CHARLOTTE, NC: During Donald Trump’s State of the Union address in January, the president urged Congress to focus on a new health initiative called the “Right to Try” program. “Right to Try” gives patients who are terminally ill the opportunity to use experimental medications that have not yet been approved by the Food and Drug Administration (FDA). The bill passed and, in the process, it has spurred a vigorous debate which, on the surface at least, appears to have merits on both sides.
Other Experimental Drug Program
So far, so good, but here’s where the initiative gets muddy and tricky. The FDA already has a something in place known as the “Compassionate Use” program which essentially functions the same way as “Right to Try.”
Since its inception “Compassionate Use” has approved 5,816 of 5,840 drugs for an approval rate of 99.4 percent.
Back in the early 1990’s when HIV was hitting critical mass, the FDA began another program called the “Accelerated Approval Program (AAP) that allowed earlier approval of medications that treat serious conditions and that fill an unmet medical need.
With so much seemingly redundancy, the argument bogs down in the weeds with valid points on both sides.
FDA Drug Approval Phases
When a new drug is developed that is intended for patient use, it undergoes three clinical trial phases.
First, a company is required to prove the drug is relatively safe for humans. In the simplest of terms, that means the drug itself will not poison the patient. Note that phase 1 trials are often conducted on as few as 30 patients.
In the remaining two phases, trials test whether the drug is effective at treating the condition for which it is intended. They are also looking for any side effects. It is here that most drugs fail to pass approval. Many turn out to be ineffective or they result in severe side effects.
“Right to Try” legislation authorizes doctors to administer drugs that have cleared the first phase. However, they have not yet completed later human clinical trials. As such, terminally ill patients have the right to use potentially lifesaving medications without rigorous testing from the FDA. Or without waiting years for the drugs to become commercially available.
Right to Try failures
All of which, again, sounds promising for terminal conditions like ALS. Because it theoretically offers hope for patients in the midst of fighting the disease. Increasingly, however, policy analysts believe the legislation will not dramatically alter a patient’s situation. Therefore, they fear, the process could actually make it more difficult to obtain new drugs.
Experts say that, in the long run, rather than offering hope, such legislation could provide a false sense of hope. That falsity may be more harmful than the counterpart.
Adding to the muddle is that nothing in the legislation makes it mandatory for pharmaceutical companies to make medications available despite the fact that patients have the right to try them.
And there are several reasons for a company to deny a patient access to pre-approval drugs, with cost being the most prominent. Part of the expense is due to the limited supply of experimental drugs. And the fact that virtually no medical insurers will cover experimental treatments.
Consequently, the program would likely only be feasible for wealthy patients who can afford to pay the tab for the drugs as well as any complications that might arise when there are negative side effects.
Also to be considered is the threat of lawsuits against drug companies that provide the medications before they have been approved. In that case, however, the new “Right to Try” legislation does offer some protections for the pharmaceutical companies.
However, since there is no precedent in court as yet, the drug industry remains gun-shy. Another consideration is the long-term negative effect that trials could have if the data derived from experimental patients have a negative outcome. That could lead to further delays in the overall approval process.
Experimental drug law’s de minimis impact
Politically, “Right to Try” legislation is a win-win situation for the president and both houses of Congress. It has the appearance of offering hope for terminally ill patients. In reality, most experts believe the program will change little for either patients or their families.
Even if patients do somehow make a case to get drug companies to offer medications outside the trials, victims will still bear the burden of out of pocket costs. Unfortunately, this will be prohibitive for most Americans.
So the conundrum of ALS continues with its slow progression of morphing its patients from Dr. Jekylls into Mr. Hydes without no way to reverse the process.
About the Author:
Bob Taylor is a veteran writer who has traveled throughout the world. Taylor was an award-winning television producer/reporter/anchor before focusing on writing about international events, people and cultures around the globe.
Taylor is founder of The Magellan Travel Club (www.MagellanTravelClub.com)
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