Next week there are two workshops going on at FDA that are important to our Hunter Syndrome community. They are both gathering vital patient and clinician perspectives that relate to the future of Hunter Syndrome treatments and research. Although I posted them on Facebook and Twitter, I wanted to make sure that the word was out and solicit any input you’d like to provide if you’re not going to be able to attend in person or online. If you will be attending or would like us to highlight particular thoughts you have on either topic, please comment below or contact me.
This program is co-sponsored by NORD and will provide a forum to discuss the role of immune tolerance induction (ITI) in patients receiving replacement biological products. The panels and speakers include physicians, researchers, pharmaceutical companies and patients/caregivers. NORD describes the goals of the workshop as: to discuss the impact of anti-drug antibodies on efficacy and safety of enzyme replacement therapies intended to treat patients with lysosomal storage diseases, and the risk and benefit of implementing prophylactic immune tolerance regimens to reduce the potential clinical impact of antibody development. I will be a panelist at this workshop and you can find the agenda here.
This is a very important topic to our community given the findings that patients with the more severe mutations and deletions causing Hunter Syndrome are both more likely to have adverse reactions (which can be life threatening) and more likely to create antibodies and neutralizing antibodies to the replacement enzyme (see the disclosure for Elaprase or www.elaprase.com), thus potentially neutralizing the effect of the drug itself. Although not a stated goal of the workshop, it is my great hope that as a result, there will emerge consensus guidance, even if only in the minds of parents and metabolic specialists, that will answer the questions of:
Who are the appropriate patients for ITI and in what circumstances?
What drug combinations might be most effective in conducting ITI?
Where are the clinicians, researchers, and facilities who have experience with ITI?
When should ITI be used prophylactically, to prevent antibody formation, versus therapeutically, only after antibody formation?
How should ITI be monitored and evaluated?
I hope you’re able to participate in this important workshop either in person or via webcast.
This program will provide patient input to FDA on the impact of the neurological manifestations of inborn errors of metabolism on daily life and patient views on treatment options. Below are topics to be addressed at the meeting. In addition to input generated through this public meeting, FDA is interested in receiving patient input addressing the questions below through written comments, which can be submitted to the public docket. I will be speaking at the meeting on Topic 2. Please let me know if you will be there and/or speaking as well!
Topic 1: Disease Signs, Symptoms, and Daily Impacts That Matter Most to Patients
1. Of all the signs or symptoms that you/your child experiences because of the condition, which 1–3 neurologic/neuropsychological signs and/or symptoms have the most significant impact on your/your child’s life? (Examples may include seizures, decreased muscle tone, sensory issues, etc.)
2. Are there specific activities that are important to you/your child but that you/your child cannot do because of these neurologic/neuropsychological signs or symptoms? (Examples of activities may include sleeping through the night, daily hygiene, going up the stairs, etc.)
3. How have your/your child’s neurologic/neuropsychological signs or symptoms changed over time?
Topic 2: Patient Perspectives on Current Approaches to Treating Neurologic Manifestations of Inborn Errors of Metabolism
1. What are you/your child currently doing to help treat the condition or its signs/symptoms? (Examples may include prescription medicines, herbal therapies, acupuncture, over-the- counter products, and other therapies including nondrug therapies such as diet modification.)
How well does this current treatment regimen treat the neurological symptoms of your/your child’s disease? For example, how well do the treatments improve your/your child’s ability to do specific activities?
2. Assuming there is no complete cure for your/your child’s condition, what specific attributes would you look for in an ideal treatment for the condition?
3. The process of informed consent is an important way for researchers to communicate the purpose of a clinical trial and the potential benefits and risks of the trial so that people can make an informed decision about whether to participate. Informed consent also ensures that parents are fully informed and are given opportunities to ask questions about the clinical trial. In addition to informed consent from parents, assent from children may also be needed. Assent is the term used to describe when a child agrees to be in a clinical trial. Among other considerations, children should be old enough to understand basic facts about the clinical trial in order to provide assent to participate.
In the informed consent process, what are important considerations to take into account in cases when the potential participant is a child? For example, how should the informed consent clearly communicate to the patient the potential benefits and risks of a study?