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Fibromyalgia: Creating a Claim

Fibromyalgia: Creating a Claim. James Witter MD, PhD Arthritis Advisory Committee June 23, 2003. Goals of Meeting. Gather input regarding development and approval for drugs that treat fibromyalgia Enrich analgesic guidance document Address an important public health issue

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Fibromyalgia: Creating a Claim

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  1. Fibromyalgia: Creating a Claim James Witter MD, PhD Arthritis Advisory Committee June 23, 2003

  2. Goals of Meeting • Gather input regarding development and approval for drugs that treat fibromyalgia • Enrich analgesic guidance document • Address an important public health issue • estimates of 4.4 - 10.0 million in U.S. • Better understand how fibromyalgia represents a “model” of chronic pain

  3. Claims and Labels • Although label claims have legal and regulatory uses, their central purpose is to inform health care providers and patients about the documented benefits and risks associated with a product • Claims describe clinical benefits • Accurate product labels allow for effective risk management

  4. Fibromyalgia (FM): What is it?Arthritis Foundation: 2003 • Arthritis-related condition characterized by: • Generalized muscular pain and fatigue • Condition referred to as a “syndrome” because it is a set of signs and symptoms that occur together • Confusing…often misunderstood • Common symptoms with no specific labs

  5. FM: ClassificationAmerican College of Rheumatology: 1990 • History ( > 3 months) of widespread pain • Left and right sided • Above and below waist • Axial skeletal pain must be present • Pain (not tenderness) on digital (4 kg) palpation in 11 of 18 tender points • Both criteria must be satisfied

  6. FM: How is it treated?Arthritis Foundation: 2003 • Education (understand and manage) • Relaxation (ease tension and anxiety) • Exercise (flexibility and CV fitness) • Drugs (decrease pain and improve sleep) • Antidepressants (tricyclics, SSRIs) • Benzodiazepines • Other…

  7. FM: History of Claim • NIH-FDA workshop (March, 2002) • Chronic pain is unmet medical need • Fibromyalgia, example of chronic pain • Arthritis Advisory: Pain (July, 2002) • Claims for marketing for analgesics • www.fda.gov/cder

  8. NIH-FDA Analgesic Drug Development Workshop: Translating Scientific Advances into Improved Pain ReliefClinical Journal Pain, 2003May-June 19(3) 139-147

  9. Chronic Pain: Unmet needsNIH-FDA 2002 • Need new models to better: • Understand important clinical aspects of chronic pain • Understand chronic pain mechanisms which may serve as treatment targets • Design better clinical trials • Ultimately improve treatment of chronic pain

  10. “Models” of Chronic Pain? NIH-FDA 2002 • Osteoarthritis • Lower back pain • Diabetic neuropathy • Cancer pain • Fibromyalgia • AIDS • TMD

  11. Chronic Pain: OutcomesNIH-FDA 2002 • Pain • Patient global • Health-related quality of life • Physical function/disease specific • Rescue meds • Economics • Adverse events

  12. July 2002 AAC: Pain • Analgesic claims described as: • Clinical • acute • chronic • Mechanistic • Minimal clinically-important difference in pain relief • Responder approach in analgesia • Need to revise analgesic guidance

  13. Chronic Pain: Labels July 2002, AAC • General claim (treats all chronic pain) • Replicates of three different pain models • OA, fibromyalgia, cancer pain • Clinical Claim (treats musculoskeletal pain) • OA, fibromyalgia, chronic lower back pain) • Disease claim (treats specific cause) • Osteoarthritis • Fibromyalgia • Chronic lower back pain

  14. Fibromyalgia:How to Structure the Claim? • Treat fibromyalgia as a symptom, or cluster of symptoms • Treat fibromyalgia as a complex disease state with varying clinical presentations

  15. FM: Symptom Approach • Pain outcome • Obvious and necessary • Patient global outcome • Physical function or HRQOL outcome • Adversely impacted by pain • Analgesics need to improve, not worsen • Combination allows improved assessment of the patients’ experience with analgesic

  16. FM: Disease Approach? dysfunction fatigue pain cognitive difficulties depression diminished QOL sleep disturbance

  17. Fibromyalgia/Chronic Pain:What is Important to the patient? • Patient Reported Outcomes (PRO) are: • Patient report of a health condition or treatment • Scientific patient-centered measures that can evaluate change in health outcomes • Handled like other endpoints for both drug approval and promotion • Selection, development and validation issues same as other clinical measures

  18. Ideal Metric-Pain • Understandable to patients and clinicians • in clinical trials • in product label • Applicable across studies • to allow cross trial comparison • Detects a clinically meaningful result • Responsive to differences in analgesia • Valid

  19. WOMAC pain indexWestern Ontario and McMaster Universities 1. Walking on flat surface 2. Going up or down stairs 3. At night while in bed 4. Sitting or lying 5. Standing upright

  20. Osteoarthritis: treatment of signs and symptoms claim • Co-Primary efficacy endpoints: • Pain • Function • Patient global • Trial length: • 3 months

  21. FM Outcome: Considerations • Single or composite question • Statistically/clinically meaningful results • Inclusion and exclusion criteria • Landmark vs. time-weighted approach • Daily pain vs. weekly assessment • Length of clinical trial • Superiority to placebo

  22. FM: Responder approach • Outcomes of interest in same patient • May lessen or eliminate data imputation • Allows flexibility in design to capture different aspects of condition • Widely utilized in RA (ACR 20%)

  23. ACR 20 responder index • > 20% improvement in swollen and tender joint count…….plus... • > 20 % improvement in 3 of following 5: • patient global • physician global • patient pain (VAS) • modified HAQ • acute phase reactant (CRP or ESR)

  24. FM: Responder endpoints? • Required Outcome • Pain • Other Important Outcomes • Quality of life (general or specific) • Dysfunction • Sleep disturbance • Fatigue • Cognitive impairment • Patient Global • Responder is four of six plus pain? • Percent of improvement like ACR 20?

  25. Selecting Core Outcome Domains in Chronic Pain Clinical Trials:IMMPACT recommendations: 2003 • Pain • Physical functioning • Emotional functioning • Patient Global • Negative Health States • Patient Disposition

  26. Fibromyalgia: The Label • End result of randomized, controlled trials • What should it mean to the health care provider? • Who should take it • What type of risk management is involved • What should it mean to the patient? • Relief of pain • Relief of associated symptoms • Duration and degree of relief

  27. Assess the patient… …not just the pain IMMPACT II

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