The world is entering the next big wave of medical and pharmaceutical breakthroughs.
The first wave began with the widespread use of vaccines and antibiotics in the 1940s and 1950s, which caused death rates in the industrialized world to plummet.
The second wave of breakthroughs occurred with the introduction of statins, treatments for HIV/AIDS, and the refinement of chemotherapy drugs in the 1990s.
Since that time, however, we’ve generally seen a period of incremental progress with few huge breakthroughs.
To be clear: This steady improvement in established fields of pharmacology has helped expand our understanding and provided more effective treatments to people with a variety of diseases. Steady and meaningful incremental improvement is still definitely positive.
After all, if a treatment helps a patient live six months longer than he or she would without the treatment, those six months of life are still significant.
Yet some have criticized this period of incremental improvement as evidence that something is broken in the medical-pharmaceutical field.
Every month, someone writes an analysis describing an “innovation crisis” in the industry because the number of so-called blockbuster drugs is declining. In fact, those inclined to conspiracy theories hold that there is a perverse incentive to treat rather than cure diseases, because treatments are more profitable.
The truth is much simpler: Science takes time. Developing new drugs and treatments does as well. The reason we have seemed stuck in a period of incremental progress is because the most promising new fields for breakthroughs have been in their infancy.
Fortunately, this appears to be changing.
The past two years have shown signs of astonishing progress in the field of gene therapy. We have every reason to believe we are at the beginning of the third wave of medical breakthroughs.
In the upcoming episode of my new podcast “Newt’s World,” which will be released Sunday, I speak to doctors who have been working on methods of nonhereditary gene therapy since the 1980s. Their efforts have led to the creation of the drug Luxturna, which corrects and reverses a rare genetic disorder that leads to childhood blindness.
In the episode you’ll hear from Christian, a young patient who describes what it was like to see the moon for the first time in his life after receiving the treatment.
I have written in more depth on the work of these doctors in a previous newsletter, but I mention this work again because it represents the starting point for a host of potential cures in the pipeline.
Already, similar methods are being used to test cures for macular degeneration – a far more common genetic disorder that causes blindness – and sickle cell disease, which leads to pain, frequent infections and anemia.
The area of gene therapy and genetic medicine will soon expand to treat and cure many currently debilitating diseases.
In addition, we have seen the development of astonishing cures for cancer that have the potential to completely upend the chemotherapy-based model that has defined the last 30 years of treatment.
CAR-T immunotherapy treatments combine gene therapy and other treatments to enable a patient’s own immune system to attack and defeat cancer cells. Like gene therapy, the initial approved treatments have been for relatively small patient populations, but they have provided the model that can be used for more common cancers.
While it is true that these new cures are currently costly, as more and more pharmaceutical companies commit resources to gene therapies and CAR-T, the prices will come down. This is a common, well-documented phenomenon in the industry.
Consider the recently developed cure for hepatitis C. The first treatment to come to the market that can cure most patients of this terrible virus cost more than $94,000. After three years, as more companies developed drugs based on the basic science of the first cure, the cost of curing hepatitis C has collapsed to $26,400 – and the price is likely to continue to decline as competition in the market expands.
Already, the Food and Drug Administration is expecting huge growth in the genetic medicine arena. By 2020, the FDA has said it is bracing for upwards of 200 gene therapy applications per year.
As competition increases – and more data become available to more companies – the prices will plummet.
I am focusing on this story for two reasons.
First, the stories of these doctors and patients are inspiring. The doctors’ decades-long struggles to take gene therapy from theory to practice is the definition of steadfast determination. Additionally, the patients’ willingness to volunteer for untested treatments for the betterment of humanity is a model of courage.
Second, as the nation debates how to make drugs more affordable to patients, it is important to keep in mind that reform should not come at the expense of innovation. It would be a tragedy if we allow misguided big government schemes like “Medicare-for-all” or government-imposed price controls to stop innovation in its tracks – the way it has in other countries with government-run health care programs.
This is the question we should be seeking to answer over the next 18 months: How do we accelerate progress while expanding access?
Republican (and Democratic) lawmakers must look for solutions that don’t sacrifice one for the other.