Contributor: Steven M. Solomon, DVM, MPH, Director, Center for Veterinary Medicine
The U.S. Food and Drug Administration’s (CVM) Center for Veterinary Medicine (CVM) plays a critical role in promoting the development and availability of innovative medical products for animals. Our role in promoting public health drives us to take steps that can facilitate access to safe, effective and innovative products to address existing, new and emerging animal health challenges. The FDA takes a proactive approach and uses all of our available tools and flexible processes to provide timely and efficient testing approaches to ensure safety and efficacy and to bring these innovations to market.
The FDA today released four final guidance documents demonstrating our commitment to innovative approaches. The guidance documents describe ways for veterinary drug sponsors to use new approaches such as adaptive study designs, real-world evidence and biomarkers to assess drug efficacy, and more detailed guidance on how to use data collected abroad to approve their products in Supporting the United States Guidance documents encourage veterinary drug sponsors to consider these innovative approaches as they prepare for regulatory evaluation and review to be submitted as part of the FDA’s New Veterinary Medicines Approval Process. The recommendations in these guidance documents are closely aligned with those already issued by the other FDA medical device centers.
CVM is also committed to identifying and using alternative methods to bring innovative products to market by promoting the use of novel approaches to meet regulatory standards. The typical process used to demonstrate the effectiveness of a drug is to conduct a clinical trial; However, there are other ways to prove that a veterinary medicine works and alternative study designs that can be used if the standard design doesn’t fit the situation.
Steven M. Solomon DVM, MPH
For example, CVM evaluated a number of data sources showing the safety of ThyroKare in dogs, a drug used to treat hypothyroidism, as an alternative to conducting laboratory animal studies. These sources included (1) a comprehensive review of the public literature on levothyroxine use in dogs; (2) Pharmacovigilance data for ThyroKare that was voluntarily reported to Sponsor when it was previously marketed as an unapproved drug; and (3) reporting accidental overdoses of natural or synthetic thyroid hormone products in dogs to an animal poison control center.
In 2018, the CVM approved Experior (Lubabegron Type A Medicinal Product), the first veterinary medicinal product to release less ammonia as a by-product of their waste when fed to beef cattle under certain conditions. The drug sponsor collected data in special environmental chambers in which the gas emissions could be contained and precisely measured. These studies showed that the product partially reduced ammonia gas emissions from manure from an individual animal or an animal pen under semi-controlled conditions in closed stables.
In addition, novel products such as deliberate genomic modifications (IGAs) in animals and products based on animal cells and tissues have the potential to treat or even cure diseases or conditions for which treatment options are limited. The areas of regenerative veterinary medicine (e.g. stem cells) and IGAs in animals are fast growing sectors of industry and an active area of animal health research. While the same laws and regulations apply to all other veterinary medicines for these fast-moving products, in 2019 the FDA launched the Veterinary Innovation Program (VIP) to enable advances in the development of innovative animal products. The goals of the VIP are to provide more security in the regulatory process, promote development and research, and support an efficient and predictable path to the approval of cell products and IGAs in animals. In September 2021, more than 40 veterinary medicinal products were registered for the VIP program. IGA animals have potentially significant and broad uses in the advancement of animal and human health, including developing animal models of human diseases, manufacturing human pharmaceuticals, producing organs / tissues for xenotransplantation, improving animal health / husbandry, and the improved food production. Given the uniqueness and developing science associated with these products, the FDA uses a science and risk-based review approach to assess the type and amount of information needed to support approval.
In December 2020, CVM approved a first IGA of its kind in a range of domestic pigs known as GalSafe pigs that can be used for food or human therapeutics. This is the first IGA in an animal that the CVM has approved for both human consumption and as a source for potential therapeutic uses. This approval underscores the FDA’s success in modernizing our scientific processes to optimize a risk-based approach that drives cutting-edge innovations that consumers can trust. The developer of GalSafe pigs was the first product to be approved under the VIP program, demonstrating the effectiveness of this initiative in helping the sponsor throughout the product evaluation process.
The FDA also helps support innovative product developments by engaging lawmakers to increase drug availability for smaller animal species or minor uses in major animal species while ensuring that critical standards continue to be met. The agency does this, among other things, through the conditional approval route for new veterinary medicinal products. Conditional approval was introduced in 2004 under the Minor Use and Minor Species (MUMS) Animal Health Act, or for use in any major animal species (horses, dogs, cats, cattle, pigs, turkeys, and chickens) affected by a rare disease or disease (minor use) after the medicine has been shown to be safe and there is a reasonable expectation that it will be effective.
Recently, this program helped facilitate the development and approval of certain innovative cancer drugs for dogs. For example, in January 2017, the FDA approved the first conditional drug to treat lymphoma in dogs, and that drug was fully approved as the first conditional veterinary drug in July of this year. In January 2021, the FDA conditionally approved the second drug and first oral tablet for the treatment of lymphoma in dogs. The ability to use the MUMS conditional approval route provided access to critical treatments for canine lymphoma before evaluating all of the efficacy data typical for a full approval was completed.
In line with the goals of reducing, replacing and / or refining the use of animals in research, CVM is also conducting a study at its research facility to validate an alternative in vitro dissolution model approach to bioequivalence studies for certain veterinary medicinal products. If this study validates the bioequivalence model, this alternative approach will provide veterinary drug sponsors with a science-based method of demonstrating the bioequivalence of certain drugs that does not require dogs to be euthanized as part of the study.
CVM’s flexible, collaborative and proactive approach to the regulation of veterinary drugs has facilitated the availability of much-needed drugs for conditions such as heart failure, hypertension, cancer, epilepsy and pain, and has brought a range of generic veterinary drugs to market to increase competition and Driving innovation. These approaches have also facilitated the adoption of new technologies with one-health implications beyond animal health, such as: We strive to continue working with a wide variety of stakeholders to ensure the efficiency of our testing and approval process for new veterinary medicines and to support innovative approaches that will ultimately help ensure safe and effective veterinary medicines come to market.
For more informations: Innovation in the development of veterinary drugs