What One Tool Is the Most Useful in Guiding Clinical Drug Development?

The title question may seem unrealistic: the scientific depth, medical intricacies, high cost, significant resources, and cross-functional logistics needed to bring a molecule from pre-IND through approval involve head-spinning complexity. Yet seasoned drug developers have found that one tool is central to ensuring a focused path through the labyrinth. That tool is the label. At this point, you might be asking how on earth the label could serve this role. Isn’t the label a long list of details, printed on tissue-like paper, written at the very end of phase III to include conclusions from pivotal trials? The answer is yes and no. Here is how the label, officially called Physicians’ Prescribing Information (PI), serves as an indispensible compass. The First PI—It’s Foundational Many companies use essential label components to drive decisions from pre-IND through approval. Market Planning PI (MPPI) is the first draft of options for claims in the eventual PI, typically including the desired indication statement, efficacy outcomes, key safety features and others. This document is based on an assessment of preclinical results, competitive landscape and clinical outcomes needed for a commercially viable, approvable new drug. Typically, there is a MPPI for each major market for the drug (e.g. US, EU, Japan), and each includes key label attributes in several sections: 1. minimum for registration and break-even commercialization, 2. target for success and 3. high commercial success. The reason for dividing into markets is that they vary in regulatory requirements and competitor drugs, and in some cases variations in clinical trials will be required. For example, the minimum case for US efficacy might be statistically significant superiority vs....

The Evolving Biotechnology Regulatory Landscape

From blood products and vaccines to gene markers, gene therapy and RNA interference, the technology of biological based therapeutics is changing at an exponential rate. Challenges for the efficacy and safety of these stem from characterization of a therapy, manufacture, quality control, distribution and use. Regulatory authorities in the USA and around the globe are working to keep up with technology which results in a rapidly changing regulatory landscape. This document is intended to summarize some of the key changes that have occurred over the last several decades. Contents: Primary Regulatory Institutions The Transition from Small Drugs to Biologics Humanization and Engineering of Biologics Biosimilars Elucidation of the Human Genome Companion Diagnostics Gene Therapy and RNA Interference Monoclonal Antibodies Lead New Biotechnology Development Medical Devices and Diagnostics Primary Regulatory Institutions In the USA, federal regulation of drugs and devices including diagnostics begins with the Food, Drug and Cosmetic Act approved by Congress in 1938 and is supported by the US Constitution’s requirement for the Federal Government to regulate interstate commerce. The intent is to protect the American public from products that have limited or no efficacy or significant safety concerns when used as recommended by a manufacture either for the treatment of a medical condition or to aid physicians in making decisions about medical treatment. The FDA is currently structured in several divisions: Center Drug Evaluation and Research (CDER) for drugs, Center for Biologics Evaluation and Research (CBER) biologics, and Center for Devices and Radiological Health (CDRH) for devices including diagnostics. In Europe and internationally, centralized regulation of drugs and devices originated with the formation of the European Agency...

5 Things You Need to Know About Expedited Drug Development

When you are looking for ways to make drug development as effective and efficient as possible, take advantage of all the acceleration options from regulatory authorities. They may be more helpful than you think. The trend is unmistakable Increasingly, FDA and similar regulatory agencies are offering a range of expedited development and review programs, and companies are applying for and receiving the benefits. In the last few years, an average of approximately 60% of recent drug development has been in therapies approved by FDA for at least one of the expedited programs. Of recently approved drugs, about a third qualified for two or more of the programs.1 The options are diverse, and the benefits useful FDA’s four expedited programs for drugs treating serious conditions represent the largest opportunities for most companies. Fast Track Designation may be granted for a qualified infectious disease drug or one with nonclinical or clinical data demonstrating the potential to address an unmet medical need. Breakthrough Therapy designation requires preliminary clinical evidence indicating the drug may demonstrate substantial improvement in a clinically significant endpoint(s) over available therapies. These designations confer more frequent FDA interactions, potential for rolling reviews, and eligibility for accelerated approval and priority review. For Breakthrough Designation, companies receive intensive FDA guidance on their drug’s development program. For the Accelerated Approval Pathway, companies can use and gain approval based on a compelling surrogate endpoint or an intermediate clinical endpoint reasonably likely to predict a drug’s clinical benefit. The Priority Review Designation allows shorter submission review clock of 6 months vs. the usual 10-month standard review. The FDA has developed additional incentives, some quite...

5 Key Facts About EMA

If you are working in the pharmaceutical industry and are used to dealing with the US FDA, looking across the Atlantic might cause some bewilderment and confusion. The European Medicines Agency’s (EMA) place within the EU regulatory landscape, its set-up and workings can be puzzling to say the least, but this feeling should not have a negative impact on your plans for expanding your drug development or regulatory filing strategy to Europe.   Here are 5 key facts that just might improve your understanding of the EMA:   European Union 101 – The set-up You don’t need to be familiar with the intricacies of the set-up of the European Union, but understanding the EMA’s principle role in the EU regulatory landscape is essential. Think of EMA as a virtual agency set up in 1995 (in London) as a scientific consultancy to the European Commission (EC), the EU executive body located in Brussels. As such, EMA has no legal power to grant the actual marketing authorizations for the medicinal products they regulate – the agency’s remit is to do the medical and scientific assessment and to issue an opinion to the EC, who will base their ultimate (legal) decision on this scientific judgment. The goal of this new agency was to pool the best available scientific and regulatory expertise from all EU Member States for the assessment of the most innovative and cutting edge medicinal products. The scientific assessment is done by the 7 scientific committees (most notably the CHMP, but also PDCO, COMP and PRAC etc.) that meet at the EMA’s premises in Canary Wharf, London. However, the expert...