On February 22, FDA approved Genentech/Roche's TDM-1, a drug that combines the active ingredient of the company's blockbuster
breast cancer drug Herceptin (trastuzumab) with ImmunoGen's DM1, a chemotherapy.
The approval of TDM-1—branded as Kadcyla—occurred four days prior to its PDUFA date. But for some patients, that wasn't soon
enough. Lorraine Heidke-McCartin, a breast cancer patient (HER2-positive), made her criticism of FDA visible to everyone attending
the annual BIO convention in Boston last year. Along with her husband, Phil McCartin, and several others, Heidke-McCartin
set up a picket line at the entrance to the convention center to publicize the need for broader access to TDM-1. Phil McCartin
told Pharm Exec that the drug—through an expanded access program—saved his wife's life, and that FDA has an ethical obligation to "give stage
four [breast cancer] patients a chance to take this drug."
In 2001, Frank Burroughs founded the Abigail Alliance to "help create wider access to developmental cancer drugs and other
drugs for serious life-threatening illnesses." His daughter, Abigail, died of cancer at age 21 after unsuccessfully seeking
access to then unapproved therapies Erbitux and Iressa. Burroughs sued the FDA, and the Washington Legal Foundation joined
him, arguing that restricting drug access to terminally ill patients constituted a violation of the Fifth Amendment clause
stating that individuals cannot be "deprived of life...without due process of the law."
For a time, it seemed the Abigail Alliance might prevail, but in early 2008, the Supreme Court refused to hear an appellate
court decision ruling in favor of FDA. The case was put to rest, but Burroughs tells Pharm Exec that Rep. Brian Bilbray (R-CA) will reintroduce his Patient Choice Act 2012 into the new Congress this year. PDUFA V, signed
into law by President Obama last summer, adds another expedited drug approval designation—breakthrough therapies—to FDA's
other mechanisms for accelerating products to market.
In late February, the Abigail Alliance met with Janet Woodcock, director of the Center for Drug Evaluation and Research, to
discuss new ways in which the agency can expedite life-saving drugs to patients. The Alliance is scheduled to meet with Margaret
Hamburg, FDA's current commissioner, in March. Steve Walker, co-founder of Abigail Alliance, said FDA's approval of Kadcyla
came "two-plus years later than it should have." Walker raised the issue with Woodcock, and criticized FDA for "having absolutely
no sense of urgency." Woodcock responded, per Walker, that the agency "had taken some steps to address the reasons" for TDM-1's
delay, "but provided no additional information, as usual."
Burroughs says the Abigail Alliance, since its inception in 2001, has advocated for early access to a total of 21 drugs, all
of which were eventually successful in clinical trials. Asked how the Abigail Alliance assesses whether an investigational
drug is worth the inherent risk to patients, Burroughs says, "We just look at the available data." Good science can't be rushed,
but the Internet has facilitated access to unprecedented amounts of information on new drug candidates, causing patients to
question the sometimes sluggish nature of regulatory science. Despite new legislation and new mechanisms like the breakthrough
therapies provision, FDA is likely to face an increasing amount of scrutiny on approval timelines for new drugs, particularly
drugs with the potential to save lives. Nothing is more urgent than the disease progression of a loved one.
Ben Comer is Pharm Exec's Senior Editor. He can be reached at firstname.lastname@example.org