What is gene therapy? With over 450 gene therapy and gene-based medicine companies worldwide and around 800 gene therapy and gene-modified cell therapy clinical trials being conducted, it’s time to find out.
What does the FDA’s Office of New Drugs ongoing reorganization process mean for you? Read the latest on what transpired at CBI’s Rare Disease Clinical Development & Access conference in Washington to find out.
Perhaps the greatest value of the TPP is as a communication tool. The ultimate goal of the development program is shared clearly and continually with all the company disciplines, e.g., clinical, preclinical, chemistry manufacturing and controls, regulatory, and marketing. The TPP is also able to be used as an external communication tool that facilitates interactions with regulatory authorities, investors, and the media.
Since the enactment of the Orphan Drug Act in 1983, the number of orphan drug approvals has risen steadily. Rho has received an increasing number of sponsor requests for support of programs working towards an orphan drug approval. Can a product be approved with support from a single trial? To better understand when a single trial approval is possible, we look to FDA guidances.
The 33rd Annual NACF conference continues to be an action packed 3 days bringing together scientists, clinicians, health care providers, and caregivers to discuss the latest advances in CF research, care, and drug development. These are our top 3 takeaways from the meeting and how they will impact clinical research moving forward.
Patient-centricity and family-centricity has been a hot topic for the past few years in clinical research, as more groups in pharmaceutical companies and CROs are working on patient engagement and patient-centric approaches to clinical research programs. Recruitment and retention are one part of patient-centricity, but the more important up-front and overarching aspect of patient-centric research comes from Patient-Focused Drug Development (PFDD).
With the announcement of Project Orbis, FDA’s Oncology Center of Excellence (OCE) excited both patients and the pharmaceutical industry with the program’s future possibilities for collaborative regulatory authority interaction in accelerating product approvals worldwide. Is this the tip of the iceberg for process improvement?
In January 2019, the Food and Drug Administration (FDA) released 2 draft guidance documents regarding assessment of Risk Evaluation and Mitigation Strategies (REMS). . These documents are meant to provide industry additional information to more accurately assess the effectiveness of their REMS.
In September of 2018, FDA released a new FDA guidance on master protocols. So, what are master protocols, how can they benefit sponsors and patients, and what are the challenges? Kevin Barber shares his thoughts and expertise on the topic.
The Common Terminology Criteria for Adverse Events (CTCAE) was developed to standardize reporting of adverse events (AEs). In order to integrate the patient’s perspective into AE reporting, the NCI developed a patient-reported outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE).
Attending PAINWeek this year was especially meaningful because pain research has been a major part of my career. Over the past 12 years, I have worked on more than 50
Staying cohesive through long and complex Parkinson’s disease trials Parkinson’s disease trials, like most clinical trials, are rarely a straight path from start to finish. Protocols evolve, real-world conditions change,
Six challenges in acute pain trials and one differentiator—coordinated execution We’ve all been there before. Your acute pain trial strategy is sound, the protocol looks solid, and everything progresses as
Acute pain trials start with assumptions. How you execute—when reality sets in—determines the outcome. When you’re designing an acute pain clinical trial, it’s perfectly normal to have some early assumptions
Early in clinical development, it’s easy to focus on what’s directly in front of you. After all, your goal is to hit milestones, generate strong data, and keep the program
Early clinical decisions shape regulatory risk and the path to approval Assumptions about study design and real-world execution don’t stay contained to a single milestone. Over time, they influence
Four scientific principles. One unifying concept. The Cohesion Effect experience. SCOPE Summit is a place where innovation thrives. Thousands of attendees and hundreds of exhibitors from around the world, all
When Tonix came to us, the stakes were high, and the timelines were tight. Their goal? To submit a New Drug Application (NDA) for TONMYA™ and, for the first time
Every CRO out there will tell you they’re site-centric, and on pitch decks, they use the right buzzwords to prove it. Curious. Collaborative. Dependable. Problem-solvers. Sound familiar? That’s great in
You’re responsible for showing risk oversight, protocol clarity, and a site-friendly design—but you’ve got a lean team, a looming trial, and no time to decode the 110-page ICH E6(R3) guidance.
