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	<title>Regulatory Corner</title>
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	<pubDate>Tue, 14 Apr 2026 07:37:25 -0400</pubDate>
	<ttl>60</ttl>
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		<title>Substantial Evidence of Effectiveness – Are We Ready for One Pivotal Study?</title>
		<description><![CDATA[Let’s start by going into the way back time machine. It was in 1962 that Congress amended the Food, Drug and Cosmetic Act (FD&#x00026;C Act) to require that drugs be effective as well as safe prior to approval for marketing. The law required that effectiveness of a drug was to be established by substantial evidence, defined as “adequate and well controlled investigations.” The standard FDA interpretation has been that “investigations” means more than one study. Thus, the requirement for two adequate and well-controlled studies. In 1997, Congress amended the FD&#x00026;C Act to make it clear that FDA may consider data from one adequate and well-controlled study and confirmatory evidence sufficient to establish efficacy.]]></description>
		<pubDate>Tue, 07 Apr 2026 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/substantial-evidence-of-effectiveness-are-we-ready-for-one-pivotal-study/</link>
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		<title>The Perils and Pitfalls in the Development of Novel Combinations of Cancer Drugs</title>
		<description><![CDATA[Those of us working in drug development for several years have likely, at one time or another, focused on the requirements for the development of a combination product. They are listed in 21CFR300.50 Fixed-combination prescription drugs for human use; a regulation dated March 27, 1975 and amended on January 5, 1999. FDA also issued a guideline in 2013, Codevelopment of Two or More New Investigational Drugs for Use in Combination (the 2013 Codevelopment Guidance) that further expands on the information in the regulation.]]></description>
		<pubDate>Mon, 30 Mar 2026 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/the-perils-and-pitfalls-in-the-development-of-novel-combinations-of-cancer-drugs/</link>
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	</item>
	<item>
		<title>Do No Harm – Importance of Overall Survival in Randomized Oncology Clinical Trials</title>
		<description><![CDATA[In August 2025, FDA issued the draft guidance, Approaches to Assessment of Overall Survival in Oncology Clinical Trials. This guidance is based in part on discussions held at a joint FDA, American Association for Cancer Research (AACR), and American Statistical Association (ASA) public workshop held in July 2023. It also puts into writing feedback that I have received from the Agency on oncology clinical trials and guidance that I have related to our oncology clients.]]></description>
		<pubDate>Tue, 06 Jan 2026 00:00:00 -0500</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/do-no-harm-importance-of-overall-survival-in-randomized-oncology-clinical-trials/</link>
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	<item>
		<title>What is FDA’s Authority to Revoke an Approval and How Does this Work?</title>
		<description><![CDATA[Continuation of marketing approval of a product differs between the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). Marketing applications approved by the EMA are valid for 5 years and must be renewed to continue marketing of the product. If conditional approval was granted by EMA, a yearly reassessment is required. In the US, the situation is quite different. Once an application is approved by FDA, this approval continues unless the company requests that FDA withdraw the application or the FDA initiates a withdrawal process.]]></description>
		<pubDate>Mon, 01 Dec 2025 00:00:00 -0500</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/what-is-fdas-authority-to-revoke-an-approval-and-how-does-this-work/</link>
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		<title>Optimizing Dosage in Oncology - More May Not Always Be Better</title>
		<description><![CDATA[When FDA published the guidance, Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases in August 2024, I read it with great interest. At first, I thought that it was a draft guideline, as this was the first time I had seen a guideline addressing dose selection in the oncology setting. But I quickly realized that this final guidance implements the information gathered in the FDA’s Project Optimus.]]></description>
		<pubDate>Thu, 01 Aug 2024 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/optimizing-dosage-in-oncology-more-may-not-always-be-better/</link>
		<guid>https://www.prometrika.com/thought-leadership/regulatory-corner/optimizing-dosage-in-oncology-more-may-not-always-be-better/</guid>
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		<title>FDA&#x00027;s Platform Technology Designation - What is it and how can it help me?</title>
		<description><![CDATA[In May 2024, FDA issued the draft guidance, Platform Technology Designation Program for Drug Development. This guidance is applicable to drug products approved through the New Drug Application (NDA) or Abbreviated New Drug Application (ANDA) routes and biological products approved through the Biological License Application (BLA) route.

