Andrea Sloan, a 7-year survivor of stage 3c ovarian cancer, is seeking a ”compassionate use” exemption from pharmaceutical company BioMarin to save her life. Sloan is scheduled to start treatment at the M.D. Anderson Cancer Center on September 5 and would like to obtain access to the “PARP 1/2 inhibitor” drug known as “BMN 673″ by that time. A robust grassroots campaign in support of Sloan has emerged on social media and Change.org in an effort to get an affirmative response from BioMarin.
Drugs that are being tested but have not yet been approved by the U.S. Food and Drug Administration (FDA) are called “investigational drugs.” These drugs are generally available only to people who are taking part in a clinical trial. The FDA “Expanded Access” protocol – sometimes referred to as a “compassionate use” exemption — involves the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition, who has no comparable or satisfactory alternative treatment options.
FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial. Before an investigational drug can qualify for compassionate use, the patient’s physician, the FDA, and the drug manufacturer must approve such use.
Unfortunately, drug manufacturers may not always be willing or able to provide access to a drug outside of their clinical trials. By law, drug companies are not required to make their drug available through the FDA expanded access protocol, or to make more of a drug for that purpose.
Andrea Sloan, a 7-year survivor of stage 3c ovarian cancer, is seeking a compassionate use exemption from pharmaceutical company BioMarin to save her life. Sloan is scheduled to start treatment at the University of Texas M.D. Anderson Cancer Center on September 5, and she would like to obtain access to the “PARP 1/2 inhibitor” drug known as “BMN 673” by that time. A robust grassroots campaign in support of Sloan has emerged on social media and Change.org in an effort to get an affirmative response from BioMarin.
“BioMarin’s BMN 673 offers me the best chance at a long life,” said Sloan. “My doctors and the FDA agree that I am an excellent candidate for this drug and meet the criteria for compassionate use exemption. However, BioMarin’s lack of a policy on compassionate use is preventing me from gaining access to the drug I need to save my life. I respectfully implore them to reconsider and make the ethical decision to help me.” [Emphasis added]
Sloan has endured two full rounds of chemotherapy, five surgeries, and a stem cell transplant. While her cancer remains responsive to treatment, her bone marrow can no longer tolerate traditional therapies. Her world-class oncology team at M.D. Anderson believes that BioMarin’s PARP inhibitor BMN 673, which is currently being tested in a phase I solid tumor clinical trial, is the best option for Sloan’s BRCA-1 gene-mutated form of ovarian cancer.
Unfortunately, Sloan hit a barrier in gaining access to BMN 673. Further enrollment of ovarian cancer patients in the phase I solid tumor trial is now closed, and the publicly announced portion of the trial that will be entering phase III testing is only open to BRCA gene-mutated breast (but not ovarian) cancer patients. Therefore, Sloan is left with the compassionate use exemption as her only option to access the drug she needs to fight her cancer. Based on FDA requirements, Sloan qualifies for the compassionate use exemption. Data emerging from the phase I BMN 673 study suggest that the drug is a safe and effective treatment option for patients with BRCA gene-mutated ovarian cancer.
Moreover, on August 16, 2013, BioMarin announced that its medical study abstract, entitled “PARP inhibition with BMN 673 in ovarian and breast cancer patients with deleterious mutations of BRCA1 and BRCA2,” has been selected as a “late breaking” abstract by the 17th ECCO — 38th ESMO — 32nd ESTRO European Cancer Congress, which will be held from September 27 through October 1, 2013 in Amsterdam, The Netherlands. BioMarin’s oral presentation at the European Cancer Congress (scheduled for September 29, 2013) will include data presented from 28 ovarian cancer patients with deleterious germline (inherited) BRCA gene mutations, including 17 patients from the phase I BMN 673 trial dose escalation cohort (range 100 µg to 1100 µg) and 11 patients from the dose expansion cohort. Presumably, the most recent ovarian cancer patient data to be presented at the European Cancer Congress will expand upon the positive data presented by the company at the 2013 Annual Meeting of the American Society of Clinical Oncology, which indicate that positive RECIST (“Response Evaluation Criteria in Solid Tumors“) and/or CA-125 ovarian cancer patient responses occurred at BMN 673 drug doses ≥ 100 µg/d in 11 out of 17 (64%) BRCA gene-mutated ovarian and peritoneal cancer patients. The positive RECIST medical imaging findings and the CA-125 blood test results highlight the promising effectiveness of BMN 673, albeit among a small group of ovarian cancer patients.
