When it comes to dermatologic conditions, gene therapy is still in its very early stages. However, we are seeing promising potential solutions for some rare genetic dermatology diseases, as well as ongoing research in more common skin conditions. The success of gene-based therapies relies on three critical elements:1 A well-defined disease geneA therapeutic geneAn efficient...
Summary The Research to Accelerate Cures and Equity (RACE) for Children Act The Research to Accelerate Cures and Equity (RACE) for Children Act aims to improve and expand treatment options for pediatric cancer patients by mandating that all new adult oncology drugs also be tested in children when the molecular targets are relevant to a...
As a regulatory affairs professional advising various stakeholders on the interpretation and implementation of guidance documents from the FDA, I can safely say that the task is challenging in the best of cases. FDA guidance documents have a tendency to be vague and difficult to decode. As experts, we are always debating about what the...
Gene therapy holds the potential of long-term efficacy or even a cure for diseases with an underlying genetic basis, offering hope to patients with currently incurable diseases. The use of gene therapy for dermatologic conditions is attractive for many reasons:1 As the largest organ of the human body, skin is easily accessible for gene deliveryThe...
As those working in the development of combination products likely know, the Food and Drug Administration (FDA) issued two new draft guidances in December 2019. The guidances – Requesting FDA Feedback on Combination Products and Bridging for Drug-Device and Biologic-Device Combination Products– were published in response to the 21st Century Cures Act and Prescription Drug...
In October 2018, the Food and Drug Administration (FDA) issued Standard Operating Policy and Procedure (SOPP) – SOPP 8214 Version 1.0: INTERACT Meetings with Sponsors for Drugs and Biological Products. This document details recommendations for the Center for Biologics Evaluation and Research (CBER)’s engagement with sponsors (such as industry and investigators) in an early development...
Innovation in oncology drug development is being driven by “precision medicine.” Precision medicine promises a new paradigm in oncology where every patient receives truly personalized treatment. This approach to disease diagnosis, treatment and prevention utilizes a holistic view of the patient—from their genes and their environment to their lifestyle—to make more accurate decisions. Growing at...
For medical device developers seeking to enter the diverse, complex Asia-Pacific (APAC) market, having a well-planned reimbursement strategy is critical for commercial success. Thoughtful consideration of coverage, coding, and payment should be factored into the product development cycle at its earliest stages, and understanding the reimbursement landscape in all the countries in which you intend...
Unlocking the Full Potential of Precision Medicine in Oncology Precision oncology promises a new standard of care where therapies are tailored to the molecular profile of a specific tumor. For the full potential of precision medicine to be realized, regulatory, technical, clinical, and economic frameworks will need to evolve to the nuances of these novel...
“Big pharma’s new role in this is to partner and fund the innovation that it so sorely needs to bulk up its dwindling pipelines. This is a cost-effective move for larger drug companies to leverage outside scientific talent to gain access to breakthrough discoveries. “‘This is a dominant trend in the industry,’ says Nach Davé,...