Department of Health and Human Services Secretary Robert F. Kennedy Jr. is having an identity crisis. On the one hand, he’s paying lip service to his boss, President Trump, and his laudable deregulatory agenda. During a June roundtable, he stated, “We are going to continue to figure out new ways … of accelerating approvals for drugs and treatments that treat rare diseases.”
Yet, lofty rhetoric aside, RFK Jr. can’t seem to escape his conspiratorial, risk-averse mindset at the core of the Make America Healthy Again movement.
Recently, the Food and Drug Administration — which is a part of the RFK-run HHS — announced a ban on “export(ing) Americans’ cells to foreign labs in hostile countries for genetic engineering” without providing evidence of this malicious “genetic engineering.” If RFK Jr. wants to embrace innovation, he needs to free himself and his agency from the bizarre conspiracizing that fuels even more bizarre regulations. Kennedy’s identity crisis is costing taxpayers and consumers dearly.
On June 18, the FDA raised eyebrows by “announc(ing) an immediate review of new clinical trials that involve sending American citizens’ living cells to China and other hostile countries for genetic engineering and subsequent infusion back into U.S. patients — sometimes without their knowledge or consent.”
Pending review’s results, cell-related exports and international collaboration have ground to a halt. For all this intrigue, the FDA didn’t show a shred of evidence that anything sketchy is going on. In fact, FDA Commissioner Marty Makary stated in a recent podcast that it is “most likely that there is nothing nefarious going on” with the gene editing of American patients’ cells. The question then is why the FDA is opting for such a restrictive policy that will prevent promising international research.
Countries such as China host scientific programs producing research that can and will benefit millions of patients. For example, Chinese researchers are testing “a single-arm trial of adeno-associated virus (AAV)-OTOF gene therapy in 10 participants with autosomal recessive deafness 9 aged 1.5 to 23.9 years. The results showed that hearing improved in all 10 patients who were treated, and that the treatment was well tolerated.” International collaboration allows researchers to compare notes, ensure the replicability of trials, and bring various technologies home to their countries. Gene protectionism benefits no one.
The good news is that the FDA appears open to accelerating domestic gene therapy approvals. Recently, therapy manufacturer Sanofi announced “that the FDA has granted a fast-track designation to its one-time intravitreal gene therapy, SAR446597, for the treatment of geographic atrophy due to age-related macular degeneration.” And, Rocket Pharmaceuticals recently announced that the FDA “has granted Regenerative Medicine Advanced Therapy designation to RP-A601, the company’s investigational adeno-associated virus-based gene therapy for the treatment of PKP2-arrhythmogenic cardiomyopathy a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death.”
These developments are promising for patients, who must often live with conditions that are cost-prohibitive to manage and have no cure.
And, the FDA is doing a better job of communicating why it doesn’t approve therapies. On July 10, the FDA announced it was embracing “radical transparency” by publishing more than 200 complete response letters, which typically specify why medications are rejected. These letters are already offering a glimpse into the often-bizarre decision-making plaguing America’s drug regulator, including the rejection of a medication called Legubeti (to treat acetaminophen overdoses) because of “an unpleasant odor that may affect the tolerability of oral ingestion.”
However, until the HHS and FDA ditch strange conspiracy theories hampering international research collaboration, patients will be left without critical cures. It’s time for Kennedy and Makary to ditch needless rules and work quickly to save patients’ lives.