Advancing Gene-Targeted Therapies for Central Nervous System Disorders Proceedings of a Workshop (2019) / Chapter Skim
Currently Skimming:
5 Meaningful Engagement of Patients and Families
5 Meaningful Engagement of Patients and Families
Pages 37-44
The Chapter Skim interface presents what we've algorithmically identified as the most significant single chunk of text within every page in the chapter.
Select key terms on the right to highlight them within pages of the chapter.
From page 37... ...
. • Neutral parties such as the Critical Path Institute can provide the infrastructure for data sharing that protects the interests of patients and investigators (Panzara)
|
From page 38... ...
She suggested that these challenges can be addressed by conducting empirical analysis of informed consent procedures for gene therapy trials, interviewing participants and researchers about their views on some of these ethical issues, and developing and testing shareable tools to mitigate therapeutic misconception and optimism. Cristina Sampaio agreed that participants' expectations resulting from therapeutic misconception need to be carefully managed.
|
From page 39... ...
Rather, it requires learning about their lived experiences with their conditions without assuming they all have the same perspective and attitudes. Timothy Coetzee, chief advocacy, services, and research officer at the National Multiple Sclerosis Society, agreed, noting that patient perspectives and attitudes are evolving with regard to participation in p lacebo-controlled trials, sharing data with others and gaining access to their own data, and switching treatments.
|
From page 40... ...
, added that the National Center for Advancing Translational Sciences requires data coordinating centers in the Rare Diseases Clinical Research Network to have an engagement and dissemination core to promote better engagement of industry and advocacy groups. Characterizing and understanding patients' and caregivers' values around treatment decisions can help providers communicate more effectively about benefits, risks, side effects, and eligibility criteria for a trial, according to Tabor.
|
From page 41... ...
She added that trial participants should be more engaged in decisions companies make about sharing placebo and non-placebo arm data with the field, which may require building new strategies for getting informed consent. Michael Panzara mentioned that neutral parties such as the Critical Path Institute can provide the infrastructure for data sharing in a manner that protects the interests of companies and academic researchers as well as the confidentiality of patients.
|
From page 42... ...
All three of these drugs treat rare diseases, which somewhat mitigates the impact of the high costs at least at a societal level, but gene-targeted therapies are on the near-term horizon for many more common diseases such as hemophilia, sickle cell anemia, and macular degeneration, said Tabor. If 20 to 25 new gene therapies are approved each year over the next few years, as Peter Marks and others anticipate, the costs could overwhelm the health care system.
|
From page 43... ...
In response to public criticism, ICER issued a statement in December 2018 saying they would no longer depend solely on QALYs, but would also include a measure called "equal value of life years gained" (evLYG) , which incorporates incremental gains in length of life regardless of changes in quality of life.
|
From page 44... ...
Access and equity are key, she said. She asserted that scientists, policy makers, and clinicians have a moral and ethical responsibility to make sure that gene therapy and other innovative approaches are not only available to the very wealthy or highly insured.
|
Key Terms
This material may be derived from roughly machine-read images, and so is provided only to facilitate research.
More
information on Chapter Skim is available.