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RMAT Designation…What is it and is it Right for Your Therapeutic Product?

As research teams develop more cellular and gene therapy products for the treatment of a wide array of medical conditions, the need for custom nonclinical and clinical programs required to support market approval of products that are based on human cells, tissues, or HCT/P will rise. And for RMAT designated products, the study designs may be substantially different from more traditional IND programs.

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Blog Post

Antiviral Products in the pre-IND Phase

Productive pre-IND interactions with FDA are important to the success of any drug development program, including infectious disease product development. The Pre-IND Consultation Program through the Office of Infectious Disease (OID), one of 27 review divisions within the Office of New Drugs, provides opportunities for makers of infectious disease products to engage with FDA by facilitating early communications between the Division of Antivirals or the Division of Anti-Infectives within OID and sponsors/investigators of potential new therapeutics – these products include drugs and therapeutic biologics for the treatment of bacterial, fungal, and viral infections (including SARS-CoV-2 infection).

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Blog Post

Considerations for Implementing Decentralized Clinical Trials

The COVID-19 pandemic has accelerated the implementation of decentralized (also referred to as virtual, remote, or hybrid) solutions for clinical trials. In addition to keeping your clinical trials on-track during the pandemic, decentralized solutions can often provide added benefits, including an improved patient experience by increasing engagement and reducing patient burden. We will discuss some considerations for successfully implementing decentralized solutions.

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Blog Post

COVID-19 Treatment Development: Updates and Recent FDA Guidance

The FDA continues to release additional information and update their website as the COVID-19 pandemic continues and both regulatory professionals and drug developers react to the potential for novel and repurposed products to treat COVID-19. This blog post is the latest installment in a series of COVID-19 related blog posts and updates information provided in a recent webinar.

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Blog Post

The Sunsetting of Rare Pediatric Disease Designation

As with other incentive programs, FDA created the Rare Pediatric Disease (RPD) designation to encourage drug development in products with questionable financial viability; in this case, the treatment of certain rare pediatric diseases. One of the main benefits of RPD designation is the potential to receive priority review vouchers, which can be used to obtain priority review on a subsequent human drug or biologic application, should the product with RPD designation be approved.

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Blog Post

Maintaining Trial Integrity During COVID-19: Some Statistical Rules of Thumb

The COVID-19 pandemic is having a substantial impact on many ongoing clinical studies in all phases of product development. Numerous difficult decisions are being made and steps are actively being taken to ensure the safe execution, or future resumption, of ongoing studies. While patient safety is paramount and should drive all study conduct related decisions, many of these decisions can impact the interpretability of estimates of efficacy at study conclusion.

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Blog Post

505(b)(2) vs ANDA: How Complex Drugs Fit In

While it is important to understand how to navigate the complex generic drug approval pathway, it is first necessary to determine whether your drug product should be submitted as an abbreviated new drug application (ANDA) for approval as a generic or if it requires submission of a 505(b)(2) new drug application. This particular issue is addressed in a draft guidance “Determining Whether to Submit an ANDA or a 505(b)(2) Application.” Our blog will help you understand when a 505(b)(2) application is appropriate and when an ANDA is appropriate.

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Blog Post

Four Considerations for Rare Disease Drug Development Programs

Rho representatives joined regulators, industry scientists, and numerous patient-advocacy groups at CBI’s Rare Disease Clinical Development and Access Summit in Washington. Attendees were able to share best practices in product development for rare disease programs in formal presentations and through informal networking. Here are 4 considerations for your development program that were highlighted during the conference.

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Blog Post

Update from the top: FDA’s Office of New Drugs ongoing reorganization process

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.

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Blog Post

Is a Target Product Profile Worth the Effort?

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.