Regulatory Strategy & Submissions Articles and Insights

What is the Type D meeting? The FDA introduced the Type D Meeting on October 1, 2022, as part of the Prescription Drug User Fee Act (PDUFA) VII Commitment Letter. This new meeting is designed to make it easier and faster for sponsors to get

All clinical trial or marketing applications submitted to the FDA must include a form that summarizes the content of the submission and any relevant information on the sponsor and drug for the reviewers. Periodically, the FDA updates these forms to enhance their usability and incorporate

The 505(b)(2) pathway can yield significant benefits in drug development cost and time. But what are the differences between 505(b)(1) and 505(b)(2)? Drug development pathways in the United States are referred to by their corresponding section in the Food, Drug, and Cosmetics Act: 505(b)(1), 505(b)(2),

A clinical hold from the U.S. Food and Drug Administration can significantly prolong the time and increase the cost of drug development, a particular concern for emerging and small biotech and specialty pharma companies. In this blog post, we discuss common reasons for clinical holds

When a submission is sent through the FDA’s Electronic Submissions Gateway, it goes through an automated check of many validation rules as specified in the Electronic Common Technical Document (eCTD) submission standards guidance. This validation check determines if the submission is uploaded to the FDA’s

When pitching to investors, a sponsor is not just selling them on the product; it is also promoting its ability to develop that product. Investors want to know the risks involved with the program, but they are also evaluating the sponsor as a risk factor

Preparing to meet with the FDA can be challenging and stressful for sponsors, especially when they do not know what to expect. Many have misunderstandings about how meetings should be conducted and what constitutes success. Others find themselves needing to dispel incorrect preconceptions about this

Drug development is a resource-intensive endeavor. Seeking input from the U.S. Food and Drug Administration throughout the journey can help optimize those resources and maximize the likelihood of regulatory approval. When preparing to submit a new drug application, a pre-NDA meeting with the FDA can

The Electronic Common Technical Document (eCTD) is the standard format for submitting applications, amendments, supplements, and reports to the Food and Drug Administration. The eCTD harmonizes the regulatory review process for global drug development, as its structure is based on the International Council for Harmonisation

As the COVID-19 national emergency stretches on in the United States, the eyes of the pharmaceutical industry are trained on developing diagnostic, therapeutic, and preventive measures to combat the novel disease. Given the significance of the pandemic and the critical need, the FDA has developed

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

On January 31, 2020, the Secretary of Health and Human Services (HHS) declared a national emergency to combat the COVID-19 pandemic caused by the novel coronavirus SARS-CoV-2. As a result, the FDA is authorized to grant Emergency Use Authorizations (EUAs) for any medical countermeasure (MCM)

During these challenging times when the world is focused on COVID-19 and the matters at hand, it’s understandable if you didn’t take much notice of March 23 as a significant date – one that’s been on the FDA’s radar for a while. March 23, 2020,

In response to the Coronavirus Disease 19 (COVID-19) pandemic, the scientific community, industry, and regulatory agencies are pulling together to facilitate the development of diagnostic tests, drugs, and vaccines to help prevent the spread of the disease. The timelines for development of new drugs and

Transdermal and topical delivery systems (TDS) are important dosage forms that allow delivery of a drug to local tissue or provide systemic delivery through the skin. These drug products provide a number of advantages for patients, but can be challenging to develop. In November 2019,

If you are planning to submit a New Drug Application (NDA) for an oncology drug product in 2020, revised pediatric study plan requirements could have an impact on the submission. FDA recently released draft guidance on changes to pediatric study requirements put in place by Section

Breakthrough therapy designation will speed the development and review of your 505(b)(2) product Breakthrough therapy designation is the most recent addition to the suite of expedited programs offered by the FDA. Compared to fast track designation (discussed in a prior blog post), the qualifying criteria for

One of the tenets of the FDA is to get safe and effective drugs to market as soon as possible. To expedite approval of products for which there is the greatest clinical need, the FDA offers four expedited programs to get beneficial therapies to patients

For centuries, botanical remedies have been used in many cultures to treat various diseases. In recent years, pharmaceutical companies have turned to botanical drugs for alternative medicines to treat diseases with unmet medical needs. The FDA also recognizes the interest in developing botanical products and

We decided to mention some noteworthy firsts from the FDA during August 2018 and a change in the FDA’s policy on obtaining orphan incentives via pediatric-subpopulation designation. Competitive Generic Therapy Designation The FDA granted Competitive Generic Therapy (CGT) designation for the first time for Potassium

The increasing complexity of brand-name drugs has made development of generic alternatives more challenging as well. As complex generic drug products can provide a high-value opportunity for drug development companies, they are similar to 505(b)(2) products in that they may require early and flexible interaction

