The National Health Service (NHS) in the United Kingdom has announced plans to halt funding through the Cancer Drug Fund for regorafenib (Stivarga), thereby preventing GIST patients from having access to this life-saving medication.
We at the Life Raft Group find this decision to be unacceptable, as UK patients will no longer have access to this effective treatment. The decision to “delist” the drug is additionally disconcerting, as it was approved last year as a third line treatment for patients with advanced Gastrointestinal Stromal Tumor (a rare type of cancer found in the digestive system). Regorafenib is used to treat GIST cancer that is unresponsive to existing drugs such as imatinib and sunitinib. It has proven very successful at stopping disease growth or causing significant shrinkage, and is better tolerated by patients. Younger patients with a rare form of “wildtype” GIST respond very well to regorafenib.
This essentially means that patients with GIST in the UK will no longer be able to access this medication that will provide them with valuable time with their families. The withdrawal of this drug from the Cancer Drug Fund means that patients whose GIST cancer is inoperable or has spread widely and is not being contained by the first and second line treatments licensed in the UK will have no other treatments available, and will die sooner than might have been the case if regorafenib was administered.
This is a direct contradiction of the purpose of the Cancer Drug Fund that was created in 2011, following a Conservative pledge that treatments should no longer be denied on the basis of cost. The Cancer Drug Fund was designed to make drugs available where there is no access through the NHS as in the case of drugs not approved or assessed by the National Institute for Health and Care Excellence (NICE).
Originally set up by the Coalition Government, The CDF had an initial budget across three years to fund the cancer drugs. The British government has extended the CDF’s run, and added additional funding until the end of March, 2016. However, the NHS is revising the way the CDF functions by adding an assessment in order to remove drugs with the “lowest levels of clinical benefits.”
The NHS states, “Drugs on the national list will be subject to an affordability test within the overall CDF budget, being added or removed according to their relative aggregate score using the national CDF Priorisation Tool of clinical benefit in conjunction with the assessment of the drug cost per patient. Drugs whose aggregate score is below the threshold applicable at the time will not be added or will be removed from the list.”
Research supports efficacy of Stivarga (regorafenib)
According to a study in the Lancet in January 2013, regorafenib in patients with metastatic or unresectable GIST who progressed after failure of first line and second line treatments of imatanib and sunitinib showed that “the drug provided a significant improvement in the progression-free survival compared with placebo in patients with metastatic GIST after progression on standard treatments.” (Prof. George Demetri, M.D. et al, Lancet, Volume 381, No. 9863, p. 295-302, January, 2013)
Furthermore, the results of an international phase three trial reported in 2012 that regorafenib had the potential to fill the unmet need for advanced GIST patients. The study involved 199 patients across 17 countries. Results from the trial showed a statistically significant improvement in progression free survival and the safety and tolerability of regorfenib for the treatment of metastatic GIST.
Following the decision, NHS has put in place some patient protections:
- Any patient currently receiving a drug through the CDF will continue to receive it, whether or not it remains on the list
- Drugs which are the only therapy for the cancer in question wil remain available through the CDF
- If the CDF panel removes a drug for a particular indication, some patients may be able to receive it in another line of therapy or receive an alternative CDF drug
- Clinicians can apply for their patients to receive a drug not available through the CDF on an exceptional basis (Source: Sarcoma.org.uk)
LRG’s Executive Director, Norman Scherzer stated: “Regorafenib continues to demonstrate its efficacy as a third line treatment for patients with GIST. Furthermore, there is currently no other alternative demonstrated by clinical trial data. The result of this decision in the UK is to therefore to condemn British patients to a premature death. This is simply unacceptable.”
The LRG intends to provide support for our fellow GISTers in the UK, adding to their advocacy efforts in any way feasible. Please stay tuned for updates. We are all in this boat together, and need to present a united front so that GIST patients worldwide receive the treatment they need.
Please go to the GIST Support UK website for more information. We support their efforts for reinstatement, and invite you to support them on social media, and to reach out to any UK contacts you may have to sign their petition.