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Gene Therapy Calls for Shared Decision Making

As more gene therapy treatments are approved, this framework can help patients work with their doctors to make decisions about complex medical matters.

DNA strand

Almost 20 years after the completion of the Human Genome Project – a scientific achievement that generated the first sequence of the human genome – gene therapy is no longer a futuristic vision. Today, 27 cell and gene therapies are approved in the United States.

CSL Behring recently announced the first patient had received its FDA-approved gene therapy in the United States. And another 2,000 potential treatments in are development worldwide.

True to its name, gene therapy targets the cause of genetic diseases. Almost 80 percent of rare diseases are caused by a variant in a single gene, making these rare diseases potential candidates for gene therapy. By adding, inactivating, or repairing genes, gene therapy offers individuals with genetic diseases options for treatment.

Patients considering gene therapy as a treatment option face a complex decision – one that requires close coordination with their physician. A framework called “shared decision making” can help patients and their doctors communicate effectively while both weigh the options to select the best one for the patient’s health.

What Is Shared Decision Making?

Health care is full of hard-to-understand medical terms. But “shared decision making” plainly says what it is. It sounds like common sense, but anyone who has had to confront complicated medical situations knows that doctor–patient conversations can be overwhelming. First, the patient must understand the basics about the condition and a potential treatment. Then, with a physician’s counsel, the patient can gain an understanding of the risks and potential benefits.

That’s what shared decision making is all about. Your doctor might already practice this style of communication, or you might want to learn about it yourself. The approach allows the patient’s voice to come through while leaning on the physician as the expert who can provide evidence-based research needed for educated choices.

The optimal decision takes into account evidence-based information about available options, the provider's knowledge and experience, and the patient's values and preferences,” according to the U.S. Agency for Healthcare Research and Quality (AHRQ). describes the process this way: “Shared decision making occurs when a health care provider and a patient work together to make a health care decision that is best for the patient.

How to SHARE

The acronym SHARE can guide doctors to meet patients in a neutral, collaborative space:

S for Seek your patient’s participation.

H for Help your patient explore and compare treatment options.

A for Assess your patient’s values and preferences.

R for Reach a decision with your patient.

E for Evaluate your patient’s decision.

Find a toolkit for shared decision making, webinars and other resources at the AHRQ website.

For more information about gene therapy, patients can turn to the American Society for Gene and Cell Therapy (ASGCT), a professional organization that includes scientists, physicians and patient advocates Its Gene Therapy 101 guide includes an overview of several gene therapy approaches: gene addition, RNA therapy, gene editing, cell therapy and gene modified cell therapy.

Those who want to better understanding of genomics and genetic diseases, can visit the U.S. National Human Genome Research Institute, which provides a glossary of basic terms like gene and vector.