BMN 673 is a targeted therapy designed to disrupt the tumor without traditional chemotherapy drug side effects, thereby making it an optimal treatment for Sloan.
Despite Sloan’s best efforts, she has been unable to convince BioMarin to allow compassionate use of BMN 673. BioMarin, according to Sloan, has not been cooperative, merely citing their lack of a policy on the issue. Sloan, the executive director of a non-profit that advocates for survivors of domestic violence and abuse, now finds herself forced to publicly advocate for herself in a last chance effort to save her life. Sloan is committed to advocating for meaningful reform on this topic and hopes BioMarin will lead by example in starting a national dialogue.
For those interested in supporting Andrea Sloan, please sign her petition on Change.org that urges BioMarin to grant her a compassionate use investigational cancer drug exemption for BMN 673. Also, you can follow Andrea on Twitter at @andi_sloan.
Yesterday, BioMarin issued a statement to KXAN, an Austin, Texas local affilate of NBC, in response to a in-depth news story that KXAN aired tonight regarding Andrea Sloan’s dire situation. Effectively, BioMarin rejected Andrea Sloan’s request for compassionate use of BMN 673, although its statement was worded as a general drug “expanded access” policy explanation.
BioMarin acknowledged that it allowed preapproved expanded access to one of its investigational drugs which completed phase III clinical testing last year. In terms of general guidelines for its expanded access programs, the company stated:
“We implement these [expanded access] programs when we have sufficient scientific evidence to support both the safety and the efficacy of a product for an indication. Additionally, we implement these programs only when we can ensure that access will be provided equitably, ensuring that the process is appropriately blinded, and when we are confident that the expanded access will not inhibit our clinical trial plans or clinical trials for a disease generally.”
In terms of Andrea Sloan’s specific case, BioMarin stated that it does not comment on the status of individual patients. Apparently in a refusal to grant expanded access to any preapproved patient who requests compassionate use of BMN 673 prior to completion of phase III drug testing, the company stated:
“… However, we note that, although the current data [for BMN 673] that we have looks promising, there is no data at this point to support anything beyond dosing and some preliminary safety. It is too early to know if the experimental therapy is safe or effective, or will even prolong life, until we conduct the appropriate Phase 3 trials. The data that we have is from an ongoing early stage clinical trial, and it is the first trial that we have ever done with this therapy in humans.”
Accordingly, it appears that BioMarin’s current expanded access policy for its investigational drugs, such as BMN 673, will only extend to drugs that have already completed phase III clinical trial testing.
- Ovarian Cancer Survivor Andrea Sloan Seeks Compassionate Use Exemption From BioMarin to Save Her Life, Press Release, Digital Journal, August 29, 2013.
- BioMarin Provides BMN 673 Program Update, BioMarin Pharmaceutical Inc, Press Release, July 25, 2013.
- De Bono JS et. al. First-in-human trial of novel oral PARP inhibitor BMN 673 in patients with solid tumors. J Clin Oncol 31, 2013 (suppl; abstr 2580).
- Shen Y. et al. BMN 673, a novel and highly potent PARP-1/2 inhibitor for the treatment of human cancers with DNA repair deficiency. Clin Cancer Res. 2013 Jul 23. PMID: 23881923 [Epub ahead of print]
- A Phase 1, First in Human, Single-arm, Open-label Study of Once a Day, Orally Administered BMN 673 in Patients With Advanced or Recurrent Solid Tumors. ClinicalTrials.gov Identifier: NCT01286987.
- Advocate for others needs help in a fight for her life, by Shannon Wolfson & Joe Ellis, In-Depth Investigation, KXAN News, August 29, 2013.
- Statement From BioMarin on Expanded Access, August 28, 2013.
- BioMarin Announces Oral Presentation of BMN 673 Most Recent Data on Breast and Ovarian Cancers at the European Cancer Congress 2013, Press Release, August 16, 2013.