Breakthrough Prizes in Life Sciences are Cool

Research we need even more is boring, uninteresting, and has little or no funding. I was pleased to read the news last week of the Breakthrough Prizes in Life Sciences, giving $3 million each to 11 actively working basic scientists in medical and biological research. This is a great idea and a way to promote basic research. Hopefully one or more of these scientists will produce research results that lead eventually to progress in the treatment of cancer, neurologic disease management or other fields of medicine. Still these prizes reward researchers in areas where there is already enough interest, money and sexiness to have attracted these brilliant researchers. Hopefully the awards will attract even more.

Unfortunately we also need to find some way to incentivize someone to do less intellectually attractive studies to help us with mundane but also important decision options we face daily in practice. Treatment of such common and high-morbidity problems as hypertension, osteoarthritis and diabetes have many questions still unanswered and with little hope of definitive answers coming soon or ever. This is because there is little incentive to anyone to do the studies needed. They don’t involve a new high-profit drug so big pharma has no financial incentive. Physician groups have little incentive to see if non-physician providers can assume some of the things we now get paid to do ourselves. Many questions that need answers are simply head-to-head studies of commonly used treatments or treatment models. Another problem is often we don’t even have appropriate outcomes to use to measure the results of the things we do.

As a primary care physician

I was pleased to read the news last week of the Breakthrough Prizes in Life Sciences, giving $3 million each to 11 actively working basic scientists in medical and biological research.  This is a great idea and way to promote medical basic research.  Hopefully one of more of these scientists will produce research results that lead eventually to progress in the treatment of cancer, neurologic disease management or other fields of medicine.  These prizes reward researchers in areas where there is already enough interest, money and sexiness to attract brilliant researchers.  Unfortunately we also need to find some way to incentivize someone to do less intellectually attractive studies to help us with mundane but also important decision options we face daily in practice. Treatment of such common and high-morbidity problems as hypertension, osteoarthritis and diabetes have many questions still unanswered and with little hope of definitive answers coming soon or ever.  This is because there is little incentive to anyone to do the studies needed. They don’t involve a new high-profit drug so big pharma has no financial incentive.  Physician groups have little incentive to see if non-physician providers can assume some of the things we now get paid to do ourselves. Many questions that need answers are simply head-to-head studies of commonly used treatments or treatment models.  Another problem is often we don’t even have appropriate outcomes to use to measure the results of the things we do.

It is frustrating a as a primary care physician that there is almost no money to fund research to get answers to many questions we routinely face in our practice of medicine.  There is little or no data to tell us which of several options are best for the treatment of numerous health conditions.  Here are just a few examples of questions to which we do not have answers or any expectation of answers on the way:

  • Which thiazide diuretic is the best choice to treat hypertension?  Hydrochlorothiazide certainly dominates the U.S. market, but much of the early research to support the benefits of thiazide use to prevent stroke from hypertension was done using chlorthalidone, and more than a few practitioners feel that chlorthalidone is a better option.  Those of us who practiced long enough ago to remember using chlorthalidone regularly preferred hydrochlorothiazide because of frequent hypokalemia (low potassium) using chlorthalidone, but this was also when use of digoxin was common and the risk of hypokalemia when on digoxin was a concern.  Indapamide, an infrequently used thiazide is also a consideration.  A head-to-head trial of some sort using these three drugs, along with a potassium sparing combination like triamterene-hydrochlorothiazide should be relatively easy to do and the information obtained could be practice changing and powerful.
  • Which of the many supplements commonly used by patients and recommended by providers really work, and what is the best way to use them?  Controlled and appropriately powered trials of many of the commonly used supplements would really help patients know what is worth trying.  Does glucosamine-chondroitin sulfate work well enough to try for most patients with osteoarthritic pain? If so at what dose is needed, and how long does a patient need to take the it to see if it works, and for which areas of pain?  Is red-yeast-rice effective at lowering cardiovascular risk by lowering LDL?  Is it safer than the prescription statins and is it safe to try in patients who have not tolerated statins?  What conditions is fish oil really good for and what types work best?  The list of studies needed in this arena is long.
  • What model of primary care is most effective, gives the best outcomes and meets patient’s needs and expectations the best?  Are physicians better than, as good as or inferior to teams of physicians and physician assistants in primary care outcomes?  How do either of these compare to nurse practitioners?  How are other professionals like nurses, therapists, counselors, coaches, nutritionists, and case managers best used, and does their use save money, lead to better outcomes, or have other benefits?  Do alternative care providers like naturopaths, chiropractors, etc.  give equal, better or inferior outcomes to traditional medicine. There are hints that some of these models are good, but no solid evidence guides us in trying toward change in our care models.
  •  What method of payment for health care will best serve our populace and be acceptable to physicians, patients and payers?   This is a question that the Affordable Care Act hopes to try to address, but prior attempts to promote behavior change by financial incentives have led to undesirable unanticipated consequences. Somehow the incentive is going to have to lead to proven high-quality care choices by providers while reigning in costs.  Words like rationing, cookie-cutter care, and best-practice come to mind with the positive and negative connotations attached.

