Expanded Access: Programs and Possibilities

Current Expanded Access Programs

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The FDA requires that all cancer expanded access programs be listed on the government website www.clinicaltrials.gov. To directly access information on Expanded Access programs that are open for enrollment, from the ClinicalTrials.gov home page, click on "Search for Clinical Trials." You will then see a page called "Basic Search" with an open box for you to enter the topic of your interest. Skip this and instead, click on the "Advanced Search" tab at the top of the page. This will open a page with many open boxes. Ignore all of them except the fourth entitled "Study Type" which will have the term "All Studies" filled in. Use the drop-down button to select "Expanded Access Studies." Then go to the fifth open box entitled "Conditions" and type in the word "Cancer." Then push the "Search" button. This will take you to the page listing both older closed and new open Expanded Access Programs for various types of cancer approved by the FDA. Note that if you are interested in an Expanded Access program for a condition other than cancer, you can either leave the box blank to see all programs for all conditions, or fill in the name of the condition you are looking for.

You can also try clicking on this link to go directly to the list of cancer Expanded Access Programs at ClinicalTrials.gov: Cancer Clinical Trials

Currently Open Expanded Access Programs

The best source of up-to-date information on open expanded access programs is the ClinicalTrials.gov site. We highlight some interesting programs here, but our listing is not complete nor updated as often as the government site.

Afatinib for treatment of advanced EGFR-mutated non-small cell lung cancer

On September 13, 2012 Boehringer Ingelheim Pharmaceuticals announced the initiation of an expanded access program for its experimental lung cancer drug. Afatinib is an oral drug that has demonstrated positive results in a large phase 3 clinical trial called the LUX-Lung 3 trial.

Summaries of the technical details are available in the abstract published by the American Society for Clinical Oncology (ASCO) for its June 2012 annual meeting ( http://clinicaltrials.gov/ct2/show/NCT01649284?term=afatinib&type=Expn&rank=1 ) and also in commentary published by ASCO in its magazine for physicians, the "ASCO Post" (http://www.ascopost.com/issues/july-15-2012/first-line-afatinib-superior-to-standard-chemotherapy-in-advanced-egfr-mutated-non%E2%80%93small-cell-lung-cancer.aspx ).

Additional information on the expanded access program is available at ClinicalTrials.gov ( http://clinicaltrials.gov/ct2/show/NCT01649284?term=afatinib&type=Expn&rank=1 ) and also via the Boehringer Ingelheim Call Center (1-800-243-0127) or via email: clintriage.rdg(at)boehringer-ingelheim.com.

T-DM1 for Treatment of certain types of Metastatic Breast Cancer

On February 22, 2013, the FDA approved Kadcyla (ado-trastuzumab emtansine or T-DM1) in the U.S. for the treatment of people with HER2-positive metastatic breast cancer who have received prior treatment with Herceptin (trastuzumab) and a taxane chemotherapy. The drug was developed by Genentech, and the company (a member of the Roche Group) expects Kadcyla to available to patients in about two weeks.

T-DM1 (Kadcyla) is an antibody-drug conjugate developed by Genentech in association with ImmunoGen. An antibody-drug conjugate is composed of an antibody linked to a drug in a manner that allows the drug to be released once the antibody binds to its target. In this case, the antibody used is called trastuzumab, also known by the product name Herceptin, which targets HER2-positive breast cancer cells. The drug that is linked to the antibody is called mertansine and it interferes with tubulin, a protein essential for cell division. The idea of an antibody-drug conjugate is to enable potent doses of a highly toxic drug to be targeted precisely to only the cells that need to be killed, in this case HER2-positive breast cancer cells, without doing too much damage to normal cells.

In a Phase II clinical trial, T-DM1 shrank tumors in one-third of women who had received extensive prior treatment for advanced HER2-positive breast cancer. In some cases, T-DM1 was useful even for women whose disease progressed during treatment with Herceptin. Genentech has several clinical trials of T-DM1 underway or planned.


RO5185426 for Treatment of a subset of Metastatic Melanoma -- FDA APPROVED -- The expanded access program is no longer active. The drug (now called Zelboraf or vemurafenib) was approved by the FDA for the treatment of BRAF V600 mutation positive late-stage melanoma on August 17,2011, by Health Canada on February 15, 2012, and by the European regulatory authority on February 20, 2012.


Carfilzomib for Treatment of certain types of Relapsed and Refractory Multiple Myeloma

This is a multi-center, expanded access, open label study of carfilzomib for patients with relapsed and refractory multiple myeloma. The study is designed to provide access to patients with relapsed and refractory disease that have received at least 4 prior regimens and are not eligible for any other enrolling carfilzomib Onyx-sponsored studies enrolling patients in the United States. Check the ClinicalTrials.gov site for details.


Drugs for Prostate Cancer

Several expanded access programs for drugs targeting the treatment of advanced prostate cancer are currently open for enrollment. Check the ClinicalTrials.gov site for details.

Experimental Drugs in Phase 3 Clinical Trials

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Remember:

• No cancer drug works for every patient who uses it
• Most cancer drugs have significant side effects
• We don't know if a new treatment will be better than the existing treatment until comparison trials are complete.

There are some new drugs - for example, Gleevec - which offer a dramatic improvement to standard treatments. Unfortunately, most new drugs don't fall into that category.

Experimental drugs in phase 3 clinical trials are generally the drugs closest to FDA review and possible approval. Companies tend to be most open to considering access outside of trials for drugs at this stage of development.

Remember, you cannot force companies to allow access. However, your call may get them thinking about either expanded access or single patient use. Contact us for help.

New Uses for Old Drugs

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Companies may apply to the FDA for approval of their drug in new forms of cancer, based on clinical trial results. This approval means that treatment for the new form of cancer is considered non-experimental and your insurance will cover costs.