From Concept to Clinic: The Arduous Path of Bringing Ideas to Life
Have you ever wondered how a new drug, treatment, or medical device gets from the idea stage to the doctor’s office, where it can be used by patients? The time this process takes is called translational lag, and a much-cited study puts that magic number at seventeen years. Because that study was first published over a decade ago, that number may be even higher now. The process that any drug, device, or new treatment must go through, from its inception to clinical practice, is arduous, expensive, and incredibly complex. In fact, because of the difficulty involved, and the high rate of failure, the journey is colloquially referred to by scientists and researchers as the “valley of death.”
The concept of the valley of death is so prevalent and well-known by those in research that it is referred to extensively in scientific literature. A review article published in Translational Medicine Communications explains the problem this way:
“Even with the fascinating observations and creative science, most of the basic scientific discoveries fail to get into the therapeutic development process and often get lost in translation because they are irrelevant to human disease or lack funding, incentives, and technical expertise to advance any further. These potentially important discoveries cannot cross an ever-widening gap in funding and support for the type of research that moves basic science findings down the path toward therapeutic development. This so-called ‘translational gap’ has come to be called by many the ‘Valley of Death.’”
To illustrate the reality of the valley of death, the article cites examples, stating that the process of getting a new drug from first testing to U.S. Food and Drug Administration (FDA) approval and ultimately to market takes more than 13 years, that almost 95 percent of drugs entering human trials fail, and according to the National Institutes of Health, 80–90 percent of research projects fail before they ever get tested in humans. For every drug that gains FDA approval, more than 1000 are developed but fail. The article goes on to say that almost 50 percent of all experimental drugs fail in Phase III trials—therefore, getting new drug candidates from preclinical research to human studies to approval is only approximately 0.1 percent.
Despite the width and breadth of the valley of death, many organizations have increased spending, hoping to boost their odds of success in getting their discoveries to market, but according to the review article quoted above, it hasn’t worked out that way:
“Increases in National Institutes of Health [NIH] spending on biomedical research have not resulted in increases in new treatments and cures. Despite this, early discoveries still remain in what has been called the ‘valley of death,’ the gap between bench research and clinical application. To address this issue, there has been significant discussion in the literature and scientific community about the causes of this phenomenon and how to bridge the abyss.”
Why Is It Important? Everyone involved in the process wants to make sure that discoveries in biomedical and health research make it through the research and development process (translational research) so that the findings can be adopted and used in clinical practice. The problem is that such a long drawn-out process means a lower rate of return on the research investment, making research less attractive to do in the first place. Longer lags also mean patients don’t get access to medications, treatments, and diagnostics that might benefit them. There are, however, benefits to having the time to assess the risks of any new treatment or medication, like potential side effects, adverse reactions, toxicity, injuries, or harms the treatments may cause—and sometimes, those things take time to become clear. While we don’t want to rush new treatments, drugs, or devices to market without proper testing to ensure they’re safe and that they work, the present lag seems exorbitant. Such an extended process implies that new discoveries that could be vital to helping sick people recover, diagnose issues, or improve outcomes are not getting to them quickly enough. The problem is not a lack of new ideas, innovations, or breakthrough technologies in medicine—it is the process they go through and all of the hurdles that must be overcome to succeed. And then there is the cost. The inefficiency of the process means that it is increasingly expensive to get new medical innovations (especially drugs) from research to clinical practice, and all involved parties are concerned with recouping as much of the money spent on research and development as possible. This means that now, many treatments (primarily drugs) that are being developed are the ones that will be the most profitable and not necessarily the ones that are the most useful or needed. This may explain why we see what seems like an ocean of new drugs being approved (with a 0.1 percent approval odds, imagine how many didn’t make it!) and precious few dietary therapies, lifestyle modifications, or natural treatments. Such treatments may simply not be profitable enough to justify their development, which in the landscape of treatments that can improve health, is a problem.
Can the Process Be Optimized? So great is the need to close the gap in this process that there is a new movement called “translational research” working to optimize and speed it up. The Bronfenbrenner Center for Translational Research has a short video describing what translational research aims to do and the way it is trying to streamline the process of how things go from the concept stage into the hands of the people who need them—which will ultimately cut down on translational lag. According to the video, translational research aims to connect realms that have traditionally been separate, like community practitioners, researchers, and policymakers. If health care practitioners working with patients in the community could tell researchers the greatest needs of their patients, it would help them choose research that would lead to the most impactful results for the people who need them. Similarly, if policymakers inform researchers of their needs, it could help researchers choose a path that has the best chance of succeeding. If all the involved parties were connected and communicating, it would not only streamline the process but make the types of treatments that are developed more valuable and effective for the people they are meant to help.
Final Thoughts An article published in Science Translational Medicine called “Translational Medicine: An Engine of Change for Bringing New Technology to Community Health” proposes what it would take to solve the problem of translational lag and get treatments and technologies into the hands of the patients who need them. For each area they have outlined the following:
1. NIH—Small increments in CTSA [Clinical and Translational Science Award] funding over the long-term.
2. Pharmaceutical companies—A culture shift out of the laboratory and into the community setting.
3. Medical schools—More training programs focused on the commercial aspects of medical research.
4. Academic medical centers—Dedicated outreach programs up- and downstream, to pharmaceutical companies and the community.
5. Community health centers—Recognition that they are a lynchpin in the process of translating research from bench to bedside.
6. Patients—Awareness that they are the most crucial stakeholders of all and that they need to be active participants, not passive recipients, in the health care system.
For patients—the consumers who will use these new technologies, diagnostics, and treatments—the last statement in the list above is the most important. Once patients realize they are a vital piece of the puzzle and that they should make their needs and desires known, the future of medicine may be more about treatments people want and need instead of the ones that make the most profit.
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