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Focus on Regulation

Federal Court Determines that Patient Has Liberty Interest in Receiving Investigational Drug

On May 17, 2016, a federal judge, citing arbitrary and capricious decisionmaking by FDA and notice-based due process concerns, granted a plaintiff’s emergency motion for a temporary restraining order against FDA, thereby preventing FDA from putting a clinical drug trial on hold with respect to the plaintiff, Eugene “Neil” Fachon. Fachon, a 20-year old student diagnosed with a form of brainstem cancer called Diffuse Intrinsic Pontine Glioma, was the sole participant in a Phase II study designed to enroll 10 to 40 patients for Antineoplaston therapy at the Burzynski Research Institute, Inc., in Houston, Texas.

The Fachon decision runs contrary to existing case law on patient access to investigational drugs. For example, the appellate court decision in Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach, 495 F.3d 695 (D.C. Cir. 2007), cert denied 552 U.S. 1159 (2008), held that that patients have no right to “a potentially toxic drug with no proven therapeutic benefit.” The case was filed against FDA by the Abigail Alliance for Better Access to Developmental Drugs (“Abigail Alliance”), an organization formed by the father of a college student with head and neck cancer, seeking access to the experimental drug, Erbitux. At the time of the lawsuit, Erbitux was available only in clinical trials examining the efficacy and safety of the drug in colon cancer. Abigail Alliance argued that terminal cancer patients have a constitutionally protected right to access experimental medications before the FDA approves them, but the D.C. Circuit held that this theory was not embodied in the Constitution and ran contrary to the FDA regulations which permit access to experimental drugs only in limited circumstances.

As additional background, we note that the Burzynski Research Institute and its founder, Dr. Stanislaw R. Burzynski, have received FDA, Texas medical board, and media attention for decades, notably for the alleged promotion and use of unapproved cancer drugs, questionable methods, and unsafe patient practices. Notably, the Texas Medical Board filed a second amended complaint seeking to revoke Dr. Burzynski’s medical license before the Texas State Office of Administrative Hearings on September 24, 2015. The administrative action is currently ongoing.

We are monitoring the court’s docket in the Fachon litigation and will provide additional updates as the case progresses. The case is Fachon v. United States Food and Drug Admin., et al., No. 1:16-cv-00222-M-LDA (D. RI filed May 17, 2016).

Additional Background

Under current law and FDA regulations, drug manufacturers may stop supplying investigational drugs for any reason whatsoever. While litigation typically arises from the patient’s use of investigational drugs, manufacturers may also be sued for denying patient access to investigational drugs. In cases of the latter category, courts have generally denied the validity of such claims.

For example, in April and June 2005, two separate groups of Parkinson’s disease clinical trial participants sued Amgen Inc., seeking to force it to continue to provide the investigational drug glial cell line-derived neuroptic factor (“GDNF”) after the company terminated the Phase II clinical trial in which the plaintiffs were enrolled. Suthers, et al. v. Amgen, Inc., No. 05-CV-4158(PKC) (S.D.N.Y. filed April 26, 2005); Abney, et al. v. Amgen, Inc., No. 5:05-CV-254-JMH (E.D.Ky. filed June 21, 2005). The study was designed to determine whether GDNF was a safe and effective treatment for Parkinson’s disease and involved 34 patients at eight sites. While the consent document provided that the study participants could elect to continue treatment for 24 months after the end of the study, it also provided that the study’s investigators could withdraw a subject from the study if they found that the study entailed more risk than benefit for the subject or if the study’s sponsor decided to discontinue the study for various scientific reasons. In September 2004, Amgen terminated the GDNF clinical study because of two newly-discovered safety concerns and the lack of statistical evidence of drug efficacy relative to placebos. FDA agreed with Amgen’s decision to terminate the study, finding it reasonable in light of the scientific evidence.

The GDNF plaintiffs sought preliminary injunctions requiring Amgen to continue providing them with GDNF, arguing that: (1) GDNF was beneficial to them and that Amgen had contracted with them to supply GDNF as long as it proved beneficial; (2) Amgen made promises, on which they detrimentally relied by undergoing pump implantation surgery, which were enforceable pursuant to the law of “promissory estoppel;” and (3) Amgen breached a fiduciary duty owed to them by unreasonably denying them access to GDNF. Both courts denied the requests for extraordinary relief and plaintiffs appealed. Although the New York plaintiffs withdrew their appeal, the Sixth U.S. Court of Appeals in Cincinnati, Ohio upheld the Kentucky federal court’s ruling.

The Fachon court departs from the precedent created by the GDNF and Abigail Alliance cases and goes even further by second-guessing FDA’s determination that a clinical study should not proceed. If the decision stands, it threatens to create a new basis for clinical trial participants to obtain experimental treatments outside of the FDA framework.

There is, obviously, a tension between providing early availability to investigational drugs and the lack of sufficient data to assess a reasonable expectation of clinical benefit, as compared to the potential risk of injury. Indeed, many clinical trials are discontinued during Phase II as manufacturers obtain additional clinical data points and begin to understand the risk-benefit calculus of the investigational drug. As such, providing earlier access to investigational drugs inherently elevates the potential risk of product liability claims.