A few months ago, 60 Minutes aired a segment about an investigational gene therapy that may have the potential to cure sickle cell anemia — a scourge that afflicts about 100,000 Americans, most of whom are African-American or Hispanic. The treatment uses a deactivated HIV virus to deliver copies of the corrected gene responsible for the disease. In a short time, the sickle cells are gone.
An incredible breakthrough, right? In the handful of patients treated so far, it seems to be working. In the words of one doctor: “This is a great example of why you have to put money into basic research.” Right on, doctor.
But then it stopped. That was the end of the story. No one bothered to mention — or even hint at — the millions of dollars and years of work that will be needed to take this treatment out of the lab and into a pharmacy.
No one called attention to the fact that some company must risk its future in the hope of creating a commercially viable treatment. And certainly no one acknowledged the dismal odds of success: only 1 in 5 drugs that are tested in people actually make it to market.
You see it all the time. The media overlooks and often simply denies the role pharma plays in translating science into medicine.
Why? Perhaps because giving our industry an ounce of credit would spoil the “big evil pharma” narrative. You can count on this: If a cure for sickle cell anemia, or Alzheimer’s or Parkinson’s or the dreaded disease of your choice, is eventually approved, you’ll see an entirely different 60 Minutes segment.
That’s where 60 Minutes missed the mark. It saluted the discovery, but ignored the hard work and huge investments that lie ahead.
Instead of lauding how patients are being saved from a devastating genetic disease, this story will begin like this: “We have just learned that XYZ pharma company plans to rip off the healthcare system by charging hundreds of thousands of dollars for a treatment that was basically invented by the government.”
You can predict this attack because we’re already hearing it. “How can pharmaceutical manufacturers claim they need sky-high profits to support their research? Everyone knows the real science is being funded by the National Institutes of Health!”
Anyone who swallows that logic should talk to one of the companies that have tried to develop a drug for Alzheimer’s disease. For years, scientists have predicted the secret to treating Alzheimer’s would be found in preventing amyloid buildup in the brain. And for years we’ve seen a series of anti-amyloid drugs fail in clinical trials, costing pharma manufacturers hundreds of millions.
Have you heard any sympathy for these companies? Don’t hold your breath.
But then, who wants sympathy? Our business model is based on the premise that if we undertake the risk involved in developing a new drug, we’ll be allowed to recoup our investment and make a decent profit. Fair enough? As long as this understanding exists, we can all look forward to many more medical breakthroughs.
That’s where 60 Minutes missed the mark. It saluted the discovery, but ignored the hard work and huge investments that lie ahead. That’s not how medical progress works. Can you think of a single drug that flew off the lab bench, zipped through the FDA and landed directly into the pharmacy?
So you say there’s a possible cure for sickle cell anemia? Great news. Now stand back as we in pharma make it a reality.
Sander Flaum is principal at Flaum Navigators.