If you or someone you love has taken Ibrance, Verzenio, or Kisqali for appendix cancer, or was denied one of them, I want your story in the Patient-Led Global Appendix Cancer Registry. Records like yours are how these drugs get written into the treatment guidelines that insurance companies check.

What CDK4/6 inhibitors are

Ibrance (palbociclib), Verzenio (abemaciclib), and Kisqali (ribociclib) are CDK4/6 inhibitors. The FDA approved them for breast cancer. They are not FDA-approved for appendix cancer. Oncologists at major cancer centers still prescribe them to appendix cancer patients with GNAS or KRAS mutations, and some of those patients are responding.

Why insurance keeps saying no

I started collecting these stories because Dr. JP Shen at MD Anderson asked me to. He was seeing the same thing in his clinic that I was hearing in the Facebook group. Patients with documented mutations had a prescription from an oncologist who knew the science. Their insurance denied it anyway. The reason was almost always the same. Experimental. Not standard of care. Not in the guidelines.

Dr. Andrew Lowy at UC San Diego leads much of the research on these drugs in appendix cancer. He told me the real fix is not fighting insurers one denial at a time. The fix is getting the guidelines updated. When a drug is in the guidelines insurers use, coverage usually follows. When it isn’t, every patient has to fight on their own.

Right now, the guidelines most U.S. insurers check do not include CDK4/6 inhibitors for appendix cancer.

What it takes to change the guidelines

Guideline panels look at evidence. Published research counts. Real patient outcomes count. The more records that show how these drugs work in appendix cancer, by subtype, by mutation, and by tumor markers and scans, the harder it is to leave them out.

Dr. Lowy and his team have published case series on this. What’s missing is the bigger picture. How many patients are on these drugs. What their diagnoses and mutations are. What happened to their tumor markers and scans. Which centers are prescribing them. What insurance did.

What I’m asking you to do

If you are taking, have taken, or were denied Ibrance, Verzenio, or Kisqali for appendix cancer, please join the registry. Use the link for where you live.

• United States residents: Join the registry (US)
• Everyone outside the United States: Join the registry (International)
• Already in the registry: Add or update your record to include your CDK4/6 drug, dates, tumor markers, and scans.

Every field is optional except your pathology report. Share what you know and skip what you don’t. You can add more later when new results come in.

If a form isn’t the right fit for you, email me at info@appendicure.com. With your permission, I’ll enter the information for you.

What happens to your information

The registry is IRB-reviewed. Appendicure runs it, and your information is encrypted. Your name and anything that identifies you stay protected. Researchers work with the data without names attached. You can withdraw at any time.

I’m using these records for two things, with Dr. Shen and Dr. Lowy. The first is a peer-reviewed paper on patient outcomes and the coverage barriers patients hit. The second, once that paper is published, is a formal request to update the guidelines for appendix cancer with GNAS and KRAS mutations.

The stories that belong in that paper are real. Patients who waited eight months and aged into Medicare just to get covered. Patients who got the drug through Pfizer or Lilly compassionate programs after insurance said no. Patients at UCSD, Mayo, MD Anderson, MSK, Yale, and other centers who are responding.

Why this matters now

The Ibrance patent is winding down and generic palbociclib is coming. Pfizer has little commercial reason to expand the label to appendix cancer. That leaves patient data as one of the main ways to get these drugs recognized for this disease.

Once the guidelines include these drugs, coverage becomes routine instead of a fight. Patients stop having to wait for Medicare. Oncologists stop writing three appeal letters per patient.

If you know someone on one of these drugs who hasn’t shared their story yet, please send them this post.

Amanda Moore
Founder/Caregiver/Advocate, Appendicure
info@appendicure.com