learn about the evolution of acceptable recruitment practices, and what IRB members typically look for when reviewing recruitment materials and practices.

  8. Rare Disease: Operationalizing Gene Therapy Trials 10/9/2019

    Even measured against the vast scientific mystery that defines the biotech industry, gene therapy poses extraordinary challenges. This paper explores the history of gene therapy trials, as well as the types of gene therapy vectors and delivery strategies. Also discussed is the regulatory and operational challenges associated with gene therapy trials, including start-up regulations, site selection, recruitment, and retention.

  9. Summary, Critical Details, And FAQ For Your TMF Management 10/9/2019

    The EMA’s final “Guideline on the content, management, and archiving of the clinical Trial Master File (paper and/or electronic)” provides stakeholders with guidance on TMF creation, management, and archival. This overview summarizes and interprets the EMA’s “Guideline on the content, management, and archiving of the clinical Trial Master File (paper and/or electronic).”

  10. Registry And Natural History Studies: Vital, Contrasting Roles In Rare Disease Clinical Research 10/2/2019

    Both registry studies and natural history studies play important roles in rare disease research. Understanding the differences between the two types of studies and how they can be used to inform clinical development can help sponsors plan for success.