Nick Auden, 40, is a husband and father to three young children. Diagnosed with stage 4 melanoma, his doctors told him he has mere months to live and a zero percent chance of survival. Not willing to accept his fate without a fight, Nick pursued a clinical trial for a new drug called PD-1, which has shown promising results and could give him a 50 percent chance of survival. After being accepted into the clinical trial, he was subsequently kicked out after he suffered a bowel obstruction and, therefore, became disqualified to participate in the trial. Desperate to save their loved one, Nick’s family started an online petition to persuade the drug company to grant him compassionate use of the drug.
The Doctors reached out to the drug companies that produce PD-1, Merck and Bristol-Myers Squibb, and received this response from Bristol-Myers Squibb:
Bristol-Myers Squibb is committed to developing medicines in areas of high unmet medical need. In many cases, our medicines are being studied as potential treatments for patients with limited to no viable treatment options. It is exactly with these patients in mind that, through regulatory approval, we work to ensure broad access to our medicines as quickly as possible, while also remaining mindful of protecting patient safety.
In the long term, the best way to ensure the broadest access for patients is to successfully register a medicine with health authorities through the conduct of well-controlled clinical trials.
In certain cases, Bristol-Myers Squibb has offered investigational medicines in late stage development to health care providers for the treatment of patients outside of clinical trials, after evaluating the degree of unmet medical need, the available of other treatment options, and the extent of available safety and efficacy data on the compound at the appropriate dose.
While nivolumab has demonstrated potential based on early (Phase 1) data, there is not enough information on its use in humans at any given dose to establish a benefit/risk profile that would support its use outside of a well-controlled clinical trial. Bristol-Myers Squibb is committed to establishing the benefit/risk profile of nivolumab as quickly as possible and we are closely collaborating with health authorities. Our Phase 3 program started last year and there are multiple clinical trials ongoing.
We empathize with patients who have limited treatment options and will continue to assess available data on nivolumab to determine if the established benefit/risk profile allows for expanded access use outside a well-controlled clinical trial in the future.