Stakeholder Opinions: Endometriosis - Scientific clarity will unlock market potential

Table of Contents

• ABOUT DATAMONITOR HEALTHCARE
  • About the Women' s Health pharmaceutical analysis team
• CHAPTER 1 EXECUTIVE SUMMARY
  • Scope of the analysis
  • Contributing experts
  • Datamonitor insight into the Endometriosis market
  • Related reports
• CHAPTER 2 MARKET DYNAMICS
  • Endometriosis market definition
    • Limitations in sizing the endometriosis market with IMS data
  • Current market overview
    • Market size has yet to recover from 2006 fall
      • US market changes have contributed to 7MM market shrink
      • Political changes have boosted sales in Spain
    • POCs are the most common treatment option for endometriosis
    • Lupron remains the most popular GnRH agonist treatment
• CHAPTER 3 DISEASE OVERVIEW
  • Definition of endometriosis
    • Classification of endometriosis: Re-AFS
  • Etiology of endometriosis: still not well understood
    • Retrograde menstruation and implantation
    • Lymphatic/vascular dissemination
    • Developmental coelomic metaplasia
    • Genetic predisposition
    • Environmental factors
    • Immunological susceptibility
    • Dynamic progression of endometriosis
    • Recurrence in endometriosis
  • Symptoms of endometriosis
  • Epidemiology of endometriosis
    • Socio-demographic contributors to endometriosis prevalence
    • Limitations of endometriosis epidemiology analysis
      • Factors specific to endometriosis:
      • Factors generalized to all epidemiology analysis:
    • The gold standard endometriosis epidemiology study
    • Prevalence estimates: the general versus gynecologic population
      • Prevalence of endometriosis in the general population is assumed to be 10%
      • Prevalence of endometriosis in the gynecologic population may be more representational
  • Unmet need in endometriosis
    • Understanding the etiology of endometriosis will aid the development of prophylactic and specific treatments
    • Non-invasive diagnostic procedures will lower morbidity
    • Physician education will quicken time to diagnosis
    • Patient education promotes treatment seeking
    • Better pain management concepts will help women cope
• CHAPTER 4 TREATMENT TRENDS
  • Endometriosis patient diagnosis and referral algorithm
    • ' Under-diagnosed and over treated'
  • Treatment of endometriosis
    • Combined oral contraceptives: first-line therapy
      • Short term efficacy is similar to that of GnRH agonists
    • Progestogen treatments are most commonly used therapy
    • Antiprogestogen use is limited to gestrinone
    • Androgens: Danazol-first treatment indicated for endometriosis
      • Efficacious for pain relief
      • Side effects limit usefulness of product
      • Danazol remains useful for niche patients
    • GnRH agonists: short-term efficacy at the expense of side effects
      • Characteristics of successfully marketed GnRH agonists
      • Lupron: the ' gold standard'
      • Lupron with add-back makes side effects more manageable
    • Surgery for severe and non-responsive endometriosis
      • Laparoscopic surgery: a range of techniques can reduce pain
      • Hysterectomy: the last treatment option
  • Patient treatment goals
    • Pain reduction
    • Pregnancy
• CHAPTER 5 RESEARCH AND DEVELOPMENT
  • The ideal drug for the treatment of endometriosis
    • Efficacy must prevent recurrence
    • Safety essential in a young patient population
    • Side-effects profile must be improve compliance
    • Cost must be kept low for long-term use
  • Pipeline overview
    • Considerable variety in mechanism, delivery mode, and manufacturer
    • Large pipeline is beginning to mature
    • New methods of drug delivery attempt to fulfill unmet need
      • Nasal administration may be hindered by unwelcome irritation
      • Vaginal route unpopular in conservative patient population
    • Late-stage products offer some hope to patients
      • Visanne progestogen could benefit from specific endometriosis label
      • Balance will need marketing partner to realize Libra' s full potential
