VISION FOR THE FUTURE CREATES HISTORY. To determine the evolution and manner in which science, technology, and society will unfold requires vision. The ability to imagine what can be and work towards that goal. Without creativity, without passion, and without perseverance, we are lost to roam shiftless and blind like a ship without a sail in the night. - Eric C. Leuthardt
A while ago I received an email from a graduate student telling me I should, in essence, choose between academia and commercialization. That, in his words, “trying to go in both ways will make people in the academic circles concerned.” He was in fact right. Often trying to do translational work and basic science can often raise eyebrows amongst members of the strictly academic community. Why is that? Well let me try to encapsulate some of those arguments. Here are a couple of them. “Knowledge should not be constrained or controlled, thus patenting your discoveries is antithetical to the mission of science.” Another. “Having a financial interest in the results of your research could alter your objectivity and thus corporations and business should never be involved in the scientific process.” A lot of these statements are made with the best ethical intentions. They are, however, all misguided.
Lets start with a very fundamental principle of why we do science. First and foremost, we pursue an improved understanding of nature to improve the human condition. The scientific method is one of the greatest inventions by man to pull our species out of a million year history in the wilderness. Put simply, science serves humanity, not the other way around. That principle can be stretched pretty far, ranging from why we study fungi to astrophysics, but ultimately all of them are there to enhance our lives in one way or another, period. So if we take that core principle – science serves man- as our north star it can help guide us through some fairly confused issues in science and medicine.
The mandate for the National Institute of Health is to promote the health of the citizens of the United States. The NIH is one of the dominant funding sources of American science. Now an important question here. What happens to those scientific discoveries? How do they get translated (big buzz word at the NIH) to create drugs, medical devices, new imaging techniques that improve patient’s lives in a myriad of ways? Simple answer - Industry. This is not a function that is performed by medical schools, research institutes, or government agencies. Companies need to invest extraordinary amounts of money to the tune of tens to hundreds of million dollars to bring a discovery to something that is safe and effective in the treatment of disease.
Now let me introduce another very simple principle – economics. Finance and the exchange of money are the fuel for powering human activities. Again, to be overly simplistic about this, we need money to do things. This is not a moral situation. Money in and of itself is not an evil, no more than gravity or other natural forces are evil. It is a force that strongly influences human behavior like gravity governs the movement of planets. So when thinking about translating discoveries to clinical treatments, one has to consider financial forces at work. Why? Because it is really expensive.
Alright, given those two concepts, what is a scientist to do when he or she finds something that could significantly improve human health. Say Dr. Scientist has discovered something that could treat a disease. Now what? How do we get it to humans? How does Dr. Scientist turn this into a therapy that grandma can take? Well someone, or a group of people, have to test the safety and efficacy of that concept. Typically this involves clinical trials that have to pass through the Food and Drug Administration. All of this costs money and most of it is NOT paid for by government. Rather, it’s a cost because of the government. Moreover, this is a risky process that that may fail at multiple steps along the way. Maybe the drug has a bad side effect, maybe it doesn’t work in humans, maybe maybe, maybe. So who is going to pay for this?
Here is where market forces take hold. If there is a big need, there may be the opportunity to sell this therapy and generate a profit. So investors need to put money together to fund this idea. A key here in these early stages is to ask what are they investing in? An idea? If it takes ten years and a hundred million dollars to develop this concept, what if somebody else tries to copy all those efforts and sell something identical right before the original group gets to the finish line? That is not only unfair, it also is financially too risky for anyone to consider as a reasonable investment. So there needs to be some guarantee that if someone is putting all this time and effort into a high-risk idea, that when the idea proves successful they can benefit from their hard work. This is what a patent is. It essentially allows for investors to reduce the risk of putting money behind a good idea. It is the American patent system that was drafted into the Constitution that has driven innovation in the US like no other nation. Is it limiting the dissemination of information? No, quite the opposite; by providing a limited monopoly on the idea for twenty years, the idea has to be fully disclosed in the patent so that it can be used for the good of the public after the expiration of the patent. So returning to fundamental principles – science is there to serve man – creating a system that facilitates the development of an idea into something that improves health is fundamentally moral. Not having these guarantees, essentially allowing an anarchy and predatory situation where someone can unfairly capitalize on another person’s ideas would actually lead people to be more secretive and unlikely to invest in risky concepts.
Next irrational fear — scientists or physicians should not have a “conflict of interest” (another big buzz word) in their research. Concern here is that the opportunity for getting financially rewarded for creating and translating a medical therapy would lead the individual to falsify or misrepresent their work. Therefore, if they really stand to benefit they shouldn’t do the research. There are several issues here that need to be addressed. First, the idea that to maintain one’s objectivity you have to remove all interest from the task. Quite frankly, when really looked at closely, this is just silly. Humans do things because they have an interest in doing them. If they didn’t they would be something akin to a zombie. Now financial interest is simply one thing that motivates humans. For the scientist, getting published in a high impact journal, getting promoted to professor, being recognized for one’s accomplishments are also potent motivators. Because a scientist is motivated to succeed should not be considered a detraction from the quality of their work whether it be financial boons or professional accolades.
Now will a small subset of people make unethical choices for a potential gain? Yes they will. This brings us to another point of social cost. Which is better – try to eliminate the actions of a few bad apples by making policies that make translation harder for everyone, or create scenarios that reward people for achieving positive medical advances with the knowledge that a small percent may cheat. Again put more simply, prevent a whole lot of good things by avoiding a few bad things, or have a whole bunch of good things happen knowing a few bad events may occur. At the end of the day, there is no free risk free scenario. But the question is which risks are we willing to tolerate. Once again considering the two fundamental principles that science is intended to serve man, and that market forces govern human behavior, it is better to create policies that enable market forces to support socially positive outcomes (more translation advances) rather than creating policies that reduce that social motivation.
Now in advocating rewarding people for socially positive scientific and translational efforts, does that mean that we simply shrug our shoulders if someone violates ethical standards in their scientific research for some gain? No absolutely not. If someone were to falsify data in a clinical trial for a therapeutic for financial gain they should be criminally prosecuted. This act is in effect a form of malicious intent no different from fraud, insider trading, or even possibly assault. If a drug is falsely represented and patients take it, which results in a physical complication, the person or persons should be held criminally liable. Today, falsifying data leads to social and professional penalties (loss of a job, retraction of a paper, etc) but nothing that actually speaks to the justice of the harm they could or actually caused. Taken together, when a scientist is translating a technology to a clinical therapy the stakes should be raised for both the positives and negatives. Namely, if they do something that helps people –reward them handsomely. If they behave unethically – punish them severely. In this scenario we are likely to properly motive the right people, and halt the unethical behavior. This is likely better than the current scenario that prevents appropriately motivated and successful people from getting rewarded, and not really punishing the others when they transgress.