How to Find New Treatment Choices: Locating Clinical Trials and New Drugs in Development When Standard Treatment Is Not An Option

For patients with cancer, there are three main situations that may lead to a search for new treatments. First, if a standard treatment has not been successful. Second, if the cancer returns after being initially treated successfully. Third, when a person is faced with a diagnosis for which there is no effective standard therapy.

People needing new treatments may learn of them through news reports, their physicians, cancer advocacy organizations, support groups, or the Internet. Unfortunately, finding or taking advantage of these new treatments can be a daunting task.

This article, part of our Becoming Your Own Advocate series, seeks to help patients needing a new treatment approach find ready access to some of the most effective sources for locating information on newly emerging medical treatments.

WHAT ARE CLINICAL TRIALS?
Clinical trials are rigorously designed and closely monitored tests whereby scientists, physicians, and patients work together to find ways to improve health and patient care. Each study tries to answer scientific questions and to find better ways to prevent, diagnose, or treat disease. Studies are conducted to find out whether promising approaches to prevention, diagnosis, and treatment are safe and effective. There are several types of clinical trials:

» Treatment trials test new treatments, such as new cancer drugs, new approaches to surgery or radiation therapy, new combinations of treatments, or new methods such as gene therapy.
» Prevention trials test new approaches, such as medicines, vitamins, minerals, or other supplements that doctors believe may lower the risk of a certain type of cancer. These trials look for the best way to prevent cancer in people who have never had it or they seek to prevent recurrence or a new cancer in people who have already had a previous diagnosis.
» Screening trials test the best way to find cancer, especially in its early stages.
» Quality of Life trials, also called Supportive Care trials, explore ways to improve comfort and quality of life for cancer patients.

THE BENEFITS OF CLINICAL TRIALS INCLUDE (NCI, 2004):
» Health care provided by leading physicians in the field of cancer research.
» Access to new drugs and interventions before they are widely available.
» Close monitoring of your health care and any side effects.
» A more active role in your own health care.
» First to benefit if the treatment being studied is found to be helpful.
» An opportunity to make a valuable contribution to cancer research.
» Low-cost or free treatment (expenses paid for by researchers).

THE RISKS OF CLINICAL TRIALS INCLUDE (NCI, 2004):
» New drugs and procedures may have side effects or risks unknown to the doctors.
» New drugs and procedures may be ineffective or less effective than current approaches.
» No benefit even if the new approach is shown to work for other people.
» Potentially complicated logistics involved in participation, including a significant amount of paperwork and travel.
» Potential costs beyond what the trial sponsor and your insurance company will cover.

LOCATING DRUG CLINICAL TRIALS
The easiest way to find a drug that is in development and not currently approved by the FDA is through a clinical trial being conducted on that drug. To find such trials, there are several free internet-based resources to consult. These databases request anonymous information about your diagnosis and disease staging and most can sort the results by geographical location (See Online Resources below).

When you find a new drug clinical trial that interests you, the first step is to see if you are eligible to enroll. All the online resources listed on the next page provide the formal title of each trial, the eligibility criteria, and a contact name and telephone number. It can be helpful to review these eligibility criteria with your physician as they are usually very specific; your physician should be an important advocate in helping you gain access to a trial.

In most cases, the telephone number provided will allow contact with the principal investigator of the trial and it can be helpful to speak with them directly. In our experience, trial investigators are usually willing to speak with patients and their physicians.This is due, in part, to the fact that one of the most difficult steps in conducting a clinical trial is in the recruitment of patients.1,2

If there is a specific drug that you are interested in, but no clinical trials are listed in the online databases, contact the drug manufacturer directly for more information.

WHAT IF I AM NOT ELIGIBLE FOR CLINICAL TRIALS?
If you find yourself ineligible for currently available clinical trials, the next step is to pursue the possibility of Compassionate Use, also known as Expanded Access, Compassionate Exemption, Special Exemption, or Special Exception.

Compassionate Use is usually granted for patients with advanced disease, no approved treatment options, and no appropriate clinical trial options. It is often possible to get access to a new, unapproved drug outside of the clinical trial in which it is being tested.

There is currently no single listing of drugs in clinical trials available for compassionate use purposes. If there is a specific drug currently in clinical trials that you are seeking access to, one way to approach this is to search by drug name in Needymeds.com or CenterWatch.com to find the name and contact information of the manufacturer to whom you could send a compassionate use request.

REQUIREMENTS FOR COMPASSIONATE USE
In order to receive a drug for Compassion Use, the manufacturer of the new unapproved drug must allow access to their drug outside of a clinical trial. The FDA oncology medical officer overseeing that new drug’s development must also approve Compassionate Use access. There are several reasons why a drug company may refuse a Compassionate Use request:

» Lack of efficacy. There may be little or no evidence of effectiveness for your condition.
» Safety. The drug may have toxicity problems that have not yet been published.
» Production capacity. Prior to approval, manufacture capacity for the drug may be limited.
» Competing demands. The drug company may have plans for use of the drug in trials in other diseases.

