2 posts tagged “research pipeline”

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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MS research: What’s the latest?

Posted February 23rd, 2018 by

Keeping tabs on the latest MS research isn’t always easy. So, our team of in-house health professionals took a closer look into some of the treatments in the research pipeline for people living with MS. Most of these treatments are in the final phase of clinical development — phase III clinical trials. In this phase, researchers compare the safety and effectiveness of the new treatment against the current standard treatment.

Check out the roundup:

  1. Ozanimod – An oral treatment in phase III clinical trials with the potential to reduce relapses and prevent neurological damage. Ozanimod is reported to work like Gilenya (fingolimod) but with some potential for fewer side effects. A new drug application for ozanimod was submitted to the FDA in December 2017. This application is seeking approval for the use of this agent to treat relapsing multiple sclerosis. It is possible that an FDA decision could be made on this application in the second half of 2018.
  2. Ponesimod – An oral treatment in phase III clinical trials that prevents immune cells from damaging myelin that insulates nerve-cells in patients with MS. A new drug application for Ponesimod is possible within the next couple of years.
  3. Siponimod – Similar to Ozanimod, Siponimod is an oral treatment in phase III clinical trials that may reduce risk of relapse and disease progression. With a new drug application in the next year or two, the treatment has the potential for approval and launch 6-12 months later.
  4. ALKS 8700 – This oral treatment (currently in phase III clinical trials) is a slightly different formulation of Tecfidera (dimethyl fumarate) but, according to Alkermes, has fewer gastrointestinal side effects. With a new drug application in 2018, the treatment could be available 6-12 months later.

In other treatment news:

Laquinimod – You might recognize the name because initially, it showed some promise. More recently it’s performed poorly in clinical trials. Laquinimod is still being developed but in Phase II studies (vs. Phase III which is the final phase of clinical development), which could mean it’s at least a couple years away.

Looking for more info on research and treatments? Join the community today to learn more and connect with more than 60,000 members living with MS.

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