      • FP-1096 benefits from a novel drug delivery technique
  • Mechanisms of action of pipeline products
    • Aromatase inhibitors may offer long-term remission
    • Chemokine receptor antagonists center on inflammatory and immune response
      • Immunomodulators are still regarded warily
    • Combination products will not radically alter treatment
    • Promising estrogen receptor beta agonists require more research
    • GnRH antagonists have important advantages over agonists
    • Progestogen agonists are as effective as marketed therapies
    • Selective progestogen receptor modulators
  • Clinical trial design in endometriosis
    • Difficulties inherent in clinical trials of endometriosis therapies
      • Patient recruitment must be carefully considered
    • Endpoints in clinical trial design
      • Arguably pain is the endpoint of most relevance to the patient
      • That lesion size should be a secondary endpoint is a contentious issue
      • Quality of life scale expands on the endpoint of pain
• CHAPTER 6 CURRENT MARKET ISSUES
  • Raising the profile of endometriosis
    • Breaking the taboo
      • Raising awareness: lessons from other disease areas
    • The EEA takes the lead in lobbying in Europe
  • Current issues in diagnosis of endometriosis
    • Biomolecular and genetic diagnostics are desirable but not trusted
      • Current research
      • CA-125 testing has had a mixed response
    • Imaging diagnostics currently offer the most promise
  • The endometriosis indication
    • Physicians find specific labeling increasingly useful
    • The potential of endometriosis as a secondary indication
      • Uterine fibroids
      • Menorrhagia (excessive bleeding)
• BIBLIOGRAPHY
  • Journal papers
  • Websites
• APPENDIX
  • IMS data
    • Medical sales data caveats
    • Medical sales data methodology
  • Contributing experts
  • About Datamonitor
    • About Datamonitor Healthcare
  • Datamonitor Healthcare' s therapy area capabilities
    • About the Disease analysis team
    • Disclaimer
  • List of Tables
    • Table 1: ICD-10 codes used to define the endometriosis market
    • Table 2: Frequency and location of endometriosis implants in the pelvis
    • Table 3: Frequency of symptoms among surgically diagnosed endometriosis patients, 2007
    • Table 4: Common symptoms of endometriosis and other conditions
    • Table 5: Epidemiology overview of endometriosis, 2007
    • Table 6: Endometriosis prevalence in the 7MM general population, 2007
    • Table 7: Endometriosis prevalence in the CPP (gynecological) population, 2007
    • Table 8: Endometriosis prevalence in the infertile (gynecologic) population across the seven major markets, 2007
    • Table 9: Summary of endometriosis treatment with danazol
    • Table 10: Endometriosis-specific sales of GnRH agonists in the seven major markets combined, by molecule, 2006-07
    • Table 11: Cost of select GnRH agonists in the UK, 2007
    • Table 12: Lupron product range, 2007
    • Table 13: Overview of the endometriosis pipeline, 2007
    • Table 14: Mechanism of action of the drugs in the endometriosis pipeline, 2007
    • Table 15: Action taken by the European Endometriosis Alliance in H1, 2007
  • List of Figures
    • Figure 1: The seven major endometriosis markets by country, 2003-07
    • Figure 2: The five major European (EU) endometriosis markets. 2003-07.
    • Figure 3: Seven major endometriosis market size, split by region, 2003--07
    • Figure 4: Volume sales of products within endometriosis treatment types in the seven major markets combined, 2007
    • Figure 5: Market share and market growth of key GnRH agonists in the seven major endometriosis markets, 2006-07
    • Figure 6: Prevalence of CPP and endometriosis in the female population and prevalence of endometriosis in the CPP population (000s), 2007
    • Figure 7: Causes of infertility for US couples undergoing assisted reproductive technology using fresh, non-donor eggs or embryos, 2004
    • Figure 8: Prevalence of infertility and endometriosis in the female population and prevalence of endometriosis in the infertile population (000s), