Access to Investigational Drugs
Investigational or experimental drugs are new drugs that have not yet been approved by the FDA or approved drugs that have not yet been approved for a new use, and are in the process of being tested for safety and effectiveness.
Patients may decide to seek access to investigational drugs for different reasons. Some patients with serious or life-threatening illnesses seek treatment with investigational drugs if FDA-approved therapies are not working or if their side effects are too severe. Others may have heard about promising early study results for a specific investigational drug, and they might want to learn more.
Investigational drugs are available through two pathways designed to protect patients, because an investigational drug may pose unknown risks to patients and we do not know if it is effective. Patients may be eligible to receive an investigational drug as (1) a participant in a clinical trial, or (2) as part of an expanded access program.
Access to Investigational Drugs, For Consumers, U.S. Food & Drug Administration, August 12, 2009.
Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access)
Expanded access, sometimes called “compassionate use,” is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options.
FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial. They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.
Just as in clinical trials, these investigational drugs have not yet been approved by the FDA as safe and effective. They may be effective in the treatment of a condition, or they may not. They also may have unexpected serious side effects. It is important for you to consider the possible risks if you are interested in seeking access to an investigational drug.
Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access), For Consumers, Food & Drug Administration, August 8, 2009.
“Educational Materials About Clinical Trials (Introduction to Clinical Trials; Patient Costs; Special Topics; and NCI Programs that Conduct Clinical Trials);” National Cancer Institute.
“Clinical Trials,” Cancer.Net.
“How To Find A Clinical Trial,” American Association for Cancer Research (AACR), October 19, 2007.
“ClinicalTrials.gov — A Service of the National Institutes of Health.”
“Oncology Clinical Trials Information for Patients (Clinical Trials; Finding Clinical Trials; and View the NCCN Cancer Resource Links), National Comprehensive Cancer Network (NCCN).
“Welcome to eCancerTrials (About Cancer Trials; Cancer Drugs; Find a Clinical Trial),” ECancerTrials.com.
“Clinical Trials (Introduction; What Are Clinical Trials?; Phases of Clinical Trials; How Are Clinical Trials Conducted?; Clinical Trial Safeguards; Participating In Clinical Trials; The Cost of Clinical Trials; Finding Specific Clinical Trials; and The Future of Clinical Trials),” University of Florida Shands Cancer Center.
“How to Find a Cancer Treatment Trial: A 10-Step Guide,” National Cancer Institute, March 21, 2005.
“Cancer Clinical Trials (What Are Cancer Trials; Ways to Actively Participate in Your Treatment),” The Wellness Community.
“Clinical Trials and Special Patient Populations – What role do patients’ ethnicity, sex, age and geographic location play in cancer research and treatment?,” by Alex A. Adjei, MD, PhD, senior vice president of clinical research and the chairman of the department of medicine at the Roswell Park Cancer Institute in Buffalo, N.Y., American Association For Cancer Research, February 6, 2007.
“Ovarian Cancer – Clinical Trials,” Johns Hopkins Medical Institute, July 8, 2002.
“Clinical Trials (State Listing): Ovarian Cancer,” CenterWatch Clinical Trial Listing Service.
Search Results for “Ovarian Cancer,” Center Watch Clinical Trial Listing Service.
“Find Cancer Clinical Trial Option,” Emerging Med.com
“Search for Clinical Trials: Basic Search,” National Cancer Institute.
“TrialCheck,” Coalition of Cancer Cooperative Groups.
