Use FDA’s Processes to Speed US Patient Access to Cuban Lung Cancer Vaccine

Medical researchers in Cuba invented a vaccine, called CimaVax-EGF, for lung cancer. CimaVax has been tested in about 7 well-designed clinical trials in patients with advanced non-small-cell lung cancer (NSCLC), and used to treat about 5000 patients over the last 2 decades. This one of a kind vaccine costs about $1 per shot and patients need to take one shot every month. Compare that to the 11 drugs approved by FDA for treatment of NSCLC which costs thousands of dollars per month and carry extensive side effects. In clinical trials, patients getting this vaccine survived on an average 6-9 months longer compared to control with few side effects. The vaccine is particularly beneficial to younger patients with those less than 60 years seeing the maximum survival time of up to 2 years. There are reports that clinical trials have been initiated in Canada, Serbia, Japan and some other European countries. The vaccine works by inducing a robust immune reaction to epidermal growth factor (EGF). EGF plays a central role in the growth of tumors, so by depleting EGF levels in the blood, CimaVax blocks tumor growth leading to longer survival in cancer patients. Although the vaccine has been tested and used in the treatment of patients with NSCLC only, this has potential to help patients with cancers of the colon, kidney, and head and neck. However, due to the Cuban embargo, US patients could not get this vaccine. With the thawing of relations between the US and Cuba, this vaccine is finally coming to the US. Last year, the Governor of New York visited Cuba and got an agreement to bring this vaccine to the US. Roswell Park Cancer Institute of Buffalo, New York, announced its plans to conduct Phase 1 and 2 clinical trials with CimaVax under an IND with the FDA. Since the announcement almost a year ago, there is no indication of any clinical trial being initiated. The current projections are that it could take 5-7 years to get it to the US patients. It seems, based on the publicly available information, that the regulatory processes adopted to get it approved in the US is flawed. There exist several regulatory mechanisms in the US to expedite the availability of new promising therapies to patients, particularly those with life-threatening diseases. Companies can use data from non-US trials, and innovative regulatory pathways such as the breakthrough designation, priority review and orphan drug to drastically reduce the time to patients. Several things should happen. The clinical trials conducted by Cuban scientists were published in peer-reviewed journals and appear to be well-designed. Clinical study reports of the Phase 1 and 2 clinical trials conducted by the Cuban researchers should help eliminate the need to re-conduct Phase 1 and 2 studies in the US under an IND. The program in the US can directly commence with the Phase 3/pivotal clinical trial. Since NSCLC is an orphan indication, an orphan designation of CimaVax seems obvious. The clinical trial data demonstrates exceptional efficacy compared to other treatment options for stage IV NSCLC making CimaVax a good candidate for breakthrough designation and priority review. Due to the incentives available to the product, the developers can potentially reduce the clinical trial requirement to a relatively short clinical trial aimed at confirming the results of the previous clinical trials. The vaccine can also be made available immediately to the patients under a compassionate use IND. The product manufacturing facility in Cuba must meet the US GMP requirements verification of which should be the first step. FDA accepts non-US non-IND data in support of a market approval application. So once the quality of the clinical research in confirmed, CimaVax should have a very short path to the US patients and the US market. A lot depends on the quality of data available from Cuba; publicly available information indicates that Cuban researchers generated good quality data. With the vaccine being extremely inexpensive and treatment being so simple, any delay in getting this treatment to patients in the US should be avoided. It is possible that the developers are keeping the regulatory strategies under wraps for financial reasons. It is also possible that Cuban government has been less cooperative about sharing clinical data. However, these issues should be discussed publicly due to the nature of this deal. For the sake of the patients, hope this vaccine is available in US soon.