Alison Bateman-House, PhD, Assistant Professor Division of Medical Ethics at NYU Grossman School of Medicine, discusses ethical concerns in rare diseases and expanded access programs.
Rare disease patients, caregivers, and healthcare practitioners often face unique challenges. This includes the ethical concerns involved in drug development, clinical trials, and treatment plans. There are also ethical decisions being made by drug developers and clinical researchers on which therapies to develop.. Some rare diseases have a numerous sponsor interest due to being seemingly easy to develop a drug or being a guaranteed money maker. However, most rare diseases do not have any drug development programs.
Ethical concerns are also evident in the designing of clinical trials where researchers have to choose which patient populations will be enrolled. Oftentimes, choosing between the least-affected patients versus most-affected or whether to include children or adults. Interesting questions arise in the use of children in clinical trials, such as allowing parents to make difficult decisions on behalf of their children, or waiting until the children are old enough to make their own decisions. This leads to further concerns about drug effectiveness at different disease stages and when to best start treatment to slow disease progression.
Additionally, rare disease communities are often well-networked and tightknit and clinical trials are small. Parents may struggle with balancing emotions of grief and empathy for other families, while still needing to keep their own interest in mind. For example, learning that a child has been rejected from a clinical trial, supporting the families of those involved, while trying to get their child enrolled in these limited slots.
Expanded Access Programs
Dr. Bateman-House explains how Expanded Access Programs can be beneficial to rare disease patients and communities. Expanded Access Programs are a pathway for patients to access drugs and treatments that are usually unapproved by any regulatory agencies. These treatments are only typically available to those enrolled in a clinical trial. However, because of the small populations in rare diseases and the prominent need for therapies, these treatments can be beneficial to the patients that receive them. However, having patients exposed to the treatment before it is approved does create ethical and safety concerns that need to be addressed and understood by all parties involved.
There is a large need for education about clinical trials and Expanded Access Programs geared towards patients and to physicians who share information with patients. Dr. Bateman-House stated, “I do work in clinical trials, I do work in expanded access, and frequently I find that people just don’t know the basic rules of operation or what’s available to them… I frequently am pushing for more knowledge, more understanding of what’s there, more awareness of resources so people who don’t know what’s there can go find out.”
For more information on Expanded Access Programs, visit https://www.fda.gov/news-events/public-health-focus/expanded-access
To learn more about rare disease communities and their needs, visit https://checkrare.com/
