It has been a very long week! It has been hard to pick out a single subject to talk about because the news is changing by the hour, so I thought I would give some background on a subject I know well–medial research. Many of you out there don’t understand or appreciate how medical research benefits you or why it is important. I am going to write a simple primer on the subject so you can understand the different parts of medical research in our society. Then you can understand how the Trump administration’s cuts might affect it and how that is not in your best interest.
The Trump Administration has put freezes on all federal grants, including devastating freezes on communications, study sections (review committees for grant awards), and hiring at the NIH. Rumors are that they want to cancel funding for any research that mentions diversity and may want to shrink the size of the NIH by eliminating some of the 27 institutes.
Why is medical research important?
All of us have been sick at some time in our lives. Some of us have had simple things like infections which only needed antibiotics for you to feel better, while others might have on-going serious or chronic conditions that have on-going treatments. If you haven’t, then a loved one most likely has had something.
Many think that the government should not be funding this research to begin with, and that the private sector should be paying for it. The problem with this is that the private sector only does one part of medical research, not all of it. Drugs and therapies that come to the market now are based on research from over 20-30 years ago. That’s a lot of investment in R&D that most companies won’t do. If the private sector had to invest in the basic science research that the NIH funds (that we will go over later in the post) then drug prices would be even higher than they are now. Or worse, the research would not be funded or performed due to the long lag in the payoff.
If Trump’s threats come to fruition, then we may not have any new drugs or therapies in the future. We have come so far with new drugs and treatments for cancer, but there is a way to go. Take a look at this article “Hogg, P., The most significant FDA approvals of the decade (2010-2019). Proclinical, Sept 2020” to review many of the new drugs that would not be available today if federal funding for medical research was reduced or eliminated 25 years ago. We would have a world with no gene therapy, no immunotherapy. Have you heard of the BRCA1/BRCA2 mutations? That would not exist. The popular drugs Ozempic and Wegovy would not exist. The human genome would not have been sequenced.
All of the devlopment for these drugs/therapies were based on basic science research that was funded by the government. The new knowledge that was learned about how the body functions and how disease progresses was published by academic researchers. This new knowledge led to the new development of the new drugs and therapies.
The different segments of medical research
Now let’s go over, who does what in each part of the research timeline in drug/therapy development. I am going to give a summary of the focus for each segment in medical research. This does not mean that each are restricted to this definition, but that most of their time and dollars go to these efforts. This is a simplified version which is meant for the lay person and not the scientist.
ACADEMIA
Academia consists of the National Institutes of Health (NIH) and all major universities that do research. They tend to focus on basic science research to understand the biological mechanisms and pathways in the body, with the purpose being to gain knowledge of how the body works and/or how disease permeates throughout the body. The primary goal is to publish the findings and to add to the vast collection of knowledge on disease and the human body. PubMed® is a public database of all the publications that is housed at the The National Institutes of Health (NIH).
PubMed® comprises more than 37 million citations for biomedical literature from MEDLINE, life science journals, and online books. Citations may include links to full text content from PubMed Central and publisher web sites.
Animal models (mostly mice) are typically used in experiments to show cause and effect of disease. Mice are widely used for studying cancer, diabetes, genetic diseases, and neurological disorders due to their relatively short lifespan and ability to be genetically modified.
Academia is not profit driven. Academic medical research is primarily funded by NIH grants with some funding from non-profit foundations specific for each disease. (e.g., Leukemia & Lymphoma Society, American Heart Foundation, etc.).
INDUSTRY
Pharma
Pharmaceutical Companies (e.g., Pfizer, Merck, Lilly) concentrate on drug development. They do little basic science research. Each Pharma company has a library of unique chemical compounds that they use for drug discovery. This library can be as large as hundreds of thousands of unique chemical compounds. They know the chemical structures of the compounds, but they don’t know what these compunds can do or how they will act in the human body. That is where the academic publications come in. Pharma uses the academic’s publications pertaining to a specific disease, (e.g., a new pathway or a protein that may inhibit or turn on/off a specific gene) and designs experiments around it to test the unique compounds in their library. When they narrow down a subset of compounds that have an effect on the disease, the drug compounds advance to a different testing phase to see efficacy and safety. They also use animal models in their testing. They do little work on finding the new pathways to study and rely on the academics to publish new ideas and concepts.
