By the time a potential new medicine makes it to the clinical trial stage of research and development, it has typically gone through at least three years of preclinical testing to see if the treatment is likely to be safe and effective for use in humans. Then, clinical trials in humans must be conducted before a drug can be approved.
As with any medical treatment, participation in a clinical trial offers benefits and risks. To help a clinical trial volunteer make an informed decision, the clinical study team is required to disclose all known risks, benefits, and available alternative health care options. This is called informed consent and is an ongoing, interactive discussion to help patients make informed decisions about whether to begin or continue to participate in a clinical trial.
The potentially life-long effects of genomic medicine designed to alter the genetic instructions inside a body’s cells to slow or stop the progression of disease require an enhanced approach to patient informed consent.
Unlike traditional medicine clinical trials, it is impossible for physicians to reduce the dose or stop the treatment of a genomic medicine if the effects are outside of what is expected. The effects of genomic medicine are intended to be long-term and thus the consent must be equivalent. Genomic medicine informed consent is effectively consent for life, which demands unmatched accountability and proactivity – it is the responsibility of industry to set this new bar.
Our focus at Sangamo is working to create genomic cures for patients with genetic diseases – conditions that are severe and progressive, with no known cures.
We take very seriously the notion that genomic medicine is a pioneering field and poses inherent risks that are not yet fully understood. As the industry continues to monitor the long-term effects of genomic medicines, we must be fully transparent to inform future clinical trial patients of the potentially lifelong nature of their commitment, as well as our understanding of the risks and benefits.
The informed consent process must create an environment of trust to support a patient’s complete understanding of risks and potential benefits of participating in study. We work with patient advocacy stakeholders to ensure informed consent documents are developed with attention to language, literacy, and focus on what outcomes are most important to the patient.
A patient’s role in a clinical trial goes beyond receiving an investigational treatment. Active participation and patient partnerships are critical for clinical trials where there is a smaller patient population, such as those researching treatments for genetic disorders.
The research community must build approaches to informed consent specific to genomic medicines that are useful and support a patient’s understanding of the commitment as they decide whether to participate in a clinical trial.
For patients living with end-stage renal disease (ESRD), a kidney transplant often is the treatment of choice. To prevent graft rejection of the transplanted organ, patients are treated with lifelong immunosuppressive therapy. This standard-of-care therapy impacts the body’s immune system and can have side effects including an increased risk of infectious complications, cancer, and other drug- related toxicities.
One patient with ESRD knew her diagnosis would mean a lifetime of dialysis or a kidney transplant and spent years learning to live with a debilitating condition, not knowing what the future held. When she received the news that her kidney function was so weak that her life was in jeopardy, she and her physicians decided a kidney transplant was the best option.
A living kidney donor was identified, and the transplant was successful. More than five years later, the patient works with her medical team to counteract the side effects from immunosuppressant therapy. The therapy, while not ideal, remains the standard of care until there is a better option for kidney transplant patients.