Wins and losses
Change.org petitions can be about anything, from asking an insurance plan to cover the costs of a child's chochlear implant,
to the global eradication of cancer, and everything in between – the aspirations and feasibility of the asks vary dramatically.
More than one petition asks for a ban on pharma DTC advertising in the US. Any cause that can be squeezed into the format
of a petition is acceptable, not just health issues, but one successful petition often begets another, sometimes exponentially.
The number of Change.org petitions seeking compassionate use grew significantly last year. "We call it wildfire," says Cayo-Cotter.
"When one person starts a petition for access to a drug, you'll start to see dozens and dozens of petitions...that ends up
creating a space for there to be a real policy change, or an industry shift around an issue." Canada and the EU also have
expanded access programs allowing some patients to receive experimental therapies.
In Canada, Khizar Hayat petitioned Amgen last year on behalf of his nine-year-old son, an acute lymphoblastic leukemia patient.
Hayat asked Amgen for access to blinatumomab, a phase II drug. Amgen declined, but Pfizer stepped in with inotuzumab ozogamicin,
an experimental antibody-drug conjugate. In late October, Hayat wrote a "victory note" on his petition page thanking supporters
and noting that his son had begun treatment. That petition only had 1,581 signatures. In his note, Hayak credited the "well-wishers
and the persistent efforts of the physician who is treating my son to persuade the pharmaceutical companies [to] release the
drugs on compassionate grounds." It's unclear whether the petition played a role in bringing Pfizer on board. But why not
The important thing, for Hayat, is that his son got access to treatment. Change.org may not have been wholly or even partially
responsible for that decision, but the company is pushing the issue of compassionate use back into the public eye, which could
help to reignite the discussion about whether it's ethical to give patients a drug that might do them harm. The first large
compassionate use program instituted by FDA was to grant AIDS patients access to AZT in 1986, on the basis of phase II data.
Dying patients and politicians with a libertarian streak agree that drastic times call for drastic measures, particularly
when everything else has failed. Who better than the patient himself to make that determination? But many physicians remain
uncomfortable prescribing an unapproved, untested drug, even as a last resort. If the patient dies as a result, does that
go against the medical vow of nonmaleficence? If a patient's odds of survival are just as similar with an experimental drug
as without, what is the proper prescription?
The largest compassionate use petition in 2013 was started by Amy Auden, wife of Nick Auden, a stage four melanoma patient.
The petition, asking for access to PD-1 drug candidates from either Merck (lambrolizumab) or Bristol-Meyers Squibb (nivolumab)
garnered over half a million signatures. Both companies declined, and Nick Auden, a father of three, died in November. In
a note to petition signers, Amy Auden expressed "deep gratitude for those who fought with us," adding that "Nick's death beams
a spotlight on a glaring need for change in compassionate access practices for life-saving drugs in late-stage investigational
Change.org chalked up a number of victories in the healthcare space last year, and more than one insurance carrier changed
its coverage policy as a result of the petitions. In Georgia, for example, Coventry Health Care changed its statewide policy
to include the coverage of cochlear implants after a petition on behalf of five-year-old Carson Rubin was signed 111,825 times.
In North Carolina, BlueCross BlueShield (BCBS) agreed to cover 14-year-old Morgan Gleason's life-saving intravenous immunoglobin
treatment. In the victory note, Morgan's mother Amy Gleason said BCBS had pre-approved future treatments, and erased a $240,000
Everyone's a critic
Before Andrea Sloan died, she appeared in videos to discuss her situation, and submitted to numerous interviews. In the comment
sections on industry outlets, including Ed Silverman's currently defunct Pharmalot blog, onlookers wondered why Sloan, an
attractive white woman, felt that she deserved a compassionate use exception while many other patients had been turned away
from clinical trials and denied access to therapy. "Anybody can use Change.org," responds Cayo-Cotter. "It's a smart thing
to do if you're being denied access to drugs or medical care...it can really raise a patient's profile in a way that was impossible
even three years ago."
As to the Hippocratic question, Cayo-Cotter notes that both Sloan and Auden "didn't get access to the drug that they wanted,
and they both ended up dying, so would [access] have been riskier than no access? It seems like a moot question." Before campaigning
on behalf of a patient seeking compassionate use, Change.org first verifies that it's what the actual patient wants (many
petitions are started by friends or family), and that a medical professional has been consulted.
Cayo-Cotter says she hopes to engage further with organized patient advocacy groups to promote policy changes that extend
beyond individuals to large groups of patients. She applauds David France's stirring documentary – How to Survive a Plague
– which chronicles the AIDS epidemic and the role of HIV activist groups Act Up and TAG, and the lives they saved by pushing
regulators and pharmaceutical companies to act. "I just remember watching that piece of the history of compassionate use activism
and thinking, 'Wow, Change.org is becoming a digital platform where that's happening today.' And I was moved by that comparison."