WHAT IF THE COMPANY DOES NOT OFFER A COMPASSIONATE USE PROGRAM FOR THE DRUG I AM INTERESTED IN OBTAINING?
If none of the previously discussed options are available, access through Single Patient Compassionate Use may be possible. This is also called Single Patient Access or an Emergency Investigational New Drug (IND). This is done directly with the FDA, who may authorize shipment of the drug directly to your physician. Your doctor must first request permission for access to the drug from the manufacturer. Once received, they work together to apply for FDA approval. In an emergency, the FDA can walk-approve the paperwork in 24 hours. For instructions on how to do this, see Physician Request for a Single Patient IND for Compassionate or Emergency Use.

ARE THERE OTHER WAYS TO GET ACCESS TO A DRUG?
The “Group C” Treatment IND is a program established by the National Cancer Institute (NCI). It is a program that makes investigational drugs available outside a clinical trial,but only to specially authorized NCI oncologists. Group C drugs are study drugs in Phase 3 testing that have shown evidence of relative and reproducible efficacy in a specific disease (see What are the Phases of a Clinical Trial? below). Your physician may be able to facilitate such access for you by introducing you to an authorized NCI oncologist.

References
1. Corrie, P., J. Shaw, et al. (2003). “Rate limiting factors in recruitment of patients to clinical trials in cancer
research: descriptive study.” Bmj 327(7410): 320-1.
2. Friedman, L. M., C. D. Furberg, et al. (1998). Fundamentals of Clinical Trials. New York, Springer-Verla.

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What are the phases of clinical trials?

Most clinical research that involves the testing of a new drug progresses in an orderly series of steps, called phases. This process allows researchers to ask and answer questions in a way that results in reliable information about the drug while protecting the patients. Clinical trials are usually classified into one of three phases:

Phase I Trials: These first studies in people evaluate how a new drug should be given (by mouth, injected into the blood, or injected into the muscle), how often, and what dose is safe. A Phase I Trial usually enrolls only a small number of patients, sometimes as few as a dozen.

Phase II Trials: These studies continue to test the safety of the drug and begin to evaluate how well the new drug works. Phase II Trials usually focus on a particular type of cancer.

Phase III Trials: These studies test a new drug, a new combination of drugs, or a new surgical procedure in comparison to the current standard. A participant will usually be assigned to the standard group or the new group at random (called randomization). Phase III Trials often enroll large numbers of people and may be conducted at many doctors’ offices, clinics, and cancer centers nationwide.

Source: National Cancer Institute

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Online Resources For Locating Clinical Trials

CENTERWATCH
Contains information about clinical research, including listings of more than 41,000 active industry and government-sponsored clinical trials as well as new drug therapies in research and those recently approved by the FDA.

Special Features
· Drug Directories include comprehensive list of drugs recently approved by the FDA organized by therapeutic area.
· NMT Weekly Trial Results database provides the results of recently completed and ongoing clinical trials. The information in this resource is based on published materials from medical conferences,
journals, and company reports and interviews.
· CenterWatch Email Notification is a free service that notifies you when new trials are added to their site on therapeutic topics of interest. You can also set up email notifications to receive results of clinical studies and about drugs recently approved by the FDA in the categories you choose.

CLINICALTRIALS.GOV
Provides regularly updated information about federally and privately supported clinical research in human volunteers. More than 4400 trials are currently listed on this site.

Special Features
· Understanding Clinical Trials section includes explanations about clinical trials in general.
· Genetics Home Reference contains consumer information about genes and genetic conditions.
· NIH Health Information section contains research supported by the National Institutes of Health.

CURRENT CONTROLLED TRIALS
Published by BioMed Central, an independent publishing house. Includes a detailed listing of trials being conducted in Europe and the US.

Special Features
· ISRCTN Register is a database of randomized controlled trials with an International Standard Randomized Controlled Trial Number (ISRCTN). There are more than 1900 trials listed on this site.
· metaRegister of Controlled Trials (mRCT) is an international database combining registers of ongoing randomized controlled trials in all areas of healthcare. There are more than 5800 trials are listed on this site.

NATIONAL CANCER INSTITUTE
There are approximately 2100 cancer trials currently listed on the site, searchable by type of cancer (currently 150 different types), stage/subtype of cancer (usually Stage I through IV, and recurrent), type of trial (treatment, supportive care, screening, genetic, diagnostic), location of trial (within specified number of miles of a zip code which you supply). Site also features a dictionary of cancer related terms, results from recent clinical trials, and information in Spanish.