2 posts tagged “benefits and risks of clinical trials”

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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How Social Media Is Changing Research (Part II): A Guest Post by MS Clinical Trial Participant and Blogger Jeri Burtchell

Posted September 13th, 2012 by

Today’s guest post is written by PatientsLikeMe member Jeri Burtchell (TickledPink), who has been living with multiple sclerosis (MS) for 13 years.  A tie dye apparel store owner and mother of two, she writes a blog entitled “Gilenya and Me:  My Story of Being an MS Patient, a Hypochondriac and a Guinea Pig.”  Her patient advocacy and social media presence led to her being invited to speak the Disruptive Innovations conference taking place in Boston this week.

Read Part I of Jeri’s guest post first!

MS Patient, Blogger and Activist Jeri Burtchell (TickledPink at PatientsLikeMe)

Because blogging a clinical trial from start to finish was unheard of, I attracted the interest of not only patients, but those in charge of clinical trials. They are interested in the impact of social media on clinical trials, and how they can utilize it to their benefit. Sites such as personal blogs, FacebookTwitter, and PatientsLikeMe are here to stay and people naturally want to share information.

I got a direct message on Twitter from Craig Lipset, who is Head of Clinical Innovation, Worldwide Research & Development for Pfizer. Social media brought us together to have a conversation about research that never could have taken place before the Internet. Everyone is more connected and approachable now. Naturally, I blogged about it.

But that was just beginning.  Tomorrow, September 14th, at 9:45 a.m. , I will be speaking along with Craig at the Disruptive Innovations conference, where the leaders in pharmaceutical research will be gathering to share ideas and come up with innovative ways of conducting clinical trials that take the “ePatient” into consideration. The 30-minute segment is entitled “Patient Leaders as Key Stakeholders in Clinical Trials,” and I will be there to represent – and put a human face to – clinical trial patients everywhere.

Knowing this is a chance of a lifetime for a trial patient to have the researchers as their audience, I wanted to reach out to those who have participated in past or current trials. My question to them is: “If you could ask or tell researchers just one thing about your own experience as a trial patient, what would that be?”

I plan to attend this conference and speak on behalf of all patients and put a face to the humans behind the data. I want to show them that we are connected now more than ever by social media. Researchers need to harness that power to their benefit. Soon they may use it to recruit and retain trial participants. I would like to see them provide a monitored gathering place for these trial patients to reduce the spread of misinformation as patients share data.

How Many of the 35,000+ Clinical Trials Currently Recruiting Do You Qualify for?  Use PatientsLikeMe's Clinical Trials Search Tool to Find Out!

To people who are considering a trial I recommend using tools like PatientsLikeMe and ClinicalTrials.gov to stay informed about ongoing research and find a doctor willing to support your interest in participating. Remember that not every trial will culminate in a drug that wins FDA approval. By joining a clinical trial you will be taking risks, but you may also be reaping benefits long before the general public will have access to the drug. Never forget that you are a pioneer and by entering a trial you are giving the greatest gift possible. Without volunteers we would have no medical advancements.

I hope that researchers never forget the impact they are having on the lives of people everywhere. They aren’t just going to work every day; they are the makers of miracles. Often patients are joining these trials as a last resort. The work of researchers gives us all promise for a brighter future.

I hope that patients everywhere will take one clear message away from this: NEVER GIVE UP! It would have been so easy that day to end it all. I was depressed and certain my life could get nothing but worse. But, by choosing to fight, I have changed my life forever and doors continue to open for me. By reaching out through social media I know I am not alone. You never know what tomorrow may bring, so don’t give up on today!

Editor’s Note:  Jeri isn’t the only PatientsLikeMe member blogging about her experience in a clinical trial.  See our interview with PGen study participant PF Anderson for another patient’s chronicle!