Feature articles over the past several weeks covered two main themes: global reimbursement strategies and advertising, labeling and marketing. Individual country profiles on England, France, Germany, Italy and Spain took a closer look at reimbursement. A candid interview with two leaders from the Alliance for Regenerative Medicine (ARM) provided insight into future challenges and opportunities for ensuring market access and value-based reimbursement for gene and cell therapies. Other articles covered new guidance for prescription drug promotion, policies and best practices for payor communications and developing advertising and marketing materials for prescription drug labeling, recent FDA enforcement trends and practical guidance related to broadcast advertisements, and the benefits of developing and maintaining a promotional claims compendium.
Global Reimbursement Strategy
Obtaining regulatory approval for medicines is the first major hurdle for most pharmaceutical and biotechnology companies looking to launch their products worldwide. However, obtaining market access once products are approved by regulatory agencies is a separate and crucial process without which patients would not have access to new therapies. Each country has a complex process with varying requirements by various agencies resulting in pharmaceutical companies having to establish product value above and beyond clinical efficacy. Four articles summarized the reimbursement process, presented key stakeholders and highlighted what should be considered early in the development process. Berto presented a profile of Italy, Brougham, O'Neil and Samaha highlighted Canada, Cuervo, Crespo and Ehreth provided their insights into Spain's strategy, Ehreth focused on France, Müller covered Germany and Patel covered reimbursement in England.
Cell and gene therapies represent a new, transformative era in medicine. For many indications, these products offer the promise of durable - even potentially curative – treatments and in many cases, with a single administration. Policy makers today face the challenge of devising and implementing a reimbursement environment allowing widespread patient access, while also supporting and incentivizing continued innovation. Given a highly fragmented, multi-payer system, such as the US healthcare system and the unique nature of cell and gene therapies, this is a task requiring a creative and wholesale adjustment in how medical treatments are valued, paid for and financed. An interview with two leaders from the Alliance for Regenerative Medicine (ARM), Slocomb and Werner, provided insight into future challenges and opportunities for ensuring market access and value-based reimbursement for gene and cell therapies.
Pharmaceutical manufacturers have abundant information regarding approved drugs. Much of this information is not included in the FDA-required or approved labeling. Numerous enforcement letters issued by FDA cite communications aimed at introducing an intended new use to demonstrate how some pharmaceutical manufacturers have questioned FDA and expressed their interest in communicating information not contained in the FDA-required or approved labeling. In his article, "FDA Draft Guidance for Medical Product Communications," Bergstrom focused on the US Food and Drug Administration's (FDA's) draft guidance as it impacts prescription drug promotion and offers examples regarding information considered consistent with labeling. The discussion emphasized the need for regulatory professionals to better understand and adhere to FDA's evidentiary standards by utilizing examples of the Office of Prescription Drug Promotion (OPDP), formerly Division of Drug Marketing and Communications, and enforcement actions that should be considered when interpreting what is "consistent with labeling."
In 1997, the US Congress passed the US Food and Drug Administration Modernization Act (FDAMA) and provided the regulated industry with a "safe harbor" from enforcement regarding off-label promotion when communicating HealthCare Economic Information (HCEI) with an appropriate audience, such as a formulary committee or similar entity. In January 2017, FDA issued a draft guidance offering FDA's current thinking on appropriate communications to payors, formulary committees and similar entities.
In his article, "New Developments in Payor Communications," Byrd discussed the state of this FDA guidance and reviewed the questions requiring clarification in order for industry to be able to effectively change how economic and other clinical information is communicated to payors and similar groups.
While the regulations for advertising and promotion of restricted medical devices are a portion of those for prescription drugs/biologics, the basic tenets of fair balance and truthful, non-misleading communications are primary factors that still apply. The Federal Food, Drug, and Cosmetic Act (FD&C Act) under §502(r) requires this information to prominently accompany the respective promotion. In the article, "Conducting a Successful Advertising and Promotion Review of Restricted Medical Devices," Deutsch provided insight for regulatory professionals regarding developing a successful advertising and promotional review of restricted medical devices She demonstrated how medical device manufacturers and pharmaceutical companies can improve communication and collaboration between regulatory and marketing to streamline and improve the compliance review process for promotional materials and achieve an effective marketing strategy.
During the past nearly 18 months, FDA's Office of Prescription Drug Promotion (OPDP) issued numerous enforcement letters, warning letters and untitled letters. Most of the letters were related to the promotion of approved products, concerns with risk presentation and promotional videos through broadcast TV or YouTube.com. In their article, "FDA Enforcement Trends for Broadcast Advertisements," Mann and Tibbets presented recent FDA enforcement trends related to broadcast advertisements and explained how regulatory professionals can approach the review and approval of promotional materials. The article also provided a review of relevant FDA authorities and guidance, as well as practical takeaways for industry with a focus on product ads appearing on TV or YouTube that may distract viewers from important risk information.
Science and Research
Error prevention is a topic all regulatory and quality professionals should find relevant and applicable in their day to day job responsibilities. In their article, "Mistake-Proofing the Workplace," Sherman and Sherman discussed mistakes or human errors made in the pharmaceutical or medical device workplace and provided the definition of a mistake. The authors also presented how mistakes are made, whether such errors are the actual "root cause" of defects, the social and company consequences of errors, and what can be done to minimize errors.
Feature Articles Coming Throughout October
Topics throughout the month of October will cover health related foods, including how dietary supplements should be regulated, particularly with regard to New Dietary Ingredient (NDI) notifications, a comparison of the legal definitions of drugs and medical food, the distinctive regulatory hurdles for medical food and the need for substantiating distinctive nutritional requirements, the benefits of the Ketogenic Diet (KD) and the Modified Atkins Diet (MAD) as treatments for intractable epilepsy and patient-oriented interpretation of regulatory requirements for medical foods, key provisions introduced by the Nutrition and Supplement Facts Labels rule with a focus on their effect on the development and marketing of dietary supplements and suggestions on how to manage different aspects and potential implications of the new requirements and changes, a European public-private healthcare initiative to implement Optimal Nutrition Care for All (ONCA) for patients, the US regulatory environment for probiotics in food, and a self-regulatory initiative of the dietary supplement industry, the Supplement Online Wellness Library (OWL), its history, and how it will serve regulatory audiences, retailers, and consumers. Look for these feature articles and other topics throughout October. If you have a suggestion for a feature article, contact me at email@example.com.