Making a transition from a life and health science research scientist to a regulatory affairs professional can be challenging. It is difficult to gain the requisite experience in an academic or industry research setting. However, the combination of an advanced science degree, transferable skills, and professional experiences can make this transition attainable.   Intellectual curiosity is often the impetus for pursuing a doctorate degree (PhD). The desire to learn a su...

Clinical development planning can be thought of as the integrated design and structuring of the steps from the first phase 1 study for the initial assessment of safety, to one or more phase 2 studies for better understanding the product’s pharmacology, safety, and effectiveness, to a phase 3 definitive demonstration of efficacy and safety. Optimal strategy can be informed by creating a vision for the product in the form of a target product profile, examining precedence (wh...

The US Food and Drug Administration (FDA) on Monday announced it will pilot a new program it hopes will spur the development of novel drug development tools (DDTs) that do not fit within the agency’s existing DDT qualification programs.   The qualification process for DDTs was revamped under the 21 st Century Cures Act and FDA currently offers specific qualification programs for biomarkers, clinical outcome assessments (COAs) and animal models for use under the Anim...

Continued progression and understanding in the development of advanced therapies has led to a significant increase in the number of products and types of indication under investigation, particularly for treating serious and life-threatening conditions. Although the principle regulatory requirements and guidance, as issued for traditional biologic products, can be generally applied, advanced therapies require additional regulatory and strategic considerations because of the...

The US Food and Drug Administration (FDA) on Monday announced it will not begin collecting drug manufacturing volume data next month as required by the Coronavirus Aid, Relief, and Economic Security Act (CARES Act).   Section 3112 of the CARES Act establishes new reporting requirements for drug manufacturers intended to improve the response to drug shortages, including expanding shortage reporting requirements for life-saving drugs to include drugs that are “critical...

This article concerns the development of advanced therapies and the challenges and complexities of getting them through the developmental pathway, which the authors call “the trip.” The authors examine at strategic levels the importance of some of the fundamental building blocks for the development program and highlight some commonly encountered challenges (trip hazards) for cell and gene therapies and offer “bench-to-bedside” and chemistry, manufacturing, and controls (CM...

Feature articles during May focused on risk management and mitigation in dealing with contractors and vendors and included articles on best practices in good vendor management, use of risk management to support outsourcing activities, testing in-house versus outsourcing, and outsourcing in regulatory operations. Also included were in-depth examinations of challenges and opportunities in “bespoke” therapies, a critical appraisal of drug shortages in Germany and an update on...

This article discusses the advent of bespoke therapies, defined as the tailoring of medical treatment to the individual characteristics or symptoms and responses of a patient during all stages of care and as a new frontier beyond personalized medicine. The author covers the revolutionary genetic tools implementing such therapies and the clinical and nonclinical safety perspectives for bespoke therapies. The author concludes that with bespoke therapies we are entering a new...

This article discusses drug shortages in Germany, the root causes of production problems with active pharmaceutical ingredients, and drug product manufacturers. The authors offer analysis from the perspective of a medium-sized generic drug manufacturer. They outline a range of reasons for the shortages, all of which point to a “broken marketplace.” The authors conclude with a discussion of mitigation activities and address the current COVID-19 pandemic. They warn that drug...

Two House committees on Thursday advanced on party-line votes Speaker Nancy Pelosi’s (D-CA) bill to allow for the negotiation of certain medicine prices as part of plans to bring down costs in the US. No major amendments were adopted in either the Energy & Commerce or the Education & Labor committees’ meetings, although the amendments in the E&C meeting ranged from carving out specific disease treatments from negotiations to requiring the Health and Human Services Sec...

For each of the last five years, the US Food and Drug Administration (FDA) has set generic drug approval records, but questions have lingered on whether these high approval numbers are translating into competition. FY 2019 figures show a total of 1,171 generic drug approvals (935 full approvals and 236 tentative approvals), which breaks FDA’s previous all-time record of 971 full and tentative approvals for FY 2018 . And although the abbreviated new drug application (...

The Congressional Budget Office (CBO) late Friday announced that House Speaker Nancy Pelosi’s (D-CA) drug pricing bill would reduce federal direct spending for Medicare by $345 billion from 2023 to 2029, but it would also lead to a reduction of approximately 8 to 15 new drugs coming to market over the next 10 years. The CBO report comes as rhetoric on both sides of the aisle has picked up in recent weeks, with industry group PhRMA referring to the bill, known as HR 3, a...