Right to Try? More like redundant begging.

Posted Monday June 16, 2014 by Melissa

Colorado’s “Right to Try” law has made headlines over the past months, prompting questions in my circles about whether it could help our rare disease families.

First, let me note that the title itself is misleading. It creates no rights.

Second, in reality, all it does is layer state procedures over an already existing federal one called expanded access or compassionate use. The law is redundant. Although the federal system is inadequate in many ways, the right to try laws do nothing to fill those gaps or mend the system.

A great commentary on these laws was penned by David Kroll at Forbes, “The False Hope of Colorado’s ‘Right to Try’ Investigational Drug Law” and notes:

States cannot trump federal drug regulatory authority, and we already have the federal equivalent of #RightToTry (as it’s being hashtagged on Twitter). The compassionate use provision, officially known as “expanded access to investigational drugs outside of a clinical trial,” already allows for companies to provide experimental drugs, if available, on a case-by-case basis, and with FDA’s approval. Even with the federal provision, companies are not compelled to provide the drug.

These laws do not address patients’ biggest hurdle – the unwillingness of companies to provide investigational drugs outside of clinical trials to patients who do not qualify. FDA approves approximately 99% of compassionate use requests once they get to them, but they have no way to compile the data of all the denials that have ended with a drug company saying “no.” Expanded access and Right to Try give patients the right to beg and the right to use social media to try to convince or shame a company into granting access. That’s the reality.

What these Right to Try laws do is provide unreal hope to patients with quotes in the media like, “Very sick people in Colorado have new options” and “This is such a paradigm shift for how we approach terminally ill patients’ access to medication in this country.” I seriously wondered if someone at the Goldwater Institute, the think tank who originated these laws, missed a page in their research about expanded access. Fixing the problems that still exist with that program – now that would be something that might actually help patients.

Below is part two of a great series on BioCenturyTV about the Right to Try laws:

BioCentury 06.15.14 - [2] Nothing Gained

One additional item of note…. Steve Usdin comments that some rare disease patients believe that “Randomized clinical trials take too long and patients can’t wait and they should just be able to have access to these drugs and we’ll find out about efficacy further on down the road.” I would agree with that statement. Where you’re watching your child die in front of you, a year alone can be a death sentence. A system of conditional marketing authorisation approval with confirmatory trials like that which exists in Europe (which one might hope that accelerated approval here in the U.S. becomes, but it’s certainly not there yet) would be the least we’d need.

NORD’s Diane Dorman’s response, however, was shocking to me. She says, “I don’t know if many patients would be willing to do that because within the rare disease community, there are many patients who not only want safe products, they also want effective products because there are so few therapies available for these rare diseases.”

If anyone was at last week’s FDA meeting about the neurological manifestations of inborn errors of metabolism, where NORD actually assisted in the cost of many patients attending, they would know that in actuality, there are MANY patients who would be willing to do this. As one parent said, patients and caregivers just want a lifeboat. Very little evidence of efficacy is enough for families who are watching their children die.

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One Response to “Right to Try? More like redundant begging.”

  1. Jamie Lynn-Brooks Fowler // June 17, 2014 at 2:13 pm

    Boy is Diane Dorman far removed from the reality of having a child or having a rare disease! Sad sad sad. And yes we are not requesting a Queen Mary to pick us up, we just want a lifeboat!