Drugs

External Resources or Information Related to Patients’ Experience

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This webpage is intended to facilitate public discussion of patient-focused drug development and evaluation. This webpage provides links to certain publicly available external reports and resources relating to patient experience data. The patient community, patient advocates, researchers, drug developers, and federal agencies may find these materials useful.

Please note that although FDA reviews the materials at these links before posting them to ensure that the materials are within the scope of the webpage, FDA does not assess their scientific merit or compliance with regulatory requirements. Our decision to post links to these materials does not reflect an endorsement of their authors, sponsors, or content.

For more information regarding what types of resources may be included on this webpage, how to submit a publicly available website link to FDA, and other general questions, please review our Frequently Asked Questions. We request that links include a cover page or similar opening statement as part of their report or resource to provide information about the authors, funding, and related information. For specific questions related to a report or resource, FDA recommends reaching out to the point of contact listed on this cover page.

To help expand the benefits of FDA’s Patient-Focused Drug Development (PFDD) initiative, FDA welcomes patient organizations to identify and organize patient-focused collaborations to generate public input on other disease areas. Submitted links to summary meeting reports from these externally-led PFDD meetings may be found here. FDA also welcomes submission of links to meeting reports from other stakeholder meetings collecting patient perspectives on disease burden and treatment burden.

  • Amyloidosisdisclaimer icon
    In November 2015, the Amyloidosis Research Consortium hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with systemic amyloidosis and their loved ones on the impact of amyloidosis on their daily lives, and their perspectives on approaches to treating amyloidosis.
  • Complement 3 Glomerulopathy (C3G)disclaimer icon
     In August 2017, the National Kidney Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with C3G and their loved ones on the impact of C3G on their daily lives, and their perspectives on approaches to treating C3G.
  • Friedreich’s Ataxiadisclaimer icon 
    In June 2017, the Friedreich’s Ataxia Research Alliance hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Friedreich’s Ataxia and their loved ones on the impact of Freidreich’s Ataxia on their daily lives, and their perspectives on approaches to treating Friedreich’s Ataxia.
  • Lupusdisclaimer icon
    In September 2017, the Lupus and Allied Diseases Association, the Lupus Foundation of America and the Lupus Research Alliance hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with lupus and their loved ones on the impact of lupus on their daily lives, and their perspectives on approaches to treating lupus.
  • Myotonic Dystrophy disclaimer icon 
    In September 2016, the Myotonic Dystrophy Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with myotonic dystrophy and their loved ones on the impact of myotonic dystrophy on their daily lives, and their perspectives on approaches to treating myotonic dystrophy.
  • Osteoarthritisdisclaimer icon
    In March 2017, the Arthritis Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with osteoarthritis and their loved ones on the impact of osteoarthritis on their daily lives, and their perspectives on approaches to treating osteoarthritis.
  • Spinal Muscular Atrophydisclaimer icon
    In April 2017, Cure SMA hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with spinal muscular atrophy and their loved ones on the impact of spinal muscular atrophy on their daily lives, and their perspectives on approaches to treating spinal muscular atrophy.
  • Tuberous Sclerosis Complexdisclaimer icon 
    In June 2017, the Tuberous Sclerosis Alliance hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with tuberous sclerosis complex and their loved ones on the impact of tuberous sclerosis complex on their daily lives, and their perspectives on approaches to treating tuberous sclerosis complex.

The proposed draft guidance relating to patient experience data that are listed here have been drafted and submitted by external stakeholders. As with other resources on the webpage, the proposed draft guidance and their content are not endorsed by FDA, and posting a link does not mean FDA has decided to adopt the proposed draft guidance.

Natural history studies track the course of disease over time, identifying demographic, genetic, environmental, and other variables that correlate with its development and outcomes in the absence of treatment. Website links to other publicly-available reports or documents providing disease-specific background on the condition and unmet medical need may also be found here.

 

 

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