Seriously Ill for Medical Research

Biomedical research (or experimental medicine), in general simply known as medical research, is the basic research, applied research, or translational research conducted to aid the body of knowledge in the field of medicine. Medical research can be divided into two general categories: the evaluation of new treatments for both safety and efficacy in what are termed clinical trials, and all other research that contributes to the development of new treatments. The latter is termed preclinical research if its goal is specifically to elaborate knowledge for the development of new therapeutic strategies. A new paradigm to biomedical research is being termed translational research, which focuses on iterative feedback loops between the basic and clinical research domains to accelerate knowledge translation from the bedside to the bench, and back again.

The increased longevity of humans over the past century can be significantly attributed to advances resulting from medical research. Among the major benefits have been vaccines for measles and polio, insulin treatment for diabetes, classes of antibiotics for treating a host of maladies, medication for high blood pressure, improved treatments for AIDS, statins and other treatments for atherosclerosis, new surgical techniques such as microsurgery, and increasingly successful treatments for cancer. New, beneficial tests and treatments are expected as a result of the human genome project. Many challenges remain, however, including the appearance of antibiotic resistance and the obesity epidemic.

Most of the research in the field is pursued by biomedical scientists in cooperation with molecular biologists.

Preclinical research

Preclinical research is research in basic science, which precedes the clinical trials, and is almost purely based on theory and animal experiments.

New treatments come about as a result of other, earlier discoveries — often unconnected to each other, and in various fields. Sometimes the research is done for non-medical purposes, and only by accident contributes to the field of medicine (for example, the discovery of penicillin). Clinicians use these discoveries to create a treatment regimen, which is then tested in clinical trials.

Clinical trials

A clinical trial is a comparison test of a medication or other medical treatment, versus a placebo, other medications and devices, or the standard medical treatment for a patient’s condition. Clinical trials vary greatly in size: from a single researcher in one hospital or clinic to an international multicenter trial with several hundred participating researchers on several continents. The number of patients tested can range from as few as a dozen to several thousands.

Funding

Research funding in many countries comes from research bodies which distribute money for equipment and salaries. In the UK, funding bodies such as the Medical Research Council and the Wellcome Trust derive their assets from UK tax payers, and distribute this to institutions in a competitive manner.

In the United States, the most recent data from 2003 suggest that about 94 billion dollars were provided for biomedical research in the United States. The National Institutes of Health and pharmaceutical companies collectively contribute 26.4 billion dollars and 27.0 billion dollars, respectively, which constitute 28% and 29% of the total, respectively. Other significant contributors include biotechnology companies (17.9 billion dollars, 19% of total), medical device companies (9.2 billion dollars, 10% of total), other federal sources, and state and local governments. Foundations and charities, led by the Bill and Melinda Gates Foundation, contributed about 3% of the funding.

The enactment of orphan drug legislation in some countries has increased funding available to develop drugs meant to treat rare conditions, resulting in breakthroughs that previously were uneconomical to pursue.

Regulations and guidelines

Medical research is highly regulated. National regulatory authorities oversee and monitor medical research, such as for the development of new drugs. In the USA the Food and Drug Administration oversees new drug development, in Europe the European Medicines Agency (see also EudraLex), and in Japan the Ministry of Health, Labour and Welfare (Japan). The World Medical Association develops the ethical standards for the medical profession, involved in medical research. The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) works on the creation of rules and guidelines for the development of new medication, such as the guidelines for Good Clinical Practice (GCP). All ideas of regulation are based on a country’s etchics standards code. This is why treatment of a particular disease in one country may not be allowed, but is in another.

Conflicts of interest

In 2001, the editors of 12 major journals issued a joint editorial, published in each journal, on the control over clinical trials exerted by sponsors, particularly targeting the use of contracts which allow sponsors to review the studies prior to publication and withhold publication. They strengthened editorial restrictions to counter the effect. The editorial noted that contract research organizations had, by 2000, received 60% of the grants from pharmaceutical companies in the U.S. Researchers may be restricted from contributing to the trial design, accessing the raw data, and interpreting the results.