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Advocacy organizations and individual patients are getting more involved in every facet of the healthcare system, from drug R&D, to federal and state policy all fueled by the hour-to-hour passion of living with a disease.
Anyone who's done battle with a bureaucracy knows that the most difficult ear to speak into is the one that's connected to a mouth with the power to say yes or no. Voices in favor of change, no matter how loud and compelling, can easily be drowned in a symphony of established structures, not to mention competing interests and conflicting policies. Advocacy of any sort can be a daunting task, but when the advocate is a patient, and the issue is his life, then "Silence = Death," as ACT UP said during the AIDS epidemic.
But the growing patient advocacy movement, in the US and globally, isn't just about getting access to new, potentially life-saving drugs. And the methods for improving patients' quality of life, their interaction with clinicians – and ultimately their health outcomes – aren't limited to picket line protests, underground clinical trials and public figures burnt in effigy. Access remains vitally important, but it's one among many issues kindled by patients who want to live well, in spite of illness.
Brianna Cayo-Cotter, Change.org’s managing director of communications (Photo: Michael Secton)
Patients with certain kinds of dementia, for example, can suffer just as badly from a wrong diagnosis as from the lack of access to therapy. Cancer patients need answers to questions specific to their own individual lives that often go unanswered. Policies that make it difficult for otherwise appropriate patients to participate in clinical trials need to be legislated. Patients spread across geographies must bridge time and space to meet in solidarity, and to push their causes past the doorstep and onto the dinner table of the decision-makers, wherever those deciders dine.
Last year, San Francisco-based Change.org , an online petition platform, made a name for itself among pharma industry onlookers after a petition for Andrea Sloan, an ovarian cancer patient, sought compassionate use access to BioMarin's PARP inhibitor (BMN-673) currently in development. BioMarin declined to include Sloan in an active clinical trial, which may have been the end of the story. But then things got strange.
Every time someone signs a petition on Change.org, an email is sent to the petition's designated target: in this case, six executives at BioMarin, including CEO Jean-Jacques Bienaime. Other supporters of Andrea Sloan emailed Bienaime directly. In an accidental "reply all" to one of these emails, Bienaime "was a little too candid in a response that he'd meant for other people on his [internal] team," explains Change.org deputy campaign director Tim Newman, who worked directly on the Andrea Sloan petition. "So we helped to link [Andrea Sloan's advocacy group] to a local news channel."
It's not a good idea to use insulting language toward a patient dying of cancer, particularly when you're in control of access to a drug that patient believes could save her life. A media firestorm ensued. Andrea Sloan lived in Austin, Texas, and politicians as varied as Newt Gingrich, Howard Dean, Karl Rove, Ted Cruz, John Cornyn and more than 70 Texas state legislators all endorsed the Andrea Sloan campaign. That's in addition to celebrities including Kathy Bates, Jon Lovitz, Mia Farrow, Rosie O'Donnell and Rosie Perez.
Change.org campaigners select individual, user-generated petitions based on their potential to succeed and to make a demonstrable impact. Once selected, campaigners help petitioners identify the decision-makers capable of responding. They also coach on the wording of petitions, give pointers – a personal photo helps – and suggest potential media opportunities and social media outreach. "Sometimes we need to tell petitioners how to set up a Twitter account, and how to use social media to increase the chances of success," says Brianna Cayo-Cotter, who helps manage Change.org's campaign team, and serves as the company's managing director of communications. Andrea Sloan, however, didn't need much handholding, according to Cayo-Cotter. Sloan had her own media savvy, full-court press advocacy group called Andi's Army; Change.org just helped extend the reach.
The BioMarin controversy came to head when Bienaime was questioned directly about Sloan during BioCentury's NewsMakers in the Biotech Industry conference in New York City last September. Change.org simultaneously issued a press release with links to the offending emails sent by Bienaime to Sloan's supporters, as well as supportive comments made by BioMarin shareholders.
