Francis Collins, director of the National Institutes of Health (NIH), has announced a plan to create a new program to jump-start the development of new drugs and therapies. The new National Center for Advancing Translational Sciences will have the mission of trying to bring promising basic NIH research closer to clinical trials. To make this happen, cuts in other NIH programs will have to be made. One program, the National Center for Research Resources, which awards grants to fund researchers and equipment, is on the chopping block. Is this is a trade-off worth trying?
The push to find new drugs has stalled badly over the past decade. Few new therapies have emerged from the NIH’s research efforts involving mental illness, for example—with no real breakthroughs against Alzheimer’s, schizophrenia, severe depression, or any other major disorders. Nor has much progress been made in the fight against Parkinsonism, diabetes, obesity, stroke, tuberculosis, lung cancer, multiple sclerosis, amyotrophic lateral sclerosis, Huntington’s, muscular dystrophy, sickle cell, or ovarian cancer, despite many billions spent by the NIH on research and many billions more raised through charitable giving.
The pharmaceutical industry—which, given its profits, ought to be leading the war against disease—is in a deep research rut. Despite spending more than the NIH on research, its companies have little to show for their efforts. Their response has been merger after merger in hopes of covering up one molecular dry hole after another. Worse, in trying to milk the last ounce of profit out of their trickling pipelines, Big Pharma companies have become entangled in a seemingly endless series of patent wars, dubious marketing campaigns, recalls, lawsuits, and enormous fines. At the same time, the industry—eager to lower costs and escape red tape—is rapidly outsourcing clinical research overseas, where ethical standards may be less strict and the integrity of data less sound.
Given this bitterly disappointing state of affairs, it is understandable why the NIH might want to try kick-starting drug development with a new program. But this effort by itself is likely to fail.
Why? First, the new Center has a budget of only a billion dollars—not that much when it comes to developing drugs, vaccines, or devices. Second, the NIH has little of the needed infrastructure in place to facilitate moving from making discoveries in the lab to manufacturing safe products. The final, most significant problem is the poor rate of recruitment for domestic clinical trials of new drugs and therapies. Many promising trials simply cannot attract enough American subjects, and creating the new NIH program will do nothing to address that issue.
Ultimately, this is a problem of ethics. For far too long, the foundation of research ethics in the United States has been the assumption that subjects need to be protected when involved in research. The rationale for this line of thinking dates back to the infamous Tuskegee study and a long parade of inexcusable abuses of human subjects during the 1960s and ’70s.
Although subjects continue to need to give consent, to know about conflicts of interest, and to rely on sound peer review of the protocols they are asked to enter, far too many seriously ill Americans find the road to needed clinical trials hindered by too much red tape, bureaucracy, and paternalism. It is time to modify U.S. research ethics by creating a new class of human subject.
We need to learn from one of the few successes in research over the past two decades: finding treatments for HIV by speeding access to trials. HIV activist groups insisted that patients dying of AIDS and those infected with HIV be given faster access to clinical trials. They worked with researchers, review boards, and the Food and Drug Administration to expedite the process of clinical testing. It worked. We now have a variety of drugs that have changed HIV infection from a lethal disease to a chronic one.
Congress should move to enact legislation that permits the terminally ill; those facing irreversible, gravely disabling physical or mental impairments such as blindness or dementia; or those whose serious chronic illnesses defy all current efforts at treatment to assume greater risks as classes of potential subjects. It is time to extend the fast-track model that worked for AIDS to other classes of potential research subjects.
True, early stage clinical research is dangerous and often nonbeneficial. But even knowing that, some in the most dire categories of the ill and dying may still choose to become involved. By creating a new category under which the seriously ill and dying can enter trials even when little or no animal data are in place or when optimal dosing and mode of administration have yet to be determined—fully understanding, of course, the grim realities of the gamble involved—our nation may be able to boost the pace of research. A new NIH institute is an OK idea. A new ethic of research that can serve the interests of those who have run out of treatment options is a better one.