Colorado Gov. John Hickenlooper delivers his State of the State address at the state Capitol in Denver, Jan. 10, 2013. During his address, Hickenlooper touched on the controversial subject of gun control in light of last year's theater shooting.

Matthew Staver, Nyt - Nyt

FILE - Colorado Gov. John Hickenlooper

Associated Press,

Terminal patients get access to experimental drugs

  • Article by: Brady Dennis and Ariana Eunhung Cha
  • Washington Post
  • May 17, 2014 - 4:34 PM

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 signed that state’s law Saturday.

Proponents of the measures argue that patients desperate for treatments must navigate a lengthy, cumbersome process to get the FDA to approve early access to experimental drugs and to persuade companies to provide them. The laws are intended to cut through some of that red tape by essentially cutting the federal government out of the picture.

“For people who are facing death and have one last hope, they should have a choice to try every possible drug,” said state Rep. Joann Ginal, a Democrat and cosponsor of the bill in Colorado. Ginal introduced it in part because she witnessed how an experimental treatment helped her older brother, who has a rare blood cancer.

Circumventing the law

Opponents of the approach call it an ill-advised effort that circumvents federal law, undermines the drug development process and threatens to harm more people than it helps.

“The notion is based on the ‘Dallas Buyers Club’ — the idea that you have to get around the indifferent and cruel government to get access to drugs,” said Arthur Caplan, director of the bioethics division at New York University Langone Medical Center, referring to the Oscar-winning movie based on an AIDS patient who smuggled unapproved drugs into Texas during the 1980s.

The reality, Caplan said, is more complicated than singling out the FDA, which approves almost all the requests it receives for “compassionate use” exemptions. He noted that the new legislation does nothing to compel cooperation from drugmakers.

Frank Burroughs, founder of the Virginia-based Abigail Alliance for Better Access to Experimental Drugs, which has long pushed the FDA to widen access, said people aren’t after just any new medication.

“We’re talking about ‘promising’ drugs,” said Burroughs, whose group has helped the Goldwater Institute, a conservative organization, push for the state laws. “Patients are much smarter and savvy than they get credit for.”

Burroughs said the FDA simply hasn’t moved quickly enough and that people who are out of options are willing to take on more risk than an ordinary person. “The risk-benefit is much different than someone who’s waiting for a new allergy medication or a new toe fungus cream,” he said.

The FDA on Friday declined to take a position on any of the state Right to Try bills. But in a statement, the agency said it is concerned about any efforts that might undermine the “congressionally-mandated authority and agency mission to protect the public from therapies that are not safe and effective.”

Some exemptions exist

FDA regulations allow for access to investigational drugs outside of a clinical trial for patients who have serious or life-threatening illnesses and have no comparable alternatives. While these “compassionate use” exemptions can apply to individuals on a case-by-case basis, the FDA also can grant expanded access for larger groups of patients.

The Right to Try bills aim to provide a streamlined alternative to the FDA process. Instead of having to fill out lengthy and complex paperwork, patients would only need to get an OK from a drug company and a simple prescription or “recommendation” from a doctor. The drugs involved also must have successfully completed an initial safety trial and have moved to the next development phase.

It’s unclear how many drugmakers might be willing to make use of the state laws at the risk of angering federal regulators. But at least one company plans to take advantage of the new legislation in Colorado.

Neuralstem, based in Germantown, Md., has begun looking for doctors in the state to use its treatment — which involves surgically transplanting neural stem cells in the spinal cord — for Lou Gehrig’s disease. The company’s chief executive, I. Richard Garr, said its first trial, involving 15 patients, was promising in slowing down the disease.

“On average, these patients die within two to four years of diagnosis, so our hope is to make this available to everyone as quickly as possible,” he said.

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