Areas of Insight
Health Information
Genomic medicines have life-changing potential for those facing serious or rare illnesses and are among the fastest-growing areas of research. Another important component of a bioethical framework is supporting a patient’s access to individual health information.
Access to Individual Health Information
As patients take greater ownership of their health and health outcomes, we support their desire to better understand the research in which they took part and see and use the data gathered about their health.
Sangamo is aligned with global efforts aimed at data sharing and clinical trial transparency to make appropriate information available to the scientific community, academic researchers, practitioners, and most importantly for patients.
Our first milestone toward this effort begins at the end of 2021 when the European Union Clinical Trial Regulation becomes effective. This is in addition to current guidance that requires clinical trial sponsors to provide summary results of clinical trial findings to participants in easy-to-read, non-technical language.
Our longer-term goal is to provide some level of individual clinical trial data back to participants. Returning individual research results to study participants is not a currently a widespread practice and is not always possible in clinical research. A variety of individual-level data return programs are being piloted in the United States and Europe in partnership with clinical trial sponsors.
These programs are looking at opportunities to share personal health information with secure delivery directly to patient volunteers or integrated into a patient’s electronic medical record. One of the ideas behind this concept is to help clinical trial participants avoid enduring multiples of the same test or repetitive invasive procedures.
Patient data return is gaining momentum within the industry and has generated a consortium of pharmaceutical companies working toward a standardized approach to data return to share data back to patients in a responsible manner that is standardized across pharmaceutical companies. The Patient Data Access Initiative (PDAI) has a goal to help companies implement data sharing with patients while they are still participating in the clinical trial.
Those who participate in a Sangamo-sponsored clinical trial receive a patient information letter containing details of the study in which they participated. This includes specifics related to the genomic medicine they received, how the therapy is expected to work in the body, and, depending on the type of genomic medicine being studied, the exact sequence of the gene that may have been inserted into a patient’s cells. This letter is intended to become part of a study participant’s medical records.
Patients are asking us for access to their own data, however, barriers and challenges exist for industry – including lack of clear guidance from the FDA and ensuring approval from the Institutional Review Boards who monitor and approve communication from the trial sponsor to the patient participants.
We will continue to advocate for access initiatives that put patients at the center of the development process, acknowledges their contributions to research and enables them to work with health care providers to make more informed decisions about their own health, including a variety of treatment options.
As a society, we’ve never had more access to information than we do now. Part of Sangamo’s commitment to patients is to work with other industry leaders to develop a standardized approach to returning an individual’s health data during and after a clinical trial, and in the case of genomic therapies, as monitoring data is understood in the long-term. We must work toward cross-industry best practices that protect patient
privacy, puts patients in control of the information they wish to receive, and offers information in a contextual manner that is easy to understand by patients or their health care providers – all while maintaining clinical trial integrity and government and regulatory requirements.
— Christeen Moburg
Vice President of Patient Advocacy and Government Relations
A Patient’s Perspective:
Sickle Cell Anemia
One patient, now in her 40s, was diagnosed with sickle cell anemia at 11 months old. At the time, the life expectancy for people living with sickle cell anemia was 21 years. Her parents knew they had no time to lose to learn more about the disease and advocate for their daughter’s care.
This hereditary disease can cause episodes of extreme pain, lasting a few hours to a few weeks. sickle cell anemia accounts for 200,000 emergency room visits a year, a significant burden of disease. In her lifetime, this patient has endured countless trips to the ER, received 500+ pints of transfused blood and experienced several minor strokes that weakened her left side.
Now an advocate for herself and others, she speaks regularly to doctors, nurses, and the public about life with this invisible and painful disease, putting her years of experience to work helping others make the most out of living with sickle cell anemia.
