The shortage of some cancer drugs is having a significant negative impact on
current and future cancer clinical trials in the Cancer Cooperative Groups,
which conduct their research primarily with public funding through the National
Cancer Institute. Of the drugs on the most current shortage list, 22 are cancer
agents. Approximately half of all active cooperative group cancer clinical
trials have at least one drug on the shortages list. There are approximately
400 active studies across the Cooperative Groups at this time.
Many chemotherapy drugs on the list are older sterile injectables that are the
standard-of-care treatment for various types of cancer (see supplemental
chart). Such drugs are widely utilized in the Cooperative Group setting, mainly
due to the fact that placebos (sugar pills) are rarely used in cancer clinical
trials, and are never used alone if an acceptable treatment is available. The
Cooperative Groups design and conduct randomized phase III, and large phase II,
clinical trials, many of which compare the safety and efficacy of a potentially
new treatment regimen against, or in combination with, standard treatment. For
example, new radiotherapy approaches are being tested in combination with the
standard chemotherapy paclitaxel, which is in short supply.
There are a number of new therapeutic approaches being evaluated throughout the
Cooperative Group system, involving a variety of vaccines, new signal
transduction agents, and biologic approaches. In most instances, such new
approaches either build on existing therapies or are directly compared to
available treatments. In either case, having access to agents which are well
established in cancer care is essential to the design of these trials.
The unpredictable nature of the shortages makes it very difficult to quantify,
predict and monitor the impact across the system. Multiple research sites
participate in any given Cooperative Group study, ranging from large
comprehensive cancer centers and academic medical centers to community
hospitals and small oncology and radiology practices. When a drug utilized in a
clinical trial is in short supply or is no longer being manufactured, it
compromises the sites’ ability to enroll patients in trials. The impact of a
shortage extends across this large network—there are nearly 1,800 research
sites affiliated with the Cooperative Groups.
Large, randomized Cooperative Group trials enroll hundreds, sometimes
thousands, of patients. When research sites affiliated with the Groups are
unable to accrue patients into clinical trials due to lack of available drug,
it delays the overall pace of the trial because it takes longer to accrue the
necessary number of patients for the data collected in the trial to be
It is NOT a common practice for a Cooperative Group to halt a clinical trial
due to a drug shortage. Availability of drugs in short supply is a highly fluid
situation across the Group-wide network, and the Groups seek to keep studies
open so that physicians can enroll patients whenever drug is available.
Delaying accrual slows the pace of scientific research because the longer it
takes to accrue the necessary number of patients onto a trial, the more it
delays completion of the trial. Slowing the pace of cancer research wastes the
investment made in the treatment, data management and time investment by the
patients and clinical scientists participating in the study.
Patients may be denied the opportunity to participate in a trial for which they
are eligible, due to lack of available drug. It is a common practice among
research sites to ensure availability of drug before enrolling a patient onto a
clinical trial. It is also a common practice to select an alternative, standard
treatment when lack of drug would cause a delay in treatment for a patient.
Treatment delays of days to months are critical in the life of a cancer patient
and could limit their chances for a cure or remission of their disease.
Cooperative Groups address the impact of drug shortages on a trial-by-trial
basis, in collaboration with the National Cancer Institute. A substitution of
drug is permitted in some clinical trials, to allow for utilization of
alternative regimens that were not part of the original protocol due to a
shortage of the existing drug being used as part of the investigation. However,
for other clinical trials, particularly those with FDA registration
implications and requirements, substitutions of drugs used in the trial are not
Another residual impact of drug shortages is the delay in obtaining the data
necessary to bring new cancer therapeutics to patients. With more than 400
cancer agents in various stages of development, it is imperative that cancer
clinical trials continue uninterrupted in order to obtain the necessary data to
seek approval of new anti-cancer drugs as soon as possible.
Diane D. Colaizzi, Executive Advisor,
Participation in Congressional Testimony Submitted by the National
Coalition for Cancer Research (NCCR).
Society of Health-System Pharmacists for current shortage listings,
bulletins, and other info.
Frequently Asked Questions about Drug Shortages
Energy and Commerce Committee Website. Hearing, Subcommittee on Health,
Friday, September 23, 2011 “Examining the Increase in Drug Shortages.” Visit
this website to watch archived video of the hearing, read witness testimonies,