I found this guidance very interesting and informative. One of the first questions I receive from clients is, “Is this something that is applicable to my program?” The answer to this question has both a good news and a bad news component…]]></description>
		<pubDate>Sun, 30 Jun 2024 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/fdas-platform-technology-designation-what-is-it-and-how-can-it-help-me/</link>
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		<title>Valuable Guidance in FDA’s Considerations for the Conduct of Clinical Trials During Major Disruptions</title>
		<description><![CDATA[When I first read the guideline, Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies, which FDA issued in September 2023, my initial reaction was, ‘do we really need such a guideline?’ We have just gotten over the impact of the COVID pandemic on clinical trials, and business is getting back to usual or normal or whatever we want to call our new normal. That said, I found some interesting and very useful information in this guidance. Having lived through one pandemic, one thing I have learned is that we need to be prepared for the unexpected. While I hope that I will not experience another pandemic, other natural disasters and disruptions in clinical trial are increasingly more likely, especially in our current environment of global clinical studies.]]></description>
		<pubDate>Tue, 30 Apr 2024 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/valuable-guidance-in-fdas-considerations-for-the-conduct-of-clinical-trials-during-major-disruptions/</link>
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	<item>
		<title>Implementing the FDA Guidance on Key Information in Informed Consent</title>
		<description><![CDATA[The new FDA draft guidance, Key Information and Facilitating Understanding in Informed Consent, (March 2024) provides some food for thought regarding the informed consent process and how we should organize our informed consents to best inform potential study participants about the studies we are conducting. I’ve recently read Patient H.M. (Luke Dittrich, 2016), about a patient with severe epilepsy who was treated with a lobotomy in an effort to reduce the seizures. While seizures remained severe, the procedure resulted in eliminating the patient’s ability to establish short-term memory and erased or jumbled most of the patient’s long-term memory. While it was noted that this particular patient did consent to the procedure, the book also chronicles stories of asylum patients with mental disorders who were given lobotomies without their or their legal representative’s prior consent. I am always both amazed and horrified when reminded that these types of research methods occurred in my lifetime.]]></description>
		<pubDate>Sun, 31 Mar 2024 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/implementing-the-fda-guidance-on-key-information-in-informed-consent/</link>
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		<title>Benefit-Risk Assessments for New Drug and Biological Products - Not Just for Marketing Applications Anymore</title>
		<description><![CDATA[I used to think of a Benefit Risk Assessment as part of a marketing application. However, FDA’s recent guidance, Benefit-Risk Assessment for New Drug and Biologic Products, published in October 2023, clearly highlights the role that Benefit-Risk Assessments play throughout the lifecycle of a product, not only at the time of New Drug Application (NDA) or Biologic Licensing Application (BLA) review.]]></description>
		<pubDate>Thu, 29 Feb 2024 00:00:00 -0500</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/benefit-risk-assessments-for-new-drug-and-biological-products-not-just-for-marketing-applications-anymore/</link>
		<guid>https://www.prometrika.com/thought-leadership/regulatory-corner/benefit-risk-assessments-for-new-drug-and-biological-products-not-just-for-marketing-applications-anymore/</guid>
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	<item>
		<title>A Tale of Two Clinical Programs</title>
		<description><![CDATA[In this blog, I will be referencing data in the public domain to compare and contrast the studies submitted to FDA to support label expansions for two marketed products and the similarities and differences in the FDA review and outcomes. By way of disclosure, PROMETRIKA has not worked on either of the two products discussed and all information presented is in the public domain (the October 5, 2023 Oncologic Drugs Advisory Committee (ODAC) FDA slides and ODAC vote for the first and the April 1, 2022 Clinical Review and Evaluation and Statistical Review for the second).]]></description>
		<pubDate>Wed, 31 Jan 2024 00:00:00 -0500</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/a-tale-of-two-clinical-programs/</link>
		<guid>https://www.prometrika.com/thought-leadership/regulatory-corner/a-tale-of-two-clinical-programs/</guid>
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	<item>
		<title>The M11 Template: Clinical Electronic Structured Harmonised Protocol (CeSHarP)</title>
		<description><![CDATA[As a full-service CRO who helps many sponsors develop their clinical strategy and the associated study protocols, the release of an International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) protocol template is of great interest to PROMETRIKA. Step 2 of the ICH harmonized guideline on the clinical electronic structured harmonized protocol (CeSHarP) occurred in September 2022. This draft guidance was released for public consultation in December 2022, together with technical specifications and an electronic template. The guidance provides detailed information to be included in each section of a clinical protocol, expanding on the information currently included in the ICH GCP guidance. For those of us working in this regulated industry, it is a welcome addition.]]></description>
