Pharmacy Law: Do Patients Have a Right to Investigational Drugs?

Author: Joseph L. Fink III, BSPharm, JD


Dr. Fink is professor of pharmacy law and policy at the University of Kentucky College of Pharmacy, Lexington.


Issue of the Case

Ordinarily, patients have access to medications only after they have received premarketing approval from the FDA based on that agency?s review of safety and efficacy data for the pharmaceutical product. Does the US Constitution supersede the Federal Food, Drug, and Cosmetic Act and extend to terminally ill patients a constitutionally protected right of access to experimental medications?

Facts of the Case

The case was filed by an advocacy organization known as the Abigail Alliance for Better Access to Developmental Drugs that represents terminally ill patients and those who support them. That entity was represented in the case by the Washington Legal Foundation, a public interest law firm that has taken on the FDA in the past on other issues. Its argument was that patients with terminal illnesses should have access to medications earlier in the drug-product approval system adopted by FDA. They based this view on an interpretation of the Fifth Amendment to the Constitution.

Following issuance of an investigational new drug approval to ship the drug product across state lines for research purposes, the product?s sponsor launches the clinical trial process for the study medications, which is usually divided into 3 phases?sometimes a fourth is added. Phase 1 testing uses a relatively small number of research subjects (eg, 20-80), who are administered the medication in an attempt to ascertain how it is metabolized, its possible pharmacologic activity, the relationship of increasing the dosage to development of side effects, and perhaps even an early indication of effectiveness for a particular disorder. Although this multitude of purposes can exist, the primary focus of phase 1 testing is on assessing the safety of the drug product for further testing in humans. The alliance sought to have medications that have completed only phase 1 evaluation available to terminally ill patients.

The alliance based its legal arguments on the Due Process clause of the Fifth Amendment, which provides, in part: "No person shall be ... deprived of life, liberty, or property, without due process of law...." Through a long series of decisions, the Supreme Court has expanded meaning of the wording to mean much more. This provision has been interpreted by the court to create an expanded protection of citizens against interference by the government with certain "fundamental rights and liberty interests." Based on case decisions over the years, these include ?the right to marry, to have children, to direct the education and upbringing of one?s children, to marital privacy, to use contraception, to bodily integrity, and to abortion.? The issue in question: Should that list be expanded to include a Due Process right to have access to experimental medications?

The federal trial court had ruled the answer was no, dismissing the case before a trial was held. Following an initial review by a 3-judge panel of the Court of Appeals, the court settled on an opposite view and ruled that the case should be sent back to the trial court for further proceedings. The alliance did not agree with everything in that decision and requested a review of the case by the full Court of Appeals bench (10 judges).

The Court?s Ruling

The Court of Appeals? judges en banc (in full court) considering this case ruled by an 8-2 vote that no such constitutionally protected right exists and that the trial court judge was correct in his decision that the case should be dismissed.

The Court?s Reasoning

The majority of the judges pointed out that the expansion of the list of protections falling under Due Process should not be added to lightly. Because the protected rights added to the list are not specified in the Constitution, the Supreme Court "has cautioned against expanding the substantive rights protected by the Due Process clause." This is so because "guideposts for responsible decision making in this uncharted area are scarce and open-ended."

The judges did an extensive review of both the historical evolution of the nation?s drug-regulation schema and the long line of cases interpreting the wording of the Constitution relevant to this case. They assigned great weight to the lack of proof that there exists "evidence of a right to procure and use experimental drugs that is deeply rooted in our nation?s history and traditions." The 2 dissenting judges wrote strong decisions taking exception to both the approach used and the conclusions reached by the majority.

This case was decided on August 7, 2007. On September 28, 2007, the alliance petitioned the Supreme Court to take the case. On January 14, 2008, the Supreme Court declined to take up the case, meaning that the ruling of the next lower court stands. In doing so, the Supreme Court issued no statement about why it declined to hear the case.