Last month the US District Court for the District of Columbia ordered the FDA to grant Eagle Pharmaceuticals, Inc., 7-years of marketing exclusivity for Bendeka® (bendamustine). This was the second loss for the FDA regarding orphan exclusivity for the ‘same drug’ that is not clinically

Innovative Thinkers: FDA wants YOU FDA just released a very brief (barely three pages of actual text) guidance promoting innovation in new drug applications (NDAs) involving nonprescription (aka over-the-counter, OTC) drug products. In the guidance, “Innovative Approaches for Nonprescription Drug Products, Guidance for Industry’ (CDER

Updated on: February 9, 2021 Updated by: Stacey A. Ayres, PhD, VP of Regulatory and Strategy While the analysis in this blog post was conducted in 2018, the takeaway still rings true today: CRLs can have a devastating impact on emerging biopharma development programs, and

The recent key decision by the FDA for Exparel® stresses the importance of reevaluating 505(b)(2) strategy throughout a product development lifecycle. A solid, data-driven development strategy is important from the beginning; however, new data, including related regulatory actions, must be considered and incorporated to realign

Additional Indications: Advantages of QIDP Designation The benefits of early strategy for 505(b)(2) drug development programs are numerous, but none as substantial as market exclusivity. Incentives available to sponsors of QIDP include fast track designation, priority review designation, and 5 years of additional market exclusivity.

If you think regulatory pharmacokinetics requirements are just a box-checking exercise then you may be throwing away money and time on unnecessary studies. At Premier Consulting, we have always focused on innovative approaches for the most challenging drug development programs, but our cutting-edge pharmacokinetics strategies

A 505(b)(2) with 8 years of exclusivity. That is the prize that Calla Therapeutics will receive upon approval of its innovative vaginal cream drug candidate for the treatment of recurrent vulvovaginitis (RVVC). Premier Consulting helped Calla obtain Qualified Infectious Disease Product (QIDP) designation for its

Changing the marketing status of a drug from prescription (Rx) to over-the-counter (OTC), known as an Rx-to-OTC switch, can increase drug utilization by an average of 30% (Stomberg et al. 2013). According to the Consumer Healthcare Products Association (CHPA), more than 700 current OTC products

With a recent update from the United States Food and Drug Administration (US FDA), the quest for abuse-deterrent opioids has taken another step. But will the new Guidance lead developers forward or backward? We previously commented on the Draft Guidance for generic abuse-deterrent opioids in

Finally there is hope for Sponsors of complex generics with the new Pre-ANDA program outlined in GDUFA II. More good news: the 505(b)(2) experts have got you covered for the development of complex generics. Until now, Sponsors of complex generic products either submitted an NDA

While the old adage “It’s easier to beg forgiveness than to ask permission” might have worked well as a child, it rarely applies when dealing with the FDA. The FDA recently published a final guidance titled “Formal Dispute Resolution: Sponsor Appeals Above the Division Level”,

Earlier this year, the U.S. Food and Drug Administration (FDA) announced its Drug Competition Action Plan, which aims to bring more competition to the drug market and improve consumer access to drugs. As part of this effort, the Agency is focusing on ways to help

Gaining approval of new indications for approved or “old” drugs has always been one of the great advantages of the 505(b)(2) regulatory pathway. Couple that with the FDA’s recent enthusiasm for using real world evidence (RWE) to expand a drug’s indications, and you have a

Drug or Device? – FDA provides more clarity – or does it? Industry has complained for years, and for good reasons, that it is difficult to understand FDA’s determination of whether a combination product would be reviewed as a device or a drug. So, in

Product Selection and the Importance of Early Strategic Design for Success Why do some new drug products gain approval, but launch with lower-than-anticipated drug sales? Why then can some drug products gain approval and launch and perform well commercially? Is it possible to align a

Sponsors spend countless hours developing Investigational New Drug (IND) applications, which are the US FDA’s regulatory gateways for conducting clinical trials of investigational drug and drug-device combination products. The stakes are high for companies as they submit their initial IND, as the ability to start

From a distance, 505(b)(2) product development can seem very straightforward for products that have been on the market or in clinical use for long periods of time. However, these types of products can often present the greatest challenges when trying to modify or improve performance.

