As clinical development of an investigational product proceeds, Sponsors often conduct global clinical trials which require preparation of content to support dossiers in multiple geographies, meeting the regulatory requirements of each region. The major markets follow ICH guidelines, which affords harmonization of a significant portion
Analytical Target Profile The analytical target profile (ATP) concept was first introduced in the ICH Q141 Guideline in 2022. This guideline, which compliments the ICH Q22 Guideline, describes science and risk-based approaches for developing and maintaining analytical procedures suitable for the evaluation of the quality
In 2022, ICH Q14 officially introduced the enhanced approach1 in analytical development as an alternative path to the minimal approach, the benefits of which are worth exploring further. Analytical data is essential in demonstrating the safety and quality of pharmaceutical products and therefore the data
Adherence to cGMP regulations assures the identity, potency, purity and quality of biologics or advanced therapeutics and helps ensure adequate control of manufacturing operations. A primary way to demonstrate these attributes for a biologic product under development is through assays. Stages of Assay Development Design
The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC)[1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics. The analytical package, consisting of release, stability, and characterization tests,
The pre-IND meeting is an opportunity for highly productive interactions between sponsors and the FDA, allowing for discussion of key material attributes and development strategy plus requests for Agency review and advice on specific topics. In general, such meetings are multidisciplinary and cover the regulatory,
During the development of new small molecule drug products, developers must conduct impurity and degradant evaluation at several points in the program and to varying degrees. These evaluations include the active pharmaceutical ingredient (API), also known as the drug substance, and the drug product (formulated
In this second of our two-part series, we continue our discussion about significant recent developments regarding the regulation of ophthalmic products and discuss what to expect in the months ahead. Unsurprisingly, the U.S. Food and Drug Administration’s (FDA) released updated guidance following the landmark Genus
The global ophthalmic drugs market size was valued at $33.81 billion in 2022 and is expected to grow approximately eight percent from 2023 to 2030[1]. Given this trend, many biopharmaceutical companies are investing in strategies to develop and commercialize new or existing ophthalmic products that
For researchers who are developing or working with analytical procedures, the new ICH Q14 Analytical Procedure Development and revised ICH Q2(R2) Validation of Analytical Procedures drafts represent foundational guidance documents. Together, they describe suggested development and validation activities for the lifecycle of an analytical procedure
For researchers who are developing or working with analytical procedures, the new ICH Q14 Analytical Procedure Development and revised ICH Q2(R2) Validation of Analytical Procedures drafts represent foundational guidance documents. Together, they describe suggested development and validation activities for the lifecycle of an analytical procedure
During the development cycle of a regulated therapeutic, the transfer of the manufacturing process is inevitable. The process will be transferred from the development lab to a pilot or small-scale manufacturing facility and, if the program is successful, to a facility for commercial manufacturing. With
The technology transfer of a manufacturing process to a contract development and manufacturing organization (CDMO) is a key step on the road to developing and commercializing biopharmaceutical products. Successfully executing this step is critical to clearing regulatory expectations, avoiding production delays, and delivering drugs to
One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). PDUFA VII, as the reauthorization is commonly known, will cover the fiscal years 2023-2027. Many of the advancements included in PDUFA
The drug development process is a long journey, beginning with drug discovery, moving through nonclinical and clinical studies, and ultimately culminating in regulatory approval. With many steps in between, each as important as the next, multiple factors regarding development strategy and approach must be considered
The ultimate goal of any drug development program is to obtain regulatory approval with the desired prescribing information. Beyond that, developers hope to obtain this approval in the shortest amount of time and with the most efficient use of resources. In an article published in
Technology transfer is a critical step in any drug development program, occurring for various reasons and at different development stages. A sponsor may be looking for a contract development and manufacturing organization (CDMO) for clinical trial manufacturing; to transfer a manufacturing process to a larger,
This week the FDA released a new draft Guidance for Industry entitled “Post-approval Changes to Drug Substances” as part of the FDA’s commitment to the reauthorization of the Generic Drug User Fee Amendments (GDUFA II). The new draft Guidance fills an important void as the
Liposome drug products frequently contain an already-approved drug, and can utilize the 505(b)(2) pathway to approval. Liposomal drug products are a sub-group of Non-biological Complex Drugs (NBCD) and contain greater complexity in characterization and approval than small-molecule drugs. Further, their generic follow-on products, ‘nanosimilars’ or
One of the main causes of Refusal to File/Receive actions by the US FDA is due to inadequate Chemistry, Manufacturing, and Controls (CMC) data. This missing data relates to formulation issues, incomplete stability data, and inactive ingredients listed above the limits found in the Inactive
Hurry up and wait. That’s the seemingly eternal impact of developmental stability on the new drug development process. The question always asked is: “How can the developer minimize the wait part?” At the top level you can’t. It will always take six months to get
The FDA recently announced it has made corrections, updates, and additions of a backlog of formulations to the Inactive Ingredient Database (IID), an important modification for drug developers and development. The previous state of the IID had become an issue as excipient suppliers recommend the
At the recent GPhA meeting in Orlando, Florida, Dr. Kathleen Uhl from the FDA Office of Generic Drugs (OGD) spoke about the quality of Abbreviated New Drug Application (ANDA) submissions and highlighted the detriments of a poor quality submission. All would agree that a poor
The Food and Drug Administration (FDA) began requiring drug efficacy, in addition to safety, for approval in 1962 based on the Kefauver-Harris Amendment. Despite this requirement, many drugs that have been approved by FDA have limited efficacy (eg, drugs that treat cancer or Alzheimer’s disease).