For me the answers to this type of questions could lead to more breakthroughs in medical care than the basic science the Breakthrough Prizes in Life Sciences are seeking to reward and make popular.  

that there is almost no money to fund research to get answers to many questions we routinely face in our practice of medicine. There is little or no data to tell us which of several options are best for the treatment of numerous health conditions. Here are just a few examples of questions to which we do not have answers or any expectation of answers on the way:

  • Which thiazide diuretic is the best choice to treat hypertension? Hydrochlorothiazide certainly dominates the U.S. market, but much of the early research to support the benefits of thiazide use to prevent stroke from hypertension was done using chlorthalidone, and more than a few practitioners feel that chlorthalidone is a better option. Those of us who practiced long enough ago to remember using chlorthalidone regularly preferred hydrochlorothiazide because of frequent hypokalemia (low potassium) using chlorthalidone, but this was also when use of digoxin was common and the risk of hypokalemia when on digoxin was a concern. Indapamide, an infrequently used thiazide is also a consideration. A head-to-head trial of some sort using these three drugs, along with a potassium sparing combination like triamterene-hydrochlorothiazide should be relatively easy to do and the information obtained could be practice changing and powerful.
  • Which of the many supplements commonly used by patients and recommended by providers really work, and what is the best way to use them? Controlled and appropriately powered trials of many of the commonly used supplements would really help patients know what is worth trying. Does glucosamine-chondroitin sulfate work well enough to try for most patients with osteoarthritic pain? If so at what dose is needed, and how long does a patient need to take the it to see if it works, and for which areas of pain? Is red-yeast-rice effective at lowering cardiovascular risk by lowering LDL? Is it safer than the prescription statins and is it safe to try in patients who have not tolerated statins? What conditions is fish oil really good for and what types work best? The list of studies needed in this arena is long.
  • What model of primary care is most effective, gives the best outcomes and meets patient’s needs and expectations the best? Are physicians better than, as good as or inferior to teams of physicians and physician assistants in primary care outcomes? How do either of these compare to nurse practitioners? How are other professionals like nurses, therapists, counselors, coaches, nutritionists, and case managers best used, and does their use save money, lead to better outcomes, or have other benefits? Do alternative care providers like naturopaths, chiropractors, etc. give equal, better or inferior outcomes to traditional medicine. There are hints that some of these models are good, but no solid evidence guides us in trying toward change in our care models.
  • What method of payment for health care will best serve our populace and be acceptable to physicians, patients and payers? This is a question that the Affordable Care Act hopes to try to address, but prior attempts to promote behavior change by financial incentives have led to undesirable unanticipated consequences. Somehow the incentive is going to have to lead to proven high-quality care choices by providers while reigning in costs. Words like rationing, cookie-cutter care, and best-practice come to mind with the positive and negative connotations attached.

For me the answers to this type of questions could lead to more breakthroughs in medical care than the basic science the Breakthrough Prizes in Life Sciences are seeking to reward and make popular.

Leave a reply