Even though FDA stood ready to approve Sloan's access to BMN-673 through compassionate use, BioMarin still refused, maintaining that it was too early to know whether the therapy would be safe or effective. Critics suggested that BioMarin feared Sloan's public use of the drug would jeopardize the regulatory approval timeline. It seemed Sloan and BioMarin had reached a stalemate.
But then, as a result of Andi's Army and the Change.org campaign, which garnered over 200,000 thousand signatures and a growing number of celebrity endorsements and media reports, another, anonymous pharmaceutical company stepped in and gave Sloan access to its experimental PARP inhibitor. Sadly, it was too late. Sloan died on New Year's Day.
The vibe at Change.org's headquarters in San Francisco's Pontrero Hill district is an even commixture of dressed-down mellowness and political urgency. Sunlight blooms through unobstructed windows onto hardwood floors and illuminates the office's white, industrial-style interior. Employees freely congregate around unwalled work centers and flat screens in the main office space. Philz local blend coffee is available in the kitchen, and Kentucky, Cayo-Cotter's dog (rescued roadside on a trip through the Bluegrass State) tirelessly pursues a tennis ball. Cayo-Cotter joined Change.org in 2011, after founder Ben Rattray reorganized the company's focus.
Rattray, a Stanford graduate, launched Change.org in 2007 with a desire to "use technology on a massive scale to create positive change, not just as a way to share photos or sell coupons," says Cayo-Cotter. In the beginning, Change.org was fundamentally a blogging platform, with a fundraising capability and space for debating ideas. Bloggers on the site could start petitions around things they blogged about, and eventually Rattray decided that petitions should be open to anyone on the site.
One of the first non-blogger petitions was started by a South African woman, a lesbian, whose close friends had been victims of "corrective rape," a practice whereby gay women are raped by men in an attempt to correct them of their lesbianism. The South African government wasn't addressing the problem, and in one week the petition had racked up nearly 150,000 signatures from people all over the world. Rattray started getting calls from the Justice Ministry in South Africa. Ministry officials agreed to set up a task force to examine the problem, and to consider hate crime legislation. Corrective rape is still prevalent in South Africa, but Rattray's experience with the campaign convinced him that online petitions could serve the public good.
Others remain skeptical about the actual power of an online petition. Last fall, Change.org introduced a new tool aimed at further demonstrating the impact of its petitions, called Verified Decision Makers. Once a petition is created, Change.org can reach out to the decision maker and let them know about the campaign. The decision-maker is given a dedicated page and instructions on how to respond to the petition. If the decider responds directly to a petition, those comments show up on the petition page, to be shared and critiqued by other online commentators.
"It's really interesting to see which decision makers respond, and how they respond," says Cayo-Cotter. "So far the most responsive entities have been local government officials, followed by companies, and third is the federal government in the US, which is kind of a telling state of affairs." There has been some federal participation. Rep. Paul Ryan (R-WI) and Sen. Elizabeth Warren (D-MA) are both Verified Decision Makers.
Cayo-Cotter says pharmaceutical company decision makers "would be fantastic to have on the site...there are so many petitions around health issues." Over 10,000 health-related Change.org petitions were signed by over 18 million people, at press time. Change.org hasn't begun pitching the Verified Decision Makers tool to pharmaceutical executives yet, but "if anyone reading your article wants to sign up, we'll get them verified," says Cayo-Cotter.
Pharma isn't known for its consumer-facing digital prowess, but companies "trying to brand themselves and show their investors and their customers that they're different, and willing to engage with patients in an interesting way...I think there's a real advantage in that," says Cayo-Cotter. Pharma is "already creating responses to policies and proposals around things that are getting raised in petitions. They're just being left out of the conversation. This is an opportunity to directly communicate with the people that care the most about a particular issue."
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 try everything?
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 trials."
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 medical bill.
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."
Online petitions can travel far and wide in an instant, but sometimes its takes an old fashioned trip to Washington DC, and the courage to speak up, to make a change. Not surprisingly, it was the internet that first brought Camila Strassle, currently a freshman at Stanford University, to the Cystic Fibrosis Foundation (CFF).
"I have a brother with cystic fibrosis, so my mom and I were looking online for ways that we could get involved," says Strassle. "The CFF had a link for advocacy and through that I found out about a teen program for siblings with cystic fibrosis to actually go to Washington DC and advocate on their behalf." The reason cystic fibrosis patients can't get together to lobby on their own behalf, notes Strassle, is because they can't be in the same room together, due to the risk of cross-infection.
For the past four years, Strassle has gone to Washington to participate in the CFF's Teen Advocacy Day; she has met with more than two-dozen members of Congress. In 2010, she helped turn a bill – the Improving Access to Clinical Trials Act, or IACT – into law. "It was a bipartisan bill that was passed and signed into law by President Obama," says Strassle. IACT address a problem with clinical trial participation. Before its passage, people who received Supplemental Security Income weren't allowed to accept research compensation, because it rendered them ineligible for public medical benefits. "With rare diseases, it's a small population, so it's hard enough already to find people to participate in trials," says Strassle. "It was slowing everything down."
With a host of new therapies for cystic fibrosis entering or nearing the market, Strassle says one of the main initiatives currently at the CFF is promoting access to specialized 'care centers,' a network of CFF accredited centers built on a model that recognizes the unique needs of cystic fibrosis patients. "For example, specialists working in the care centers look at the pancreas, at nutrition, at all of these separate issues that effect patients with cystic fibrosis," says Strassle. "As a result, life expectancy for patients has increased over time, and the NIH has come out in support of this model."
According to the CFF website, over 110 care centers are accredited nationwide, with more than 260 clinics where children and adults with cystic fibrosis can get care. The centers also participate in clinical research, and data from the centers is collected in the CFF's patient registry. Aside from the care centers, the CFF runs a nonprofit drug development affiliate called Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT). The CFFT partners with pharmaceutical companies and receives royalties from drug sales developed using CFFT funding, and most cystic fibrosis drugs have leveraged the basic research and clinical trial network operated by the CFF, according to the website.
Strassle continues to advocate with the CFF on her brother's behalf, and worked as an intern with the CFF's public policy department last summer. As a freshman, Strassle is completing her general requirements, but is planning to double major in English and health policy. She'll be looking out for the health policy track next year. "I'm really looking forward to taking those classes," she says.
In some ways, patients diagnosed with a well-understood and established disease, even a serious one, can consider themselves lucky. At least they know what they're up against, and can bring the right weapons to the fight. Imagine if something began to go wrong, but no one could tell you with any certainty what was happening. Years go by, along with the possibility of treating the disease before it gets worse.
Susan Grant was a top financial advisor at American Express, overseeing millions of client dollars, when something happened to her brain. "I knew something was different, but I didn't know what it was," says Grant. "Of course, American Express didn't want me to tell my clients anything [about my condition], but when I sat down at a table with 100 clients to transition them to a new advisor, and I tried to tell them the doctors don't know what I have, they wouldn't accept it. They wouldn't let me dodge the question, so I just started saying, 'Something's different in my brain.'"
Several years and six wrong diagnoses later, Grant, at 53, was diagnosed with frontotemporal dementia (FTD), a CNS disease that can strike in adults as young as 40 years old. Up to 50 percent of the dementia cases occurring before age 65 are thought to be a result of FTD, according to the Alzheimer's Association. Grant has fought her way into clinical trials, and fought back against physicians who've attempted to downplay her symptoms, or take a "wait and see" approach to medical intervention. Many physicians believe that patients with FTD, and even Alzheimer's, aren't capable of "insight," meaning they can't recognize changes to their mental state. That is true for some patients, says Grant, but in her case and in many others, patients do recognize that something has changed, even if they don't yet have a name for it.