When the drug is developed enough for testing in humans, a clinical trial is started. The Pharma company will than sponsor the trial at medical centers and cover the costs. Data from the clinical trial is used to ask the FDA for approval to market the drug.
Biotech
There are two different types of biotech companies (at least in my mind). There are biotechs that are equivalent to Pharma companies in that they both produce medicines, but differ in that the medicines made by biotechs are derived from living organisms while those made by pharmaceutical companies generally have a chemical basis. Simply put, the biotechs work on immunotherapies, not chemical drugs. Immunotherapy is type of therapy that uses substances to stimulate or suppress the immune system to help the body fight cancer, infection, and other diseases. Some examples of these would be your vaccines, monoclonal antibodies, cytokines, etc. An example of a large Biotech is Amgen, which is well known for producing EPO (erythropoietin) which is used to treat anemia. Biotechs do more basic science research than Pharma, but only do the research in their field of interest.
There is also another type of biotech, that most in the academic world know about. These are the smaller biotechs that are spin outs from the Universities. Professors have found something in their laboratories that they want to develop and commercialize themselves. The Universities help to patent the product, maintain the IP and continue to have a financial interest in the product, but don’t invest in the commercialization of the product to avoid a conflict with their academic mission. While some of the IP is sold to larger companies right away, other discoveries are too early in their development and the science would be lost if there was no initial purchase by a large company willing to work it up. The startup allows the scientific development to progress with a commercial focus. Venture capitalists are brought in to help fund the company. Some of these companies grow to be very large corporations in their own right, (e.g., Amgen), but most startups get acquired by larger companies. The product ultimately has a better chance of success and the venture capitalist get a return on their investment.
When the therapy is developed enough for testing in humans, a clinical trial is started. The Biotech company, large or small (start up), will then sponsor the trial at medical centers and cover the costs. Data from the clinical trial is used to ask the FDA for approval to market the drug or therapy.
CLINICAL TRIALS
Once a new drug/therapy is ready to be tested in humans, a clinical trial is set up. Clinical trials are designed to have a participant get an intervention to see what happens in humans. The interventions is the thing that is being tested (e.g. the new drug, new therapy, medical device, etc.). All participants are not allowed to get the actual intervention and may get the control or placebo. This is done randomly in a blind study where the participant does not know if they got the intervention or the control. It takes years to get enough data in the clinical trial to satisfy all the FDA requirements for approval of a new drug or therapy.
DEI in Clinical trials
There is diversity, equity and inclusion in the setup of all clinical trials. There has to be.
DEI allows the clinical trial to recruit the population that actually has the disease, not the population that is easiest to find or the population that has the easiest access to the trial.
Would you want to take a drug or therapy that was not proven to work on the population you are part of? Why would you want to risk the side effects? (all drugs and therapies have side effects). Take a look at this article that references a well known problem with heart drugs. GSK, “We’re not a world of men – it’s as simple as that’: how gender diversity in clinical trials improves health outcomes for women”. 6 March 2023. Most early research in chronic heart disease was conducted on men, despite the fact 80% of women between the ages of 40 and 60 have one of more risk factors associated with CHD – a leading cause of death for women – and experience life-threatening symptoms of heart attacks differently.
Do we want to go back to those days, where we waste 30 years of research and clinical trials to bring the wrong drug compound to market so that it only helps a few? There is no guarantee that one drug will work for all, but a diversity plan in clinical trials allows industry to tweak the drug/therapy so that other populations could benefit.
DEI allows the clinical trial to recruit the population that actually has the disease, not the population that is easiest to find or the population that has the easiest access to the trial. This is not just about race. Gender is its own population.
Do you actually want to eliminate or drastically reduce funding for medical research?
Don’t throw away the future because you can’t see the potential of the future.