Extrusion-enabled pharmaceutical processing (E2P2) has long been employed in pharmaceutical development (Drug Development and Industrial Pharmacy. 33:909-926,1043-1057 (2007)). Reasons for utilizing E2P2 include: Enhancing physico-chemical properties of APIs such as solubility and stability Modifying in vivo release of a drug to enhance bioavailability and clinical
In November, this author participated in an open forum at the AAPS Annual Meeting focused on streamlining manufacturing and scale up to support ‘breakthrough therapy’ designation for solid oral dosage forms. The goal was to provide clarity for breakthrough therapies and chemistry, manufacturing and controls (CMC) challenges
After much delay, FDA just released the new guidance on the stability requirements to file and obtain approval of a generic drug product and API under 505j. The new requirements bring ANDAs closer in line with NDAs and ICH. The new requirements as summarized in
How many drug development companies leave it up to the CMO to design or execute their formulation and manufacturing without oversight? Like those who boarded the Titantic 100 years ago, they seem to trust the mantra that their contractor’s work is unsinkable. MAP Pharmaceuticals seemed
The Quality Module (Module 3 or Chemistry, Manufacturing and, Controls section (CMC)) in the eCTD format serves as the backbone of any regulatory submission, an IND or NDA. We have discussed this in a previous blog post. There is debate about the usefulness of the
From a technological standpoint, it is understood that the goal of developing 505(b)(2) abuse-deterrent products is to make changes to the API or formulation of a drug that has abuse potential in such a way that it dissuades the abuser from wanting to use the
In any project development program an understanding of the program goal is critical to finding the shortest path to the final result. Generation of a Target Product Profile early in a development program facilitates reaching the goal of a marketed drug product. It provides a
In the past two months, two appellate courts, the Fifth Circuit and the Eighth Circuit have handed down decisions which essentially state that generic pharmaceutical companies can be sued in state courts for failure-to-warn regarding serious side effects, where the generic companies had conformed their
Analytical requirements for the NDA submission of an oral solution to the FDA are very similar to those requirements for any new drug product. The analytical methods that are used for the testing of an oral solution at the NDA stage of development should be
What do you need to do when you need to change suppliers or manufacturing sites? Among the many choices is a formal FDA comparability protocol. Advance planning can improve the possibility that the FDA accepts your proposed change. One alternative that can streamline the process
Pivotal stability programs that are used to generate stability data for NDA submissions are different than research stability programs used to design the drug product, explore packaging configurations, etc. This is common sense, but we have seen instances of pivotal stability programs that have been
Premier Consulting is often called on to write and/or assemble NDAs. When we get to prepare an NDA from the beginning, all of the information builds and the resulting story is easy for the FDA to understand. Often we are retained to write portions and
Developing a product with multiple strengths? How do you go about filing multiple strengths in an IND? How and when do you draft one CMC section covering multiple drug strengths for the same dosage form? Long-term drug development goals may include multiple strengths of a
We typically work with small molecules of synthetic origin, but occasionally are retained to work with products that have active ingredients from botanical (plant) sources. Lynn Gold, our VP of CMC provides the following discussion on how to define the starting material for the Active Pharmaceutical Ingredient (API).
Today’s posting stems from a client question. The client’s product candidate is an oral product that requires both single- and multiple dose pharmacokinetic studies. Question: Do companies ever use one pivotal batch for single-dose (SD) study and another batch for the multi-dose (MD) study? What
A draft label be included with all NDA submissions. For a 505(b)(2) NDA, where do you get the information for this label? What labeling is required? What is labeling? Well, the “label” is what is on the immediate container of the drug product and can
