13 posts from March, 2018

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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Defining “good” health care: 2 new studies reveal patient perspectives

Posted March 28th, 2018 by

Do you feel you’re getting the best possible care from your doctor? In two recent studies, PatientsLikeMe members answered this question and shared their perspectives on the health care they’re receiving. The results show that while patient opinions about care and provider performance vary according to condition, diverse patient groups agree on the top factors that define “good” care. Here’s the full scoop…

Poll results: Good care is harder to get for some conditions

Last month, 2,559 PatientsLikeMe members took part in a 6-question poll about doctor-patient relationship and what it means to get “good care.”

The results suggest that patients with certain conditions, especially those living with fibromyalgia, PTSD and MDD, are less satisfied with their care.

The poll also found that patients with these conditions are less likely to:

  • Believe their provider has fully explained treatment options. Just 47% of fibromyalgia and PTSD patients and 53% of MDD patients agree their provider has done so, compared to 63% of patients living with ALS, MS and Parkinson’s disease.

  • Report that they are receiving the best possible health care for their condition. Only 40% of fibromyalgia patients, 49% of PTSD patients and 45% of MDD patients believe they are receiving the best possible care, vs. 66% of ALS patients, 61% of MS patients, and 57% of Parkinson’s disease patients.

  • Change providers even though they think they are not receiving the best care or effective treatment. More than half of these patients (53% of PTSD and 56% of MDD and fibromyalgia patients) have stayed with a provider in this situation vs. just 31% of ALS patients and 36% of MS and Parkinson’s patients.
Why is this the case?

“A positive or negative experience with care could be provider-related, but also related to the fact that patients living with ALS, MS and Parkinson’s often have access to condition-specific specialists or centers of excellence while those living with other conditions do not,” said Sally Okun, PatientsLikeMe’s VP of Policy and Ethics. “This makes it even more important that patients advocate on their own behalf to ensure all avenues to get good care are being used.”

See the full poll results at news.patientslikeme.com.

The patient definition of “good care”

Prior to the poll, more than 200 people (including PatientsLikeMe members, clinicians, researchers and more) shared how they define good health care and what matters most to them, from taking an active role in their care to accessibility and cost. PatientsLikeMe researcher Emil says, “Now more than ever we need to pay attention to that patient role.” In this video, he breaks down the key study takeaways:

 

Are you getting the best possible care? 10 Ways to tell

Based on what the study uncovered, we turned the 10 major factors that define good health care into a check list. Speaking about the poll and the survey, Sally Okun says, “These complementary studies give a snapshot of what is most important to patients, and give patients the tools to find providers willing to meet the characteristics of good care.”

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