3 posts tagged “safety”

Sally Okun explains the new research collaboration with the FDA

Posted June 16th, 2015 by

Yesterday, we announced a new research collaboration with the U.S. Food and Drug Administration (FDA) that will explore how patient-reported data can lead to new insights about drug safety. It’s the first time the FDA will analyze patient-generated data for pharmacovigilence (aka drug safety).

But we’re no strangers to drug safety. Check out some of the previous work the community has helped to drive:

To learn more about this new (and unprecedented) collaboration, we talked to our very own Sally Okun, Vice President of Advocacy, Policy and Patient Safety.

What will this collaboration do?
Patients’ lives and well-being often depend upon medical products approved and regulated by the FDA. But most of the information we see on safety labels comes from clinical trials, which aren’t typically representative of the actual populations of patients who will take the medication. Working with us, the FDA will be able to see the real-world impact of taking medications over time, which can help identify benefits and risks earlier. The FDA isn’t just talking about patient-centricity; they are partnering with us to work directly with patients, and give them a collective voice as part of the FDA’s surveillance system.

How does the FDA normally hear about side effects?
Right now the FDA uses a voluntary reporting system consisting of individual case safety reports, the majority of which are submitted by healthcare professionals and patients to drug product manufacturers, who then are required to report them to the FDA. Our data are different in that the information is generated by patients themselves, and provide real-time insights about what its like to use medical products over time, like tolerability of the drug and factors that may influence taking the drug as prescribed.

When did PatientsLikeMe’s start gathering information about side effects and adverse events?
We’ve actually been collecting information about patients’ experiences with treatments, including patient-attributed side effects, since we launched the website in 2006. In 2008 we took steps to formalize adverse event reporting by developing a customized version of the FDA’s MedWatch tool for use in a pilot project with our MS community. The pilot set us on a path to develop our future drug safety functionality. By 2009 we had created a fully integrated, standards-based drug safety platform, the first on social media. It enabled industry partners to meet their regulatory obligations.

What’s the future?
It’s pretty exciting! The patient experience can more deeply inform the way medications are regulated. And patient-reported data can ultimately have a greater impact on the way that drugs are developed. This collaboration can lead to all of that.

Share this post on Twitter and help spread the word.


Sally Okun reports back from the FDA Patient-Focused Drug Development Public Meeting on IPF

Posted October 16th, 2014 by

On September 26th I was at the FDA for the Patient-Focused Drug Development Public Meeting on IPF. This is one of 20 meetings that the FDA is holding to learn directly from patients and their caregivers about daily life with the specific condition’s symptoms and treatments. (You can learn more about these meetings here.) I have attended a number of these meetings and often have meaningful data to share from our own PatientsLikeMe members. So far, however, the FDA has only allowed patients and caregivers to present and participate as panelists.

So to get as much of our members’ real-world health data in front of the FDA as possible, I take full advantage of the public comment period at the end of each meeting. It’s a short 3-5 minutes of time, but it’s a chance to share insights from PatientsLikeMe members and to reflect on some of what’s been discussed during the public meeting. In addition, we submit a full report to the FDA’s public docket that is open for comment two full months following the meeting. The report includes a community profile and the results of any polls PatientsLikeMe members have taken part in prior to the meeting.

This meeting was different
It was my pleasure and privilege to meet and spend time with a member of our PatientsLikeMe community who was selected by the FDA to be one of the panelists. LaurCT did an amazing job sharing her challenge in getting an accurate diagnosis – which took a number of years. She also shared with the FDA and all the folks in the room that she did not learn about the Center of Excellence for IPF located only an hour from her home from her now former pulmonologist after finally getting the diagnosis of IPF – she learned about it from other IPF patients on PatientsLikeMe. (Thank you for sharing Laura!)

Many in the room acknowledged that a cure may not be found in their lifetime but they also conveyed to the FDA a palpable sense of urgency for treatments to slow the progression of the disease to reduce their symptom burden and increase their ability to participate more fully in life. What was startling was how often people shared how the lack of knowledge about IPF among physicians – even among pulmonary specialists – led to delays in their diagnosis and for some resulted in misdiagnosis and inappropriate treatment.

One specialist, Dr. David Lederer, co-director of the interstitial lung disease program at Columbia University Medical Center in New York City and well known to many patients in the room for his work in IPF, used his few minutes in the public comment period to call for therapies that help his patients “live longer, healthier, more normal and independent lives.” He provocatively said that “oxygen should be free” and called for more study of the benefits of oxygen combined with exercise which today are the only things that help people with IPF actually feel better. This was borne out in our poll as well.

A family thing
In addition, there is an important familial connection that many feel is not getting sufficient attention in research. One woman shared her family’s story starting with her father’s diagnosis and death followed by the deaths of each of his 4 brothers of the same disease. A number of people talked about family members who died over the years with similar respiratory symptoms and questioned whether they in fact may have died of undiagnosed IPF.

Your voices transformed
While the opportunity for patients to be heard cannot be diminished, I along with others in policy and advocacy circles remain concerned by the anecdotal nature of these FDA proceedings. At each meeting, there are a few polling questions with audience clicker devices, but there is no systematic way of transforming the very powerful patient narratives that are shared into meaningful and usable data. At PatientsLikeMe, your stories and your voices are best amplified and speak most loudly when transformed into data that is both meaningful and measureable.

All that said, it’s worth watching the free webcasts of the meeting, which you can find here.

PatientsLikeMe member SallyOkun

Share this post on Twitter and help spread the word for IPF.


Improving drug safety through the patient voice

Posted November 22nd, 2013 by

At PatientsLikeMe we’re interested in bringing the voice of our patients to the attention of those who make drug products and to the regulators that approve them. Since 2008 we have conducted a series of projects to collect safety information from some of our member communities. We’ve worked with our pharmaceutical partners to help them better understand the safety experiences of patients while they are using certain drug products.

I’d like to introduce you to a new acronym – P.R.O.S.P.E.R.  It stands for Patient-Reported Outcomes in Safety Event Reporting and it promotes the value of including patient experiences in monitoring the safety of drug products during clinical trials and after drugs are approved.

The PROSPER Consortium is co-led and supported by PatientsLikeMe and Pope Woodhead, a UK healthcare firm, with input from most global pharmaceutical companies, many clinical and academic groups, as well as regulators, researchers and patient advocates.

A report from the Consortium was recently published in the journal Drug Safety that provides guidance for using patient reported outcomes (PROs) for safety monitoring processes. These are just a few of the reports findings…

  • The patient perspective is an essential component of drug safety
  • Patient-centeredness and patient safety are emerging as core elements of any responsive health system
  • The potential clinical utility and value of real-world information obtained from real patients taking real drugs is great1

Although the U.S. Food and Drug Administration (FDA) has accepted safety reports from consumers for 20 years through its MedWatch program, other parts of the world only accept reports on adverse reactions to drugs from health professionals. While patient reporting has recently been encouraged there remain skeptics who doubt its value.

PatientsLikeMe is part of the PROSPER Consortium because we believe the patient voice – your voice – is essential to improving the safety of drugs and medical products. As we look ahead to 2014, we’ll be exploring opportunities to amplify your voice across all areas of health care to improve safety, starting with the FDA and the EU counterpart, the European Medicines Agency.

PatientsLikeMe member MollyCotter


1 http://link.springer.com/article/10.1007/s40264-013-0113-z/fulltext.html