Because patients with FTD can face a rapid deterioration of executive function, it's critical to get the right diagnosis as early as possible. That means using the right terminology in the doctor's office, and getting a referral for a neurologist that specializes in neurodegenerative diseases. An early diagnosis helps patients better manage the disease, and seek treatment, if not yet for the root cause of the disease, than at least for the debilitating side effects. Also, a definitive diagnosis early on gives patients time to get their finances in order, and to take advantage of disability benefits, as Grant did.
In 2007, Grant founded Planning for Hope, a grass-roots organization geared toward empowering patients with FTD. She has authored an instructive eBook for FTD sufferers, and raised $150,000 to create an hour-long documentary to promote awareness of the disease, and to change the conversation about patients and the stigmas that come with a diagnosis. The film, also called Planning for Hope, has been shown on a number of PBS affiliates, and can be watched online at FTDplanningforhope.com.
Although there is no cure for FTD, Grant speaks highly of cognitive improvement software and the use of puzzles and word games. She still bristles at the language NIH and other organizations use to describe FTD patients. They're described in the medical literature as having "no morals" or "misbehaving in public," says Grant. "Why would anyone come forward if this is the language that's being used?"
Even though a concrete diagnosis provides an answer to the question of what is wrong, it oftentimes leads to a host of new questions. The mission of Cancer101, a nonprofit organization based in New York City, is to empower, inform and engage cancer patients and their caregivers by helping them partner with healthcare providers to make informed, collaborative decisions.
Sarah Krug, executive director at Cancer101, says the organization's impact lies in its ability to educate patients based on individual needs. As cancer has splintered into a number of subtypes, many with specific treatments tailored to optimize outcomes for patients with specific genetic mutations, information specificity has lagged in terms of personalization. "If a patient is female and 35 years old, she might need information on fertility preservation," says Krug. Questions often arise when a physician isn't around to provide an answer, so Cancer101 has developed a toolkit that's personalized at the point of care, for individual patients.
Instead of sending toolkits directly to patients, Cancer101 partners with clinicians, which helps facilitate a partnership with patients. Krug calls the process a "prescription to learn;" Cancer101 incentivizes adoption by offering CME credits for clinicians learning how to use the toolkits, which are intended to be used over a 10-year timeframe. "There's a lot that goes on between visits, so it's important for patients to document their day-to-day life – an expertise in it's own right – so that when they come in for an appointment, they get medical expertise based on their own patient expertise," says Krug.
Cancer101 is currently working on an electronic version of the toolkits, but interestingly, many patients in the recent past have expressed a preference for the tangibility of a binder, and the ability to add new inserts as they become available, and to keep track of the endless paperwork generated in hospitals. The information contained in the toolkits, which have been deemed the "Cadillac of patient education," according to Krug, is aggregated from a variety of sources. "We work with different organizations and get permission to reprint their content, so if the National Cancer Institute has great information about lung cancer, for example, we'll use it – we don't have to create education from scratch," says Krug. "We only build new education in areas of unmet need." Cancer101 regularly conducts surveys with clinicians to keep toolkits up-to-date, and prioritizes health literacy, to ensure that toolkit materials are best suited for patients. The organization reaches 100,000 cancer patients each year.
Krug, who also serves as past president of the Society for Participatory Medicine, and as a board member at the National Organization of Rare Diseases (NORD), is currently working on a code of ethics to guide relationships between patient advocacy organizations and pharma companies.
The patient advocacy movement, taken as a whole, is as multifaceted as patients themselves. As new treatments, new models for healthcare delivery, new regulatory pathways, and new patients enter the system, advocacy efforts will continuously adapt and expand to support new patient needs, and to fill the gaps in the established order. No commodity is more valuable than human health, and patient advocates will only get better at protecting and defending it.
Ben Comer is senior editor of Pharm Exec. He can be reached at firstname.lastname@example.org