Patients’ Perspectives on Romosozumab

Meeting of the Bone, Reproductive and Urologic Drugs Advisory Committee Meeting; Public Hearing. Docket No. FDA-2018-N-4116

Oral Comments made by patient Judith Marple on behalf of the Global Healthy Living Foundation

“Patients’ Perspectives on Romosozumab”

January 16th, 2019

Disclosure:

I have disclosures to make. The Global Healthy Living Foundation paid for my travel here today. The foundation accepts grants and charitable contributions from pharmaceutical companies, government, private foundations, and individuals. Its medical team has been briefed on osteoporosis by independent scientists and physicians, as well as representatives from pharmaceutical companies.

Good morning. I would like to thank the FDA for this opportunity to provide comments today. My name is Judith Marple. I have osteoporosis. I am here representing the Global Healthy Living Foundation and other patients with osteoporosis.

The Global Healthy Living Foundation is a 501(c)(3) patient organization representing chronically ill patients and their caregivers across the U.S., Western Europe, Australia, and South America. GHLF works to improve the quality of life for people living with chronic disease by making sure their voices are heard and advocating for access to best-practice medical care.

I would like to start by speaking about my personal journey with osteoporosis. The inability to find proper treatment has caused a substantial burden on my everyday life. This journey started three years ago when I had my first fracture and two to three months later, a second one. I immediately went to a specialist and started physical therapy as well as trying various osteoporosis drugs. It was a struggle to endure the pain of physical therapy while healing my body to feel comfortable walking, sitting and standing. While at the same time I was feeling ill from the side effects of the drugs being prescribed to help me get better. I endured these difficulties with the constant motivation of being able to get back to the job I loved and most importantly getting back behind the wheel and driving to see my grandchildren. After a year of rehabilitation and with my doctor’s help I was able to accomplish these goals. Unfortunately, this year has taken a turn for the worse and despite my best attempts of being as careful as possible, I’ve had four new fractures with a running total of six bone fractures: five vertebral fractures and one sacral insufficiency fracture. These fractures have caused changes in the shape of my body with kyphosis, daily pain, and inability to stand, walk or sit without discomfort. Reluctantly, I have had to be placed once again on disability and am no longer able to work at the job I loved. My treatment plan has exhausted all options currently available to treat osteoporosis with no change to my risk status for fractures. My story is, unfortunately, a common story. The news that there is a new option which has shown impressive gains in

bone density is very exciting. I am hopeful for myself and many others who could benefit from this new drug.

Patients who have osteoporosis, like myself, have been living with this condition for years. We are waiting for FDA approval of romo, which I understand is the first drug in its class. It will be a welcome addition to the drugs we already have and fills some important unmet needs as the committee is aware.

I am here today to put a face to the thousands of patients who will immediately benefit from treatment of bone-building and bone degradation prevention drugs such as this one. I am also here to put a face to the optimism that many patients have towards a medication that could potentially change their lives and give them their independence back. I am 60 years young and have a full life to live. I long for the day that I am able to get back to work, go to the grocery store without having to stretch and mentally push myself, and finally, I can’t wait to get in that car outside and drive as many times as I please to go and kiss my grandchildren.

Thank you again for the opportunity to provide comments on this issue. We will be submitting written comments to the formal docket. If you have additional questions, I am available today to answer them, or refer you to the Global Healthy Living Foundation’s Medical Advocacy Liaison Dr. Daniel Hernandez, MD, who is also here today.

Sources:

1. “Romosozumab or Alendronate for Fracture Prevention in Women with Osteoporosis | NEJM.” New England Journal of Medicine, www.nejm.org/doi/full/10.1056/NEJMoa1708322.

2. “The Clinical Potential of Romosozumab for the Prevention of Fractures in Postmenopausal Women with Osteoporosis.” Journal of Research in Crime and Delinquency, journals.sagepub.com/doi/full/10.1177/1759720X18775936.