3 posts tagged “clinical trial awareness”

What’s the right-to-try bill + possible pros and cons?

Posted March 30th, 2018 by

The U.S. House of Representatives recently passed legislation that could give terminally ill patients the “right to try” experimental treatments — so what’s the controversy? Catch up on the topic and share your thoughts below.

“Right-to-try,” in a nutshell

The “right-to-try” bill aims to give terminally ill patients who’ve exhausted all other treatment options quicker and easier access to an “experimental treatment” (this is a medication or treatment product that has passed “phase one” of the clinical trial process but has not yet received full Food and Drug Administration [FDA] approval). See this PatientsLikeMe guide to clinical trials to learn more about trial phases and the FDA drug approval process.

The House failed to pass the bill on the first try, on March 13, but voted 267 to 149 to pass it on March 21.

All in favor…

Backers of the bill have cited these “pros” in their reasoning:

  • 38 states have already passed their own versions of “right-to-try” legislation, so a federal version of the law would grant access to experimental treatments nationwide.
  • Although the FDA has existing “expanded access” (or “compassionate use”) policies that allow the use of investigational treatments in the most dire cases, patients must apply to the FDA for permission to receive the treatment — so “right-to-try” would eliminate the step of needing to apply.
  • Most terminally ill patients don’t qualify to participate in clinical trials, so “right-to-try” could give them quick access to treatments that are still in the middle of years-long trials.

All opposed…

People who are against the bill have some arguments of their own:

  • Four former FDA commissioners issued a joint statement opposing the federal “right-to-try” legislation, saying it would “create a dangerous precedent that would erode protections for vulnerable patients.” (When the FDA is involved in “expanded care” cases, it tries to provide any additional information on dosages, side effects and safety considerations — so removing the FDA from the process may eliminate expert guidance and safeguards.)
  • Many drugs that make it through phase I of clinical trials (smaller initial studies to test for safety) fail to survive phase II of trials because they’re not proven to be effective — so “right-to-try” may open the door to developers peddling “snake oil” or hyped-up/unproven treatments to the most desperate patients.
  • “Right-to-try” may not significantly expand access to treatments — the FDA says it grants approval in 99 percent of requests through the “expanded access” policies and often authorizes emergency requests immediately over the phone. And few pharmaceutical companies offer access to experimental treatments without FDA approval because of ethical and legal concerns.

“No ethical company that I know of would make their experimental medicine available outside of the FDA’s regulatory oversight process,” Kenneth Moch, CEO of drug developer Cognition Therapeutics, tells the Associated Press. “The FDA are not the bad guys here. You may not like their processes but they are involved here for a variety of reasons.”

Modern Healthcare points out that using an experimental treatment can derail a patient’s health insurance coverage for additional medical treatment or hospice care thereafter.

“If the bill is passed and signed, the campaign that made it law may not stop there,” Modern Healthcare also reports. “Some believe there is a chance the program’s scope could be expanded beyond terminally-ill patients to one day include those with severe chronic conditions.”

Next up, the “right-to-try” bill heads to the Senate. If they pass it, the president (who has expressed support) is expected to sign it into law.

What are your thoughts on the “right-to-try” bill? Join PatientsLikeMe to talk about this topic with a community of other real-world patients.

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Partnering with patients: An interview with Michael Evers and Ken Getz

Posted June 20th, 2013 by

PatientsLikeMe and the nonprofit Center for Information and Study on Clinical Research Participation (CISCRP) recently announced a new partnership with the global healthcare leader Sanofi (NYSE: SNY) to raise awareness about the benefits of participating in clinical trials.

Michael Evers, Executive Vice President of Marketing and Patient Advocacy at PatientsLikeMe, and Ken Getz, Founder and Board Chair of CISCRP and an associate professor at the Tufts University School of Medicine, share their views on why spotlighting “Medical Heroes” (those who “give the gift of their participation in clinical trials” to advance medical knowledge) and increasing awareness about clinical trial opportunities will help get the best medicines to patients faster.

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Michael Evers                           Ken Getz


What’s this partnership all about?

ME- Given our missions and beliefs in the power of clinical research, it simply made sense for our three companies to collaborate. It’s an exciting alliance – and the first for PatientsLikeMe where we’re partnering with a pharma leader like Sanofi and a nonprofit like CISCRP to do a widespread awareness campaign on the power of research participation. For example, with our 200,000-member community and Clinical Trial Awareness tool, we’ll be able to match patients with relevant Sanofi trials and work with CISCRP to provide educational materials about the clinical trial process. Similarly, our partners are interested in directing their clinical trial participants to PatientsLikeMe where they can use tools to better manage their disease while participating in real-time research every day. Like I said, there was a lot of synergy here.

KG- We all share the same goal – to empower patients with information that helps them make informed decisions about whether to participate in clinical research and ultimately to ensure that the right treatments make it to the patients who need them in an efficient manner. This partnership is really about an integrative approach to empowering patients through closer collaboration with research sponsors, patient networks and a nonprofit educator.


Can you tell us a little about these “Medical Heroes?”

KG- CISCRP launched this groundbreaking public service campaign back in 2008 to portray the image of the clinical research participant as a Medical Hero. The Medical Heroes campaign provides recognition for the profound “gift of participation,” while raising awareness of the participant’s significant contributions to advancing public health. Clinical study volunteers are medical heroes found in ordinary, everyday places. Even though they advance public health, these medical heroes are under-acknowledged and under-appreciated.

ME- The Medical Heroes campaign that CISCRP has developed is so powerful. It’s an inspirational way to show how people’s participation in clinical trials is such a selfless gift and crucial part of advancing medicine for everyone. What struck a chord with us is how well this campaign described many of our members – those who participate in real-time research every day by logging onto PatientsLikeMe and sharing data about their experiences.


What else do you want people to know about this partnership or what’s ahead?

KG- It’s widely known that clinical trials are less efficient and are taking longer to complete. Patient recruitment and retention is extremely challenging and a major contributing factor to study delays. The main goal of this collaboration is to leverage the power of interactive communities with outreach and educational materials to truly engage patients as partners in advancing medical knowledge. Ultimately, we hope that this collaboration will demonstrate a new approach to improving recruitment and retention rates and to help reduce study delays. The patient is waiting for new and better treatments – and expects this from all of us.

ME- Ken said it well – this is all about engaging patients as partners. That’s the only way we can get to better research, better treatments and better medicine. The more Medical Heroes there are contributing to research, the faster we can get there. That’s a win for everyone.