Hope, Hype, and the Problem With New Drug Development

Editor's Note: Abhimanyu Mahajan, MD, MHS, Assistant Professor, Department of Neurological Sciences, Rush University, Chicago, Illinois, served as co-author on this article.

Before I started medical school, I assumed that medical practice dealt in absolutes.

The scientific method, ingrained in me from years of basic science courses, was logical and led to straightforward, immutable conclusions. Medicine was a field based in science, after all, and should follow this same linear thought process. My future patients would have a clear diagnosis, prognosis, and response to treatment.

However, as all practicing clinicians know, this rarely goes as envisaged, especially in my chosen field of neurology where uncertainty is pervasive. Even the correct diagnosis of a patient with neurodegenerative disease, based on clinical observation, assessment, and interpretation, can be challenging. Diagnoses are dynamic and evolve over months and, not uncommonly, years. Autopsy results, the proverbial ashes of the fire that once was, may frequently disagree with the clinical diagnosis.

It is, therefore, not surprising that effective disease-modifying treatments and cures are hard to come by, especially as scientific discovery is an inherently slow process.

Repetition of findings is fundamental to validation; revolutionary ideas can take years if not decades to cultivate, and serendipity cannot be planned for. All of this poses a dilemma for doctors and patients waiting for a breakthrough with bated breath, an industry worried about its monetary investment, and a society accustomed to instant gratification.

Although some of this may dishearten or discourage clinicians, more treatments that may significantly benefit our patients are getting approved than ever before. From 2000-2009 to 2010-2018, the mean number of annual drug approvals increased by 64%, and biologic approvals by 140%. The US Food and Drug Administration (FDA) has approved over 120 new treatments in the past decade alone.

ClinicalTrials.gov currently lists over 413,000 ongoing studies, 77% of which are focused on interventional therapies or treatments; 61,648 of these studies are actively recruiting participants. Although statistically the majority of these treatments will fail, some are likely to succeed. A single effective treatment backed by high-quality evidence may be the discovery we need to modify a seemingly incurable disease, benefiting millions of patients, such as the 10 million people living with Parkinson's disease worldwide.

Why We Should Be Concerned

An impediment to true, meaningful scientific progress is hype. It is often peddled through the internet. Online health information is now more accessible than ever before and patients have, not surprisingly, become more reliant on internet "authorities" rather than their own clinicians. These outside sources, especially those describing new curative treatments, are of questionable accuracy and misleading.

This has become especially apparent during the COVID-19 pandemic. As of April 12, 2022, the FDA has issued warning letters to over 200 companies with fraudulent claims of benefit against the SARS-CoV-2 virus. This is probably a significant underestimation of the number of treatments with hyperbolic claims that have emerged over the past several years which are supported by little to no evidence.

Simultaneously, the FDA has been approving a number of new treatments through new legislation (also known as the "Accelerated Approval Program") that expediates this process. In 2018, 81% of drugs approved benefited from at least one such program.

The reduction in FDA review times may also be due to the fact that new drugs are being approved based on less evidence, not infrequently just a single pivotal trial. In fact, an analysis of 154 Cochrane reviews found that about 10% of treatments were backed by high-quality evidence. This is potentially concerning in light of the fact that privately funded research through a trillion-dollar industry has significantly increased.

The Lesson of Aducanumab

The recent expediated approval of the highly controversial drug aducanumab (Aduhelm) is a fascinating case study of an amalgamation of these factors: hype by the media blitz before peer review and the hope of a cure for a disease that affects millions. While the drug's approval has been met with backlash from both national media and the scientific community, hope has led to at least partial support from patients and their caregivers as well as the Alzheimer's Association. We must be proactive in ensuring transparency and making sure that public trust in the scientific and approval process is not eroded.

Ultimately, how do clinicians approach the dichotomy between the promise and possibility of new medical treatments with the unsupported hype of those with more dubious claims in our practice, and most important, in conversations with our patients?

Ethically and morally, we should all agree that honest communication is part of a good relationship. It is our duty as physicians to point out bogus claims not based in evidence, while recognizing that the speed of scientific discovery is slower than the hope of the human spirit. As patients, we must learn to separate hope from hype and in times of uncertainty, rely on the expertise of our physicians as we navigate the journey of discovery, care, hope, and despair together.

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Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape.

Cite this: Hope, Hype, and the Problem With New Drug Development - Medscape - May 06, 2022.