A crisis in US drug pricing: Consequences for patients with neuromuscular diseases, physicians, and society, part 2

Abstract

Escalating drug costs place patients at risk for financial toxicity and demand that physicians understand and act on the ethical and economic principles related to drug pricing. This manuscript reviews these principles and provides clinicians with a framework to think about the value of the drugs prescribed for patients with neuromuscular diseases. A key component of addressing the drug pricing crisis will be establishing a value based (benefit/cost) drug pricing framework. Determining the value of a drug is difficult and requires estimating the benefit and costs to patients and society while integrating indirect and contextual variables. Other considerations in drug pricing include "externality," the value to society derived from innovation. The Institute for Clinical and Economic Review (ICER) is a leading independent research organization providing clinicians with value-based price "benchmarks." All physicians must educate themselves in drug pricing principles and be prepared to have conversations regarding individual and societal value with the patients they serve.

Lambert-Eaton Myasthenic Syndrome and Botulism

“…Prior to this approval, 3,4-DAP was available only through compounding pharmacies or a compassionate use program requiring individual Investigational New Drug programs. Since the approval of amifampridine phosphate, concern regarding access and drug pricing has raised many questions about the “financial toxicity” of drugs and their role in the management of rare diseases such as LEMS 39,40 . This is increasingly a concern as historically established, previously compounded entities are approved for disease-specific indications as trademarked products.…”

Neurologic Therapeutics in 2035

“…Since 2000, prescription drug costs have increased by 69%, compared to physician services, which have increased 23%. 16 There are a number of studies demonstrating that the high cost of drugs has led to financial toxicity 17 for patients and inequitable distribution of many of the advances described above. With commercial insurers, there has been a strong shift to move new drugs out of pharmacy benefits and into medical benefits, further increasing the costs to patients.…”

Looming Crisis of Financial Toxicity in Neurologic Drug Pricing

“…Neurologists' primary obligation is to the individual patient, but we are also compelled to support access to high-quality care for all people, which requires advocacy for appropriate policy reforms to ensure value-based and fair drug pricing and treatment access. 5,10 This advocacy is particularly powerful when neurologists partner with likeminded professional and patient organizations such as the American Academy of Neurology and Patients for Affordable Drugs. We are growing accustomed to thinking about the financial toxicity due to individual out-of-pocket costs.…”