The Expanded Access Navigator: Helping Patients Who Need Potentially Life-Saving Drug Treatments

Excerpt:

“You just diagnosed a patient with a serious condition for which there is no FDA-approved drug therapy.

“Now what?

“After the hard conversation that no physician wants to have with a patient and his or her caregivers and loved ones, you may be left feeling helpless and hopeless.

“You know there may be investigational therapies in development but not FDA approved that could help your patient, but you don’t know where to begin.”

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If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our ASK Cancer Commons service.


New Online Navigator Helps Patients and Doctors Access Experimental Treatments

Excerpt:

“When approved therapies don’t work, or stop working, for people with serious or life-threatening illnesses, it puts them in a difficult position. Some turn to clinical trials that are testing experimental treatments. But many can’t do that because they are too sick, don’t meet the requirements of the trial, or can’t afford to travel to the site of a trial. That doesn’t mean they are out of options.”

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If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our ASK Cancer Commons service.


Statement from FDA Commissioner Robert Califf, M.D. On the Release of the Final Individual Patient Expanded Access Form

Excerpt:

“Today, the U.S. Food and Drug Administration finalized its efforts to streamline the process used by physicians to request expanded access, often called “compassionate use,” to investigational drugs and biologics for their patients. As a physician, I understand the importance of being able to access investigational treatments for a patient when there are no other options to treat their serious disease or condition.

“Access to investigational treatments requires the active cooperation of the FDA, industry, and health care professionals in order to be successful. But we know that navigating that process can be challenging and time consuming, and we are committed to reducing the procedural burdens on physicians and patients whenever possible.”

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Do you have questions about this story? Let us know in a comment below. If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our Ask Cancer Commons service.


FDA Expands Palbociclib Approval for Breast Cancer

“The Food and Drug Administration (FDA) has granted an expanded indication for the cyclin-dependent kinase 4/6 inhibitor palbociclib (Ibrance). The drug is now approved for use in combination with fulvestrant in women with hormone receptor (HR)-positive, HER2-negative advanced or metastatic breast cancer whose disease progressed following endocrine therapy.

“Palbociclib was initially approved in February 2015 for the treatment of estrogen receptor–positive, HER2-negative metastatic breast cancer, in women who had not yet received endocrine therapy. The new approval was granted under the FDA’s breakthrough therapy designation.

“The additional indication for palbociclib is based on results from the PALOMA-3 trial, which was stopped early in April 2015 after an interim analysis showed benefit in combination with fulvestrant when compared to fulvestrant and placebo.”


New Laws Give Dying Patients ‘Right to Try’ Unproven Drugs

“Colorado, Missouri and Louisiana are poised to become the first states in the nation to give terminally ill patients the right to try experimental drugs without the blessing of the Food and Drug Administration, setting the stage for what could be a lengthy battle over who should decide whether a drug is too risky to try.

“Lawmakers in the three states have passed “Right to Try” laws with unanimous votes in recent weeks, after high-profile, social media campaigns in which families of dying patients have pushed for access to unapproved but potentially lifesaving drugs. Colorado’s governor is expected to sign that state’s law Saturday.”

Editor’s note: New cancer drugs can take a long time to reach the clinic, even after they have already proven safe and beneficial. Some patients are successful in gaining early access to drugs (see our Chief Scientist’s blog post on so-called “compassionate access”), but it is a difficult process. Some drug companies are trying to remedy the issue by starting “expanded access trials” that give drugs to patients unable to enroll in the clinical trials testing them. For example, Novartis made its promising drug LDK378 available under expanded access in trial number NCT01947608 in September, 2013.


New Laws Give Dying Patients ‘Right to Try’ Unproven Drugs

“Colorado, Missouri and Louisiana are poised to become the first states in the nation to give terminally ill patients the right to try experimental drugs without the blessing of the Food and Drug Administration, setting the stage for what could be a lengthy battle over who should decide whether a drug is too risky to try.

“Lawmakers in the three states have passed “Right to Try” laws with unanimous votes in recent weeks, after high-profile, social media campaigns in which families of dying patients have pushed for access to unapproved but potentially lifesaving drugs. Colorado’s governor is expected to sign that state’s law Saturday.”

Editor’s note: New cancer drugs can take a long time to reach the clinic, even after they have already proven safe and beneficial. Some patients are successful in gaining early access to drugs (see our Chief Scientist’s blog post on so-called “compassionate access”), but it is a difficult process. Some drug companies are trying to remedy the issue by starting “expanded access trials” that give drugs to patients unable to enroll in the clinical trials testing them. For example, Novartis made its promising drug LDK378 available under expanded access in trial number NCT01947608 in September, 2013.


New Laws Give Dying Patients ‘Right to Try’ Unproven Drugs

“Colorado, Missouri and Louisiana are poised to become the first states in the nation to give terminally ill patients the right to try experimental drugs without the blessing of the Food and Drug Administration, setting the stage for what could be a lengthy battle over who should decide whether a drug is too risky to try.

“Lawmakers in the three states have passed “Right to Try” laws with unanimous votes in recent weeks, after high-profile, social media campaigns in which families of dying patients have pushed for access to unapproved but potentially lifesaving drugs. Colorado’s governor is expected to sign that state’s law Saturday.”

Editor’s note: New cancer drugs can take a long time to reach the clinic, even after they have already proven safe and beneficial. Some patients are successful in gaining early access to drugs (see our Chief Scientist’s blog post on so-called “compassionate access”), but it is a difficult process. Some drug companies are trying to remedy the issue by starting “expanded access trials” that give drugs to patients unable to enroll in the clinical trials testing them. For example, Novartis made its promising drug LDK378 available under expanded access in trial number NCT01947608 in September, 2013.


Compassionate Drug Access: A Real Option for Cancer Patients?


A recent New York Times article tells the story of one woman’s quest to gain access to an experimental drug to treat her deadly cancer. Her story is familiar to many of us who have heard similar tales; a cancer patient runs out of treatment options, but with the help of proactive oncologists is able to receive a new, investigational drug; that is, a drug not yet approved by the U.S. Food and Drug Administration (FDA). This last-resort treatment approach is known as compassionate use or, as the FDA prefers to call it, expanded access. The U.S. National Library of Medicine explains: Continue reading…