		<pubDate>Tue, 12 Dec 2023 00:00:00 -0500</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/the-m11-template-clinical-electronic-structured-harmonised-protocol-cesharp/</link>
		<guid>https://www.prometrika.com/thought-leadership/regulatory-corner/the-m11-template-clinical-electronic-structured-harmonised-protocol-cesharp/</guid>
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	<item>
		<title>Navigating FDA&#x00027;s Guideline on the Use of Real-World Data and Real-World Evidence in Support of New Indications</title>
		<description><![CDATA[The Cures Act of December 2016 added section 505F to the Federal Food, Drug, and Cosmetic Act (FD&#x00026;C Act), requiring FDA to create a framework for the use of real-world evidence (RWE) in regulatory decision-making. In response, the draft FDA guideline entitled, “Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products,” issued in August 2023, provides very interesting and useful information for consideration in the planning of a marketing application (NDA/BLA) for a drug or biologic product based on RWE.]]></description>
		<pubDate>Thu, 09 Nov 2023 00:00:00 -0500</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/navigating-fdas-guideline-on-the-use-of-real-world-data-and-real-world-evidence-in-support-of-new-indications/</link>
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	<item>
		<title>The Pathway to Approval for Two Gene Therapy Products</title>
		<description><![CDATA[Earlier this year, the FDA granted approvals for two noteworthy gene therapy products. In one case, the product received accelerated approval with a review time of approximately 9 months and included an advisory committee meeting during the review cycle. The other product received full approval after a much longer review time that included a complete response letter and resubmission of the application.]]></description>
		<pubDate>Thu, 26 Oct 2023 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/the-pathway-to-approval-for-two-gene-therapy-products/</link>
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		<title>Harmonizing Investigational and Post-Marketing Safety Reporting</title>
		<description><![CDATA[The safety of patients receiving drug or biologic products is paramount in both the investigational and post-marketing settings. Preparing routine required safety updates can be an arduous and time-consuming process with different data lock points (DLPs), Reference Safety Information (RSI), and reporting timeframes for clinical studies and post-marketing reports. This month, I thought I would take a step back and talk about periodic safety reporting in both the investigational and post-marketing setting and how one can streamline the process. This blog will focus on routine required reports, not expedited reporting.]]></description>
		<pubDate>Tue, 05 Sep 2023 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/harmonizing-investigational-and-post-marketing-safety-reporting/</link>
		<guid>https://www.prometrika.com/thought-leadership/regulatory-corner/harmonizing-investigational-and-post-marketing-safety-reporting/</guid>
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	<item>
		<title>Establishing Efficacy Based on a Single-arm Trial (SAT)</title>
		<description><![CDATA[As many of us are well aware, while single arm trials (SATs) historically have been accepted by the US FDA as primary evidence of efficacy for marketing approval in the setting of accelerated approval (AA) of new oncology agents/indications, their acceptance in other therapeutic areas including rare diseases has been more selective.]]></description>
		<pubDate>Tue, 01 Aug 2023 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/establishing-efficacy-based-on-a-single-arm-trial-sat/</link>
		<guid>https://www.prometrika.com/thought-leadership/regulatory-corner/establishing-efficacy-based-on-a-single-arm-trial-sat/</guid>
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	<item>
		<title>FDA Accelerated Approval of Oncology Therapeutics Guidance</title>
		<description><![CDATA[Draft Guidance - Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics

This guidance issued in March 2023 has been the subject of much talk. While in my opinion, it does not provide any new thoughts on the part of FDA regarding how they handle requests for approval using the accelerated approval (AA) pathway, it does put in writing the policies they have implemented in recent reviews and communications with sponsors.]]></description>
		<pubDate>Fri, 14 Jul 2023 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/fda-accelerated-approval-of-oncology-therapeutics-guidance/</link>
		<guid>https://www.prometrika.com/thought-leadership/regulatory-corner/fda-accelerated-approval-of-oncology-therapeutics-guidance/</guid>
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	<item>
		<title>The Role of Regulatory Affairs in Drug Development</title>
		<description><![CDATA[Participation of Regulatory Affairs professionals is integral to the timely and successful development of all therapeutic products. With our new offering of global Regulatory Affairs Consulting, we are strategically poised to provide fully integrated services from early development to marketing application approval to post-approval support. ]]></description>
		<pubDate>Thu, 01 Jun 2023 00:00:00 -0400</pubDate>
		<link>https://www.prometrika.com/thought-leadership/regulatory-corner/the-role-of-regulatory-affairs-in-drug-development/</link>
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