ClinicalTrials.gov – Ovarian Cancer (Open) Clinical Trial Search Results Based Upon the Following Search Terms:
“Ovarian Cancer and Intraperitoneal Chemotherapy”
“Ovarian Cancer AND Radiation Therapy”
“Ovarian Cancer AND Paclitaxel [Taxol®] and Carboplatin”
“Ovarian Cancer AND Paclitaxel [Taxol®] and Cisplatin”
“Ovarian Cancer AND docetaxel [Taxotere®] and Carboplatin”
“Ovarian Cancer AND docetaxel [Taxotere®] and Cisplatin”
“Ovarian Cancer AND Pegylated Liposomal Doxorubin” [Doxil®]
“Ovarian Cancer AND Gemcitabine” [Gemzar®]
“Ovarian Cancer AND Topotecan hydrochloride” [Hycamtin®]
“Ovarian Cancer AND Bevacizumab” [Avastin®]
“Ovarian Cancer AND antiangiogenesis”
“Ovarian Cancer AND Sunitinib” [Sutent®]
“Ovarian Cancer AND Sorafenib” [Nexavar®]
“Ovarian Cancer AND monoclonal antibody”
“Ovarian Cancer AND Trastuzumab” [Herceptin®]
“Ovarian Cancer AND Tyrosine Kinase Inhibitor”
“Ovarian Cancer and targeted therapy”
“Ovarian Cancer and anti-estrogen”
“Ovarian Cancer AND immunotherapy”
The patient is my brothers wife .She has under gone surgery twice and chemo for the last 5yrs . Now she is not finding any improvement thu chemo and the Dr has stoped giving any more Chemo . He has advised her to try prep inhibitor.if available and allowed to prescribe or use .
pl tell me the possibility to get it and start treatment
Sohinder, I am so sorry to hear about your sister-in-law, who, as I understand, has recurrent ovarian cancer and resides in Delhi, India. It is also my understanding that her doctor has recommended use of a PARP inhibitor. First, I am providing you with a hyperlink to open ovarian cancer clinical trials (obtained through clinicaltrials.gov) that involve the study of various PARP inhibitors. CLICK HERE to view a list of those trials. I am also providing you with a list of open “solid tumor” (which generally includes ovarian cancer) that involve the study of PARP inhibitors. CLICK HERE to view those trials.
Unfortunately, the open PARP inhibitor clinical trials provided by the hyperlinks above are being conducted in the U.S. and U.K. In this regard, I have three suggestions.
First, each clinical trial summary lists a contact person or trial investigator. If you contact each trial investigator, you may be able to establish whether or not such drugs are being tested in India.
Second, I have found a list of “world class” India Cancer Hospitals. I have no personal knowledge about these hospitals, but your sister-in-law’s doctor(s) should be aware of their reputations within the local medical community. The hospitals are provided below.
—Apollo Hospital, Chennai, India
—Apollo Specialty Hospital, Chennai, India
—Apollo Hospitals, Bangalore, India
—Indraprastha Apollo Hospital, Delhi, India
—Fortis Hospital, Noida, India
—Narayana Cancer Hospital, Bangalore, India
—Artemis Hospital, Gurgaon ( Delhi ) , India
You and your doctor can contact these hospitals and obtain information regarding the availability of PARP inhibitor clinical trials.
Third, if your sister-in-law possesses UK citizenship, it may be possible to obtain treatment through the NHS in the U.K. I know that Cancer Research U.K. is conducting a Phase II clinical trial of PARP inhibitor AG014699 in patients with advanced ovarian cancer. You or your sister-in-law’s doctor could contact Cancer Research U.K. to determine all available ovarian cancer clinical trials that are testing PARP inhibitors. CLICK HERE to view a list of open ovarian cancer clinical trials being conducted by Cancer Research U.K. CLICK HERE to review general NHS clinical trial information.
I trust this information is helpful. If you have any further questions, don’t hesitate to contact us. Best regards, Paul
My sister has ovarian cancer with first line chemo not effective. I was told your web can help identify the clinic trails on new drugs. I am under the impression that when provided the condition, your site helps find the relevant trails for the particular condition. Can you kindly let me know how that is done using your resources here.
I am so sorry to hear about your sister. Our thoughts & prayers are with her. It is important for you to know that Libby’s H*O*P*E* does not provide medical advice. You should discuss any involvement in clinical trials with your sister’s doctor — preferably a board-certified gynecologic oncologist. The reason for this recommendation is that referral to a particular clinical trial is generally not done until at least two lines of standard treatment fail. Notwithstanding, any such recommendation should be based upon the totality of your sister’s case facts (e.g., including subtype of your sister’s ovarian cancer, age, results from primary debulking surgery, performance of any intra-peritoneal chemo, results from specialized histology testing, etc.) Without more facts, it is extremely difficult to provide general clinical trial information.
If you discuss your sister’s case with her doctor and have questions after that discussion, we’ll be happy to address specific questions based upon that discussion.