November 2018
BROADER ACCESS TO PCSK9 INHIBITORS FOR FH PATIENTS
PCSK9 inhibitor medication is now supported by the Pharmaceutical Benefits Scheme (PBS) for patients with heterozygous FH who need additional help to lower their LDL-Cholesterol levels. Sold under the trade name Repatha, the medication called evolocumab will now be more accessible to patients with FH.
What is PCSK9?
PCSK9 (proprotein convertase subtilisin/kexin type 9) is a naturally occurring protein in humans that slows down the process of clearing LDL from the blood stream.
If you’ve read information about FH, you’ll already be familiar with LDL Receptors and the fact that a deficiency in LDL Receptors is the main cause of FH. This is relevant to keep in mind because PCSK9 acts directly on the LDL Receptor.
PCSK9 particles stick to LDL Receptors and flag the receptors to be destroyed. If an LDL Receptor escapes being tagged by PCSK9, instead of being destroyed, it will continue to collect LDL from the blood. By inhibiting PCSK9, we can increase the number of LDL receptors available to clear LDL from the blood. More functioning LDL Receptors means lower blood cholesterol levels.
Why would I continue taking my old cholesterol tablets if I’m going to start taking a new medication?
PCSK9 acts independently to the pathways targeted by the standard cholesterol lowering medications known as either ‘statins’, which block cholesterol synthesis, or ezetimibe, which inhibits cholesterol absorption . PCSK9 inhibitor therapy is recommended to be taken together with a statin, ezetimibe, or both because they work safely together. The addition of PCSK9 inhibitor therapy has been shown to reduced LDL-cholesterol levels and lower the risk of heart disease.
How do I know if I’m eligible?
Access to PBS support for PCSK9 inhibitors has been approved for FH patients who cannot lower blood cholesterol to certain levels with standard medications, or those who cannot tolerate standard medications. Many FH patients manage to lower LDL-cholesterol levels sufficiently with current routine management, including;
- Following a low cholesterol diet,
- Practising healthy lifestyle habits, such as avoiding smoking and regular exercise
- Taking standard lipid lowering tablets such as Atorvastatin, Rosuvastatin and Ezetimibe.
If routine management is effective at lowering cholesterol levels, additional PCSK9 inhibitor therapy may not be needed. However, if circumstances change, FH patients who need extra help with managing cholesterol levels can be reassured that access to PCSK9 inhibitors is more affordable under PBS authorisation.
Key facts about PBS Criteria for Repatha for FH
- The diagnosis of FH can be made with the Dutch Lipid Clinic Network Score OR a genetic test result. A genetic result is not a requirement.
- All FH patients must be following a low cholesterol diet and healthy lifestyle advice.
- A standard cholesterol lowering medication (statin) should already be included in regular cholesterol management. If side effects have caused problems in the past, details need to be assessed by your doctor to meet the PBS criteria eligibility.
- A recent cholesterol result is required. A threshold of LDL-Cholesterol levels applies. There are different thresholds for FH patients who have heart disease and those who do not have heart disease.
- If your cholesterol levels are already lower than the threshold, you may not need to add PCSK9 inhibitors to your regular medications.
- If you are already receiving Repatha under the PBS, you should continue to remain eligible. Your specialist may choose to write a new application for a new authority prescription if they consider your access to be more appropriately covered by the new changes.
- If you were previously ineligible, the new slightly relaxed rules may enable you to gain PBS access. Your specialist will have to submit a new authority prescription application.
For more information about the PBS criteria for subsidised access to the PCSK9 inhibitor drug, Repatha, please consult with your specialist. The prescription for Repatha must be completed by a medical specialist (not including general practitioners).
This article does not cover all PBS criteria for Repatha and is meant for general information only. Each individual will have unique circumstances and should discuss personal details with their doctor.
For more information about FH, genetic testing and management, please feel free to contact the FH Registry and refer to the reliable information compiled on the FH Registry website.
By 
Catherine Spinks, David Sullivan, and Jerome Herren
From the FH team at Royal Prince Alfred Hospital
Copyright © 2018 FH Australasia Network. All rights reserved.
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