Would you consider stopping research for other items in our world? Think back to when the first cell phones came out. They were so cool and state of the art. You could not comprehend how they could get more advanced, Then the smart phone came out. How could they top this? Then the smart watch came out which is basically your phone on your watch. Who knows what is next? You need to remember that all the modern tech devices did have a start in academics even if it is for a shorter time period than for drugs/therapies. Can you imagine if people said that the flip phone was enough, why invest in future technology?
The rest of this post is going to be some cut and paste of the missions of the agencies that the Trump administration wants to eliminate or drastically cut the budget. I am putting this in here so that the lay person can get a summary of what these institutes do. Also, some of this information has already started to disappear from the internet so people might not be able to find it themselves. You read through and decide if you think we should dis-invest in these items. Once we cut our investment in research, we may never get back the current quality we have now, any time in the future. Open your eyes to possibility.
-MOTL
“The future belongs to those who see possibilities before they become obvious
~ John Sculley
Health and Human Services (HHS)
The mission of the U.S. Department of Health and Human Services (HHS) is to enhance the health and well-being of all Americans, by providing for effective health and human services and by fostering sound, sustained advances in the sciences underlying medicine, public health, and social services. You can go here and here to see the 13 operating divisions, but I am only going to review a few (NIH, FDA, CDC) that have to do with medical research.
From wikipedia
The United States Department of Health and Human Services (HHS) is a cabinet-level executive branch department of the U.S. federal government created to protect the health of the U.S. people and providing essential human services. Its motto is “Improving the health, safety, and well-being of America”.[2]
HHS is administered by the secretary of health and human services, who is appointed by the president with the advice and consent of the United States Senate.
The National Institutes of Health (NIH)
The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the nation’s medical research agency — making important discoveries that improve health and save lives.
Thanks in large part to NIH-funded medical research, Americans today are living longer and healthier. Life expectancy in the United States has jumped from 47 years in 1900 to 78 years as reported in 2009, and disability in people over age 65 has dropped dramatically in the past 3 decades. In recent years, nationwide rates of new diagnoses and deaths from all cancers combined have fallen significantly.
NIH’s mission is to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability.
The goals of the agency are:
- to foster fundamental creative discoveries, innovative research strategies, and their applications as a basis for ultimately protecting and improving health;
- to develop, maintain, and renew scientific human and physical resources that will ensure the Nation’s capability to prevent disease;
- to expand the knowledge base in medical and associated sciences in order to enhance the Nation’s economic well-being and ensure a continued high return on the public investment in research; and
- to exemplify and promote the highest level of scientific integrity, public accountability, and social responsibility in the conduct of science.
In realizing these goals, the NIH provides leadership and direction to programs designed to improve the health of the Nation by conducting and supporting research:
- in the causes, diagnosis, prevention, and cure of human diseases;
- in the processes of human growth and development;
- in the biological effects of environmental contaminants;
- in the understanding of mental, addictive and physical disorders; and
- in directing programs for the collection, dissemination, and exchange of information in medicine and health, including the development and support of medical libraries and the training of medical librarians and other health information specialists.
NIH works to turn scientific discoveries into better health for all. As the largest public funder of biomedical and behavioral research in the world, NIH is the driving force behind decades of advances that improve health, revolutionize science, and serve society more broadly.
Evidence of the varied, long-term impacts of NIH activities comes from a variety of sources, ranging from studies on specific health topics, to broader analyses of NIH as a whole. Explore this webpage to discover more about how NIH provides value for the public’s investment.
“ClinicalTrials.gov” is part of the National Institutes of Health (NIH), specifically managed by the National Library of Medicine (NLM) within the NIH
ClinicalTrials.gov is a website and online database of clinical research studies and information about their results. The purpose of ClinicalTrials.gov is to provide information about clinical research studies to the public, researchers, and health care professionals. The U.S. government does not review or approve the safety and science of all studies listed on this website.
