Biopharmaceutical development is an extensive and complex process. Managing processes like Investigational New Drug (IND) applications or the manufacturing, quality, and regulatory side of the processes—collectively known as CMC, or the Chemistry, Manufacturing and Controls—requires a highly skilled team with multiple areas of expertise. For many companies, developing such a team in house can be challenging. Even if they wanted to, finding professionals with the right skill sets to manage each CMC stage can be difficult. Even companies that already have internal experts sometimes have multiple ..read more

Bringing a new drug to market is a complex process that can take years with no guarantee of a successful outcome. The journey is highly regulated and includes numerous processes that require drug developers to have multiple levels of expertise within their teams. One such process is the Investigational New Drug (IND) application with the Food and Drug Administration (FDA). The IND application must include information in three broad areas: animal pharmacology and toxicology studies, manufacturing information, and clinical protocols and investigator information.[1] Any issues that occur during t ..read more

The IND Filing Process and Categories Federal law mandates that drugs have an approved marketing application for interstate transport, but developers of new drugs must distribute their potential products nationwide in order to conduct clinical trials, and so must request an exemption through an Investigational New Drug (IND) application. The FDA processes around 1,500 initial IND applications annually and assesses them primarily to safeguard the safety of study participants, but also to ensure properly designed and conducted clinical trials.  INDs are categorized as Commercial, which cor ..read more

By: Mayuri Mutha, Senior Manager, CMC Development & Project Management Joseph Sclafani PhD, Director, CMC Development & Project Management Drug development is a complex, expensive, and multistage process which could take 10 to 15 years to bring a new molecule from discovery to commercialization. Drug repurposing, also known as drug repositioning paves an alternate path for drug development which involves identifying new therapeutic indications or uses for the existing molecules that could have been initially approved for or have clinically failed in a different indication. The success ..read more

Developing biologics can be challenging. Their development protocols are significantly different from small molecule drugs, which provide a general response and comprise as much as 90% of the pharmaceutical drug market. Biologics are more complex, though, with targeted mechanisms and potent pharmacological impacts. Nonetheless, their rising popularity – eight of the top 10 best-selling drugs internationally in 2016 were biologics – underscores their increasing importance.   5 Key Steps in Large Molecule (Biologics) Development   Target Identification Drug discovery involves identify ..read more

Large molecule drug products produce highly effective medicines, but formulation can be challenging because of their complexity, sensitivity, and potential for instability. It is crucial that formulation strategies ensure their bioavailability and efficacy to develop new and successful products. Protein stabilization techniques Temperature, pH, agitation, and oxidation can degrade the stability of large molecules. Methods for counteracting that potential include pH adjustment, optimizing the pH of the formulation, buffer selection, excipients, and protein engineering. It is critical that pre-f ..read more

By Maria WIk, MS VP, CMC Development The ICH Q10 definition for a Control Strategy is: “A planned set of controls, derived from current product and process understanding, that assures process performance and product quality.” Understanding the essence of a process control strategy directly addresses a critical challenge faced by many in the biologics manufacturing industry: ensuring high-quality products while navigating a dynamic manufacturing environment. Establishing and maintaining a process control strategy are critical steps in biologics manufacturing to ensure that the biological produ ..read more

Biomedical startups experience a failure rate of 90% per year for various reasons, including flawed financial strategies, inexperienced management, subpar science, poor timing, and an aversion to risk. Another significant factor is quality control issues stemming from poor team selection and inadequate time and resources, which also applies to quality control (QC) partners. However, there are three key characteristics that reliable QC professionals bring to the table in a drug development organization’s process of creating new pharmaceutical products. These characteristics significantly improv ..read more

Personalized medicine is truly that. Based on an individual’s unique genetic profile, it’s used to make precise decisions according to that person’s biologically predetermined factors, and guides the optimum approach to disease prevention, diagnosis, and treatment. It determines the most appropriate medications or therapies and administers them. It guarantees delivery of the right drug and the right dosage at precisely the right time, tailored specifically for that person. Cell and gene therapies have been subjects of research for some time, but they have gained traction in real-world medicine ..read more

December 14, 2018 | Kathryn Tworkoski, PhD, RAC, Senior Clinical Research Scientist  │ Regulatory Affairs, Medical Writing, Drug Development Consulting Long-time readers of this blog will recall (with great enthusiasm, I’m sure) our previous posts on rare diseases, INDs, preparation for pre-IND meetings, and the submission and maintenance of INDs.  But in October 2018, the FDA released new draft guidance outlining best practices for early drug development and pre-IND meetings for rare diseases.  Given the near-perfect cross-over potential, we thought this was a sign th ..read more