FDA Asks Narcolepsy Patients How Symptoms Change Over Time
This article was originally published in The Pink Sheet Daily
Executive Summary
Agency plans a half-day meeting Sept. 24 on narcolepsy symptom burden and current treatment options, part of its patient-focused drug development initiative under PDUFA V.
FDA is asking narcolepsy patients to describe the ebb and flow of their disease symptoms at a Sept. 24 meeting under its patient-focused drug development initiative.
In the list of questions for the half-day meeting, FDA requests narcolepsy patients address how their symptoms have changed over time. “Do your symptoms come and go,” the agency asks. “If so, do you know of anything that makes your symptoms better? Worse?”
The agency also seeks patient input on a series of other questions related to symptoms and their impact on daily living, as well as current treatment options and their downsides.
FDA-approved drugs that carry narcolepsy indications include Jazz Pharmaceuticals PLC’s Xyrem (sodium oxybate), Cephalon Inc.’s Provigil (modafinil) and Nuvigil (armodafinil), Teva Pharmaceutical Industries Ltd.’s Adderall (mixed amphetamine salts) and Novartis AG’s Ritalin (methylphenidate).
Meetings Grow Shorter, More Focused
The narcolepsy meeting is the fourth of 20 disease area meetings to be conducted under the patient-focused drug development initiative, which was created through the 2012 reauthorization of the Prescription Drug User Fee Act. The program is aimed at systematically gathering patient views on the burden of disease, currently available treatment options and risk/benefit tolerance, information the agency will use in the course of its regulatory decision-making.
Narcolepsy was among the list of 16 disease areas that FDA announced will be the focus of patient meetings in fiscal years 2013-2015 (Also see "FDA’s Patient-Focused Meetings: Round One Selections Include Diseases Broad And Rare" - Pink Sheet, 22 Apr, 2013.). Inclusion on the list followed a robust letter-writing campaign by the narcolepsy patient community (Also see "Narcolepsy Patients Hope FDA Attention Will Spur Drug Research, Shed Misperceptions" - Pink Sheet, 13 May, 2013.).
FDA held the first patient-focused meeting in April on chronic fatigue syndrome and myalgic encephalomyelitis, conditions for which there are no approved treatments.
The CFS/ME session went beyond merely gathering input on symptom burden and current treatments. The one-and-a-half day meeting also included presentations from government officials, clinicians and academics on expedited drug development and approval pathways, drug repurposing, clinical trial design and endpoints and clinical outcome assessments (Also see "Chronic Fatigue Meeting Shows Benefits, Hurdles To Patient-Focused Drug Development" - Pink Sheet, 6 May, 2013.).
Subsequent meetings have been shorter, with only brief presentations by FDA officials, and more concentrated on hearing the patient voice.
FDA convened a full-day meeting June 14 on HIV/AIDS, with a key focus being patients’ perspectives on HIV cure research (Also see "FDA Informed Consent Guidance Under Development; HIV Studies Present Unique Challenges" - Pink Sheet, 24 Jun, 2013.).
At a half-day meeting on lung cancer June 28, the agency gathered feedback on how patients weigh the importance of prolonging survival versus treating disease symptoms. FDA officials also heard that it can be difficult to distinguish between symptoms that are a manifestation of the underlying disease versus those resulting from cancer treatment (Also see "FDA Sees Challenge In Distinguishing Lung Cancer Symptoms From Drug Side Effects" - Pink Sheet, 8 Jul, 2013.).
Chronic Symptoms, Current Treatments
Narcolepsy is a chronic disorder of the central nervous system caused by the brain’s inability to control sleep-wake cycles. The disease is characterized by excessive daytime sleepiness, cataplexy, hallucinations and disturbed nocturnal sleep.
“Although there is no cure for narcolepsy, medications and lifestyle modifications can help patients manage their symptoms,” the meeting notice states. “FDA is interested in obtaining a better understanding of patients’ perspectives on the severity of the disease and assessments of available therapies.” The notice is scheduled for publication in the Federal Register July 19.
Except for the question about symptom changes over time, the discussion points outlined in the meeting notice largely mirror those posed by the agency at one or more of the previous patient-focused meetings. However, the agency is continuing to refine the questions as it gains more experience with the program.
On the first topic of disease symptoms and daily impact, the agency asks, “Of all the symptoms that you experience because of your condition, which one to three symptoms have the most significant impact on your life? (Examples may include excessive daytime sleepiness, cataplexy, etc.).”
“Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition,” the agency asks, citing examples such as sleeping through the night and work and school performance.
Turning to current treatment approaches, the agency asks patients to comment on drug therapies and non-drug measures, such as lifestyle modifications, used to help treat the condition or control symptoms. The agency wants to know what specific symptoms these therapies address, how the treatment regimen has changed over time and why.
“How well does your current treatment regimen treat the most significant symptoms of your disease,” the agency asks. Patients are requested to comment on how well current therapies improve their ability to perform specific activities that are important to their daily lives and how well these treatments have worked as the condition changes over time.
The agency also asks about adverse events and other challenges with available treatments. “What are the most significant downsides to your current therapies, and how do they affect your daily life? (Examples of downsides may include bothersome side effects, inconvenient dosing schedules, access issues, etc.)”
The final question asks patients to consider what specific thing they would look for in an ideal therapy, assuming there is no complete cure.