Enables the passage of scientific discovery and innovation into a product which may have human application.
Where: academia, academia spin-off companies, research hospitals or contract research organizations.
Ideal managers: individuals with natural or health sciences backgrounds and formal translational biomedical research management training.
Advances new safe products to population-based studies and ensures their readiness for regulatory approval by the Food & Drug Administration.
Where: hospitals or contract research organizations.
Ideal managers: individuals with natural or health sciences backgrounds and formal translational biomedical research management training.
Integrates data from post-marketing surveillance and adverse event reports and creates strategies for optimized utilization and cost-effectiveness.
Where: hospitals, health maintenance organizations, accountable care organizations, big pharma or contract research organizations.
Ideal managers: individuals with social or health sciences backgrounds and formal translational biomedical research management training.
Identifies patient- and system-related opportunities for improvement and channels them back to the innovation bench creating new context for discovery.
Where: academic center hospitals or academia in partnership with health maintenance organizations, accountable care organizations or contract research organizations.
Ideal managers: individuals with social or health sciences backgrounds and formal translational biomedical research management training.
Small biotech companies are typically startups or academia spin-offs focusing on early commercial development of a single health product. The professionals involved are often the original contributors to the idea, discovery or innovation. Such companies can navigate early phase trials faster and at lower costs due to reduced bureaucratic burdens, lower indirect costs, and a lack of prior losses.
Early phase clinical trials involve the testing of new product safety (phase I) and efficacy (phase II) on human subjects and dose optimization. Phase I studies generally enroll small numbers of healthy volunteers and thus rarely yield evidence regarding efficacy or effectiveness. Phase I studies are also shorter, minimizing subject exposure. Consequently, results may lack statistical significance which is more thoroughly addressed in subsequent phases (II through IV). Roughly a third of the new therapies fail based on phase I data.
Phase II trials evaluate new health product effectiveness and side effects, often enrolling over 100 individuals. Participants will have the condition or risk the new product is intended to treat. Despite the longer duration (up to 2 years), a phase II trial is insufficient for approval. Phase II trials document important factual evidence necessary to decide whether subsequent late phase trials are worth pursuing. Roughly two thirds of new therapies tested fail based on phase II data.
Big pharma is essential to translational biomedical research. Large pharmaceutical companies enable the necessary investments associated with conducting the late phase clinical trials, obtaining regulatory approvals, and marketing. A few examples are Johnson & Johnson ($276bil market value), Novartis ($273bil), Pfizer ($212bil), Merck ($164bil), GSK ($103bil), Eli Lilly ($98bil), etc. Late phase clinical trials include phase III (before FDA approval) and phase IV (after FDA approval) human studies. Their hallmarks are their large size (several hundred to several thousand volunteers) and extended duration (up to 5 years or more). Phase III trials are pivotal studies providing evidence of clinical benefit regarding the use of a new product in the treatment or prevention of a specific health condition. The associated costs are exceptionally high. A phase III clinical study has a higher chance to detect rare side effects, but only phase IV will provide accurate information regarding these side effects. It is important to note that several phase IV studies will take place after FDA approval and will document additional effectiveness and adverse event information. This occurs under a process called post-marketing surveillance (PMS). Not all phase IV studies are PMS, but all PMS studies are phase IV clinical studies.
Following FDA approval, the replacement of established therapies with new drugs is often a slow process. Early adoption occurs sooner among physician specialists as compared to general practitioners. The uncertainty regarding unexpected adverse events plays a huge role. The process of post-marketing surveillance (PMS) begins after FDA approval and is often in its early stages during the early adoption step. Early adopters play a crucial role in identifying new evidence and sharing clinical research reports with the clinical community. The timely and effective dissemination of PMS data from early adopters to professional audiences (conferences and professional journals) is vital in advancing innovation to clinical care.
Several large phase IV studies will take place following FDA approval to document, among other important information, adverse event information. It is generally agreed that the adoption of effective innovations into clinical care is slow and results in suboptimal treatments for some patients. In addition to prescriber concerns, inefficient information dissemination preventing timely research innovation and implementation is a likely cause. This translational research sector attracts the interest of several stakeholders, including hospitals, clinics, pharmacies, insurance carriers and big pharma. Today, pharmacovigilance (a drug-related PMS process) is rapidly approaching a $6bil market. Dissemination is generally driven by medical practice guidelines, clinical journals, and pharmaceutical marketing. The implementation of research innovation in health is currently encouraged by funding from the Department of Health and Human Services.
Late adoption of a new product is common among general practice prescribers and is often due to skepticism before it has been tried and accepted by the majority. This is directly influenced by delays in peer-reviewed reports of clinical research authored by early adopters.
In this segment, the stakeholders involved (hospitals, clinics, professional associations, big pharma, and insurance carriers) gather evidence regarding real-world utilization of a new product by comparison to other similar products. These efforts will provide the following translational research step with the needed data to conduct comparative effectiveness and comparative safety studies. Given the importance for the general public, funding is often provided by the Agency for Healthcare Research and Quality, a division of the Department of Health and Human Services, the Patient-Centered Outcomes Research Institute, as well as professional and patient advocacy groups.
Personalized care is a series of assessments that evaluate both intrinsic and circumstantial patient specifics in the context of objective evidence-based outcomes research data resulting from the analysis of comparative effectiveness and comparative safety studies. Personalized care is integral to optimal healthcare delivery and it may be the only process to enable maximized efficacy. This milestone in clinical care follows and is directly influenced by the translational research conducted in the T3 segment. Although niche eligibility for a new health product may enable utilization of a personalized treatment immediately after the approval of a new drug (i.e. a specific drug given to a specific gene carrier), the term personalized care carries a broader and deeper meaning.
Between 2014 and 2019, the number of new drug applications approved by the FDA has nearly tripled and the number of new medical device approvals has almost doubled. Beyond the extensive pipeline of new molecules (500+ in early phase and 1000+ in late phase development), emerging technologies that enable real time monitoring of human physiology have set the stage for an explosion of new medical device development. This has generated a record demand for individuals capable of effectively navigating the evolving regulatory and research environment that advances health product development throughout the T1 through T4 translational biomedical research segments.