Forever etched in golf fans’ memories is not the remarkable 65 shot by Tom Watson in the first round of the 2003 U.S. Open, but the courage of his caddy, Bruce Edwards.
Edwards, who had been Watson’s caddie for 30 years, had Lou Gehrig’s disease, which is always terminal. The outpouring of fan affection throughout the tournament was deeply touching. Edwards died the following year.
Today, there is no Food and Drug Administration (FDA)-approved drug that gives people who suffer with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease, a reason to be hopeful. But what if there were an experimental ALS drug in the early stages of FDA clinical trials showing breakthrough potential? Should Edwards have been free to purchase it if all available risk-reward information were known to him and his doctors?
Under current law, he could not.
We have grown accustomed to FDA’s monopoly on market access to drugs. Prior to 1962, new drugs had to pass only safety trials to be legally marketed. Effectiveness was left to consumer and doctor evaluation.
Today, for drugs to be marketed as FDA-approved, they must pass a Phase I safety trial, Phase II safety and effectiveness testing among a small sample of patients, and then a Phase III clinical trial with a higher number of patients. On average, the three clinical trials take seven years.
A new drug application (NDA) containing relevant data to be examined by FDA is the next step in the approval process. That review process takes an additional 1.5 years on average. Consequently, those who might benefit from a promising new drug cannot get it for approximately 8.5 years after it entered FDA clinical testing.
Not only do the clinical trials and NDA submission take time, they also take money. Drug developers experience a substantial outflow of hard cash, a long delay in possible revenues, and no guarantees the drug will be approved. That combination discourages many new drugs from being tested for efficacy.
FDA’s current drug approval process is an effort to deal with a formidable dilemma. Since no drug is completely safe, FDA can mistakenly approve a drug that subsequently produces harmful side effects that greatly outweigh therapeutic benefits. Alternatively, FDA can delay or deny approval for a drug that subsequently shows clear effectiveness and possibly life-saving ability.
When FDA errs on the side of excess caution, thousands of patients may die who could have been saved. But those deaths are rarely documented and never make the nightly news. Thus, it should not be surprising that in practice FDA is much more concerned with avoiding highly visible errors and clearly identified victims than with hidden, rarely identified victims who were denied access to drugs that were in the FDA approval pipeline.
A serious debate about FDA’s regulatory role should begin with a focus on the common-sense principle that the authority to make medical decisions belongs to patients and their doctors. The U.S. Court of Appeals of the D.C. Circuit recently supported the right of dying patients to access not-yet-FDA-approved drugs.
A second focus should be on the harm done by long delays before drug innovations reach the public. Thousands of people die each year, and millions more suffer unnecessary pain, as a result of FDA’s one-size-fits-all approval procedure. Worse, as twenty-first century technology speeds medical innovations, the number of unnecessary deaths and amount of suffering increases by the day.
A third focus should be on solving an emerging dilemma facing pharmaceutical companies that are gaining insights into how diseases (often rare diseases) relate to patients’ genetic profiles. The dilemma is that the greater the gain in personalizing medicine, the smaller the target population for such drugs, the smaller the prospective revenues, and the less likely there will be a worthwhile return on investment. This is due, for the most part, to the high cost of having to conduct the full set of FDA clinical trials.
FDA is unlikely to be the source of reforms that reduce its regulatory authority. Reforms that rely on FDA for implementation may be doomed to fail. Patients and their advocates should be looking outside FDA for a solution to one of the great under-reported health care crises of our time, the withholding of experimental drugs from people who would benefit from their use.