ClinicalTrials.gov:
- Relies on sponsors or investigators to submit and update information about studies
- Lists up-to-date information on clinical research studies and their results with new studies added almost every day
- Includes studies that take place in all 50 states and over 200 countries
- Supports laws, regulations, and policies that require sponsors and investigators to publicly share information about clinical trials, including results
What is clinical research and why is it done?
Clinical research is medical research that studies people to understand health and disease. Clinical research helps improve the way doctors treat and prevent illness. Through clinical research, researchers learn:
- How the body works
- How illness develops in people, such as how diseases get better or worse over time
- How the body handles a possible treatment
- Which behaviors help people stay healthy and prevent illness, and which behaviors raise the chance of illness
The goal is to use science to improve people’s health care and health over time. The participants who join and take part in clinical research studies may or may not get any benefit for themselves.
There are 2 main types of clinical research:
- Clinical trials, also called interventional studies
- Observational studies
Both may try to learn more about an intervention, which may be a drug, behavior, or medical device. The main difference is clinical trial participants are assigned to get an intervention, but observational study participants are not assigned to get an intervention.
Clinical trials
Clinical trials are research studies in which researchers assign participants to get one or more interventions to test what happens in people. Because of this, clinical trials are also called interventional studies. Often, the intervention is investigational, which means it is not approved for doctors to prescribe to people.
In some clinical trials, researchers assign participants to interventions randomly. This means that researchers assign the participants by chance. Usually, participants (or their doctors) don’t choose what intervention they will get when they join a clinical trial.
Observational studies
Observational studies are research studies in which researchers simply collect information (called data) from participants or look at data that was already collected. The data may be about participants’ health, habits, or environments. In observational studies, researchers do not assign participants to get an intervention. If there is an intervention, participants were already using it as part of their regular health care or daily life.
Often, researchers use observational studies to look at (observe) the different ways people behave and how it affects their health. Some observational studies use patient registries. A patient registry is an organized collection of data that patients agree to give. Researchers can use a patient registry to quickly access data provided by hundreds, or thousands, of similar patients.
US Food and Drug Administration (FDA)
For more than 100 years, the FDA has been working to carry out our mission of promoting and protecting public health, and that means your health.
FDA Mission
The Food and Drug Administration is responsible for protecting the public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices; and by ensuring the safety of our nation’s food supply, cosmetics, and products that emit radiation.
FDA also has responsibility for regulating the manufacturing, marketing, and distribution of tobacco products to protect the public health and to reduce tobacco use by minors.
FDA is responsible for advancing the public health by helping to speed innovations that make medical products more effective, safer, and more affordable and by helping the public get the accurate, science-based information they need to use medical products and foods to maintain and improve their health.
FDA also plays a significant role in the Nation’s counterterrorism capability. FDA fulfills this responsibility by ensuring the security of the food supply and by fostering development of medical products to respond to deliberate and naturally emerging public health threats.
CDC Centers for Disease Control and Prevention
CDC is the nation’s leading science-based, data-driven, service organization that protects the public’s health.
CDC works 24/7 to protect America from health, safety and security threats, both foreign and in the U.S. Whether diseases start at home or abroad, are chronic or acute, curable or preventable, human error or deliberate attack, CDC fights disease and supports communities and citizens to do the same.
CDC increases the health security of our nation. As the nation’s health protection agency, CDC saves lives and protects people from health threats. To accomplish our mission, CDC conducts critical science and provides health information that protects our nation against expensive and dangerous health threats and responds when these arise.
Fromm wikipedia:
The Centers for Disease Control and Prevention (CDC) is the national public health agency of the United States. It is a United States federal agency under the Department of Health and Human Services, and is headquartered in Atlanta, Georgia.
The agency’s main goal is the protection of public health and safety through the control and prevention of disease, injury, and disability in the US and worldwide. The CDC focuses national attention on developing and applying disease control and prevention. It especially focuses its attention on infectious disease, food borne pathogens, environmental health, occupational safety and health, health promotion, injury prevention, and educational activities designed to improve the health of United States citizens. The CDC also conducts research and provides information on non-infectious diseases, such as obesity and diabetes, and is a founding member of the International Association of National Public Health Institutes.