At Premier Consulting, we are often asked for strategic advice on drug development programs seeking approval in not only the United States (US), but also approval from other regulatory agencies, including the hybrid application in the European Union (EU). It is useful at the beginning

Updated on: January 26, 2021 It is a story all too common: A drug product is developed, approved, and launched, only to flop in the marketplace. There are various reasons that such products underperform or fail, but frequently the cause is a lack of commercial strategy to help sponsors develop a roadmap to follow from the start through development, approval, and launch. While a focus on FDA

As the 505(b)(2) experts, Premier Consulting has received several enquiries about developing deuterated drugs as a means of achieving sustained-release properties for a product. The approval of Austedo (deutetrabenazine; Teva Pharmaceuticals, Inc.; NDA 208082), marked the first FDA approval of a deuterated drug. A recently

The requirements for abuse-deterrent opioids are in flux as the FDA grapples with what they can do to address the ongoing opioid epidemic. As part of their efforts in promoting the development of abuse-deterrent opioids, the Agency has required that manufacturers demonstrate that their product

The provision of Written Responses Only (WRO) by the FDA in response to Pre-IND meeting requests is here to stay. While rarely requested or preferred by Sponsors, this meeting response format is being used with increasing frequency by many Divisions at the FDA since the

The first step for every wise drug developer beginning a drug development program is to determine the feasibility of a proposed product by asking several high-level questions: Is the product scientifically, medically, and commercially viable? What are the regulatory requirements and potential hurdles? What nonclinical

One of the greatest mistakes the sponsor of a 505(b)(2) product can make is to have an unsuccessful pre-IND meeting. Errors often occur at the pre-IND meeting because sponsors and CROs that are more familiar with traditional 505(b)(1) development programs fail to appreciate the different

Patient-reported outcomes (PROs) provide valuable tools for collecting information on subjective symptomatic effects during clinical trials. They are considered the gold standard for the assessment of health-related quality of life, treatment preferences, and satisfaction with care. PRO results from a well-defined and reliable PRO instrument

In the past, after gaining approval for a drug/device in the United States, subsequent approval in Australia involved significant duplicated effort and additional regulatory hurdles. This has resulted in a lack of incentive for pharmaceutical companies and delays of up to 2 years to bring

On March 23, 2010, the U.S. FDA enacted the Biologics Price Competition and Innovation Act of 2009 (BPCIA) as part of the Patient Protection and Affordable Care Act (Public Law 111-148). Under BPCIA, applications for biological protein products, which were previously approved as New Drug

The Food and Drug Administration is responsible for ensuring that human drugs are safe and effective, while also advancing public health by helping to speed product innovations. In determining if a drug should be marketed, the Agency must weigh the benefits of the therapy against

On March 23, 2010, the U.S. FDA enacted the Biologics Price Competition and Innovation Act of 2009 (BPCIA) as part of the Patient Protection and Affordable Care Act (Public Law 111-148). The passing of BPCIA amended the definition of a “biological product” to include a

With the ongoing opioid epidemic, drug abuse, diversion, and misuse are significant concerns for the FDA. The current opioid abuse problems also present significant opportunities for companies willing to explore new formulation technologies to deter abuse. Premier Consulting has significant experience in opioid abuse and abuse-deterrent

Premier Consulting is known for its expertise in the 505(b)(2) pathway. But, in a global pharmaceutical business, many clients are looking to develop drugs for both the US and EU markets. We’ve written before about the EU process that is similar to the U.S. 505(b)(2)

The Biologics Price Competition and Innovation Act of 2009 (BPCIA) was enacted on March 23, 2010 as part of the Patient Protection and Affordable Care Act (Public Law 111-148). The BPCIA changed the regulation of certain protein products by amending the definition of a “biological

As the 505(b)(2) expert, Premier Consulting is frequently asked questions about how to get a product approved via the 505(b)(2) regulatory pathway and if this pathway is appropriate. Given the growing popularity of the 505(b)(2) pathway for approval of repurposed, reformulated, or unapproved-marketed products, we

An idea for a more convenient dosage form for an existing drug product often presents an opportunity for a commercial advantage. Fortuitously, it also presents the possibility for using the 505(b)(2) regulatory pathway to product approval, which is often faster and less expensive than the

As the 505(b)(2) expert, Premier Consulting is frequently asked questions about how to get a product approved via the 505(b)(2) regulatory pathway and if this pathway is appropriate. Given the growing popularity of the 505(b)(2) pathway for approval of repurposed, reformulated, or unapproved-marketed products, we

Traditional drug development follows a standard process beginning with nonclinical studies and moving into clinical studies, all with the purpose of proving a new drug candidate is safe and effective. When the U.S. opened up an alternate path, 505(b)(2), the primary purpose was to utilize

Strike While the Iron is Hot In December 2015, the U.S. FDA granted approval for Eli Lilly and Company’s Basaglar (insulin glargine injection), a long-acting human insulin product indicated for glycemic control in patients with diabetes mellitus. Basaglar marked the first “follow-on” insulin therapy to

If you are a frequent reader of our blog, you know that the 505(b)(2) pathway can facilitate a cheaper, faster drug approval route. This is accomplished by relying on: 1) safety and efficacy data from published literature and/or 2) the agency’s assessment of safety and

On July 2, 2015 the House passed the 21st Century Cures Act . The focus is on expediting research and development on debilitating diseases, and smoothing the process to get important therapeutic options to patients who need them quickly. The Senate has not yet approved