An unfortunate professional moment that I had to endure was years ago, when I took care of a business owner who had advanced cancer. After a long and stable period, her disease progressed quickly. Eventually, successive treatments failed. At this point, I raised concerns that her treatments were causing more harm that good. The only way to stop feeling so terrible was to avoid toxic therapies and instead focus on symptom management.She was slowly coming to terms with her mortality and was assisted by an intuitive sister. I was shocked to see her sitting in the chemotherapy chair. The nurse was dejected and told me that the patient had been given a Hail Mary. The Christian utterance of Holy Intervention, Hail Mary, was brought into oncology by doctors who began to prescribe futile treatments for desperate patients.Although chemotherapy was well-known for its dismal response and propensity to complications, I felt only the humiliation of a doctor who had lost a patient in pursuit of a better provider every time I saw her. She grew weaker each week and became more jaundiced. Yet, I believed I was wrong.I met her oncologist one day.He said, "I completely agree with you." She is in grave danger.Hence, why would you recommend more treatment?It was there.The patient died three weeks later. Although the personal cost of chemotherapy at the end was obvious, the patient died three weeks later.Recalling the patient brought back memories when I learned that the US Food and Drug Administration had approved a drug to treat dementia. This is a condition that is increasingly emblematic of an aging population.Although amyloid is thought to cause dementia, it's not clear if it causes it. The drug is designed for mild cognitive impairments in the hopes that it will stop their functional decline. After decades of disappointment, the drug's accelerated approval should have been cause for celebration. Instead, it led to the resignation of many FDA members and widespread scepticism among experts.Two pivotal trials that involved 3,200 participants produced contradictory results. The one that slightly slowed cognitive decline was better than the one that did not. Data monitoring committee decided to end the trial before it was complete. One-third of participants quit, and 40% suffered brain swelling and bleeding. This can lead to nausea, dizziness headaches, confusion, and even brain damage, which could be serious for the elderly.Statisticians claimed that there was no compelling evidence for the drugs, and the dementia experts who were part of the trial were not convinced. One of three members of the FDA advisory board resigned. He called it the worst drug approval decision in US history.FDA acknowledged that there were still uncertainties about clinical benefits. The drug was nevertheless approved at a cost of USD $56,000 per annum.It will be necessary for patients to undergo frequent scans and monitor their symptoms. Patients who are given it will also need to travel monthly to an infusion center, which can be disorienting in the case of dementia.The approval label doesn't specify that the trial was done on individuals with early dementia. The FDA has the right to withhold approval if the post-marketing trials fail to show benefits. However, it does not have the obligation. Experts believe such trials are more difficult because patients who are able to obtain the drug are less likely to accept a placebo.Advocates argue that breakthroughs are contingent on less effective therapies, and that this approval opens the door to better candidates to ease the burden of dementia. It is a hope that this will be true. In the meantime, the lamentations of a neurologist (and principal investigator), will resonate with any clinician who is struggling to find the right treatment.The patient's husband and I presented the data, but they didn't hear anything I had to say about my concerns. They only heard that there might be some benefit. I can almost hear every clinician agreeing to that.Modern medicine is exciting and rewarding, but it also comes with the responsibility for gatekeeping. Patients often assume that there is a strong reason to approve regulatory approval, while doctors are frequently confronted with drugs that are more notable for their efficacy than for their hype. It is easier to prescribe drugs than to talk to patients, so futile treatments are common.Australian doctors recently protested the Australian Therapeutic Goods Administration's (TGA) approval for a herbal remedy to treat benign prostate enlargement. This can lead to uncomplicated urinary symptoms.The TGA-assessed symbol for complementary therapies informs consumers that the body has evaluated the product's health claims and found scientific evidence to support them. This could be a positive initiative.The company behind the herbal remedy paid for a blinded trial with less than 60 men. Of these, 32 received the medicine. It is difficult to say that the drug has a significant effect on nocturnal urine production, as the company claims. The TGA has not made any further information public about the claim as the raw data cannot be verified.Complementary medicine sponsors can self-certify compliance with regulatory requirements, which allows them to state that they have evidence supporting their claims. Most products that have been evaluated have been found to be non-compliant due to the inability of companies to provide evidence.The industry of complementary medicine, which has little evidence of effectiveness, is valued at more than AUD $5 billion annually. It might seem that adding another herbal product to the already crowded market is irrelevant, but it does matter to have the TGAs stamp. It can be difficult to trust other decisions when a questionable supplement medicine is approved for a condition that already has prescription-based treatments.Patients deserve to know that drugs they read about and want are approved on the basis of evidence, regardless of whether the problem is cancer, dementia, or benign prostate enlargement. The professional societies can help by assessing the strength of the evidence and defining the context in which the drug is being prescribed. Patients can learn to ask questions beyond what is written and to probe their individual situation for the benefits and harms.Although regulatory agencies and professional societies have great power, they also carry a lot of responsibility. When it comes to their safety and well-being, patients should expect high standards.
