Please enable JS

GHLF offers comments on off label uses of medications

1/ 05


AUTHOR: 50SN Staff

DATE: Jan 05, 2017


SHARE ON: Twitter - Facebook - LinkedIn

GHLF offers comments on off label uses of medications

Director of State and National Advocacy, Stephen Marmaras, commented on off label uses of medications on November 9th, 2016. He acknowledges the promise to patients off label usage offers, as well as the disparity of education between large medical centers and more isolated rural providers. Marmaras proposes further distribution of peer reviewed articles for education, the development of a platform to research the impacts of off label use, and mimicking of existing FDA fee structure to ensure accessibility. Read the full commentary below:

Global Healthy Living Foundation

Public Meeting on Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Products; Public Hearing; Request for Comments

November 9-10, 2016


On behalf of the Global Healthy Living Foundation (GHLF), I would like to thank the FDA for giving us the opportunity to provide our comments on this very important guidance. GHLF works to advocate for improved access to care at the community level and represents patients suffering from conditions such as chronic pain, cancer, diabetes, and psoriasis.

Off-label use of medication holds a lot of promise for our patients, who are often suffering from conditions with unmet medical needs. Our patients, many of whom often spend weeks, months and even years searching for medications that work for them, have found relief in the off-label usage of medications.

The first question in the federal register notice asks what the benefits may be of increased communication of off-label use. Often, patients who are treated at large medical centers in urban areas are more likely to have well-networked doctors who are aware of off-label usages through interactions with the larger number of colleagues compared with the networking opportunities at smaller institutions or private clinics. Additionally, patients who live in rural areas may not be exposed to highly networked healthcare professionals who could know about all available off-label treatment options.

We believe increasing the ability of manufacturers to communicate with all doctors about off-label usage has the potential to deliver uniform knowledge about off-label use, whether in a large urban or isolated rural healthcare facility. This theoretically would expand access to treatments for patients who desperately need them, regardless of site of care.

With respect to the question on the effect of off-label usage and clinical trial enrollment, we feel that expanded communication could increase patients’ interest in research, and provide opportunities for manufacturers to incorporate real world evidence into their data collection which is a priority for the FDA.

GHLF is the recipient of a PCORI contract to build and populate our own patient powered research network, AthritisPower. The promise of new uses for existing treatments could encourage patients to further participate in such networks, especially if they understand that their longitudinal reporting of patient reported outcome measures may generate evidence to support off-label use.

At the same time, we don’t want off-label use to discourage manufacturers from engaging in appropriate research to prove a drug’s safety and efficacy in other indications. We believe there are ways in which the agency can enable off-label communication while still incentivizing appropriate research. One way would be to bypass the traditional clinical trial for off-label indications in favor of real-world evidence that could be provided by PCORnet studies or other large pragmatic trials.

It comes as no surprise that the use of the internet and social media has allowed patients to network with one another. As the founders of CreakyJoints, the largest online community for arthritis patients, GHLF is proud to facilitate many of these discussions. Increased communication between physicians and manufacturers about off-label uses will inevitably result in more conversations among patients who will have spoken with their better-informed physicians. This is good for personal and population health.

We hope the FDA will allow these conversations to take place, but we also understand there is a need to include parameters that ensure patient safety. To help achieve this we recommend:

  • Distributing peer-reviewed journal articles on off-label use and presenting data on off-label use at scientific meetings as these practices promote dissemination of evidence-based practice.
  • Creating an off-label research structure to ensure safety and efficacy, including comparative effectiveness research and post-marketing surveillance that incorporates patient reported outcome measures. GHLF believes it is in a unique position, along with other members of PCORnet, to contribute to this effort.
  • GHLF recommends FDA mimic its existing user fee structure in order to assure a vibrant, peer-reviewed, data-driven, and regulated off-label environment.

In conclusion, we support greater communication between manufacturers and doctors regarding off-label use and feel it will lead to improved access to much needed treatments for our patients. We also believe if the need for data on off-label use is communicated to patients, it could encourage their participation in patient-centered research initiatives, such as ArthritisPower. However, the FDA should set parameters that fully enable this valuable communication while simultaneously promoting patient safety with transparency in data and disclosure of potential risks associated with off-label usage.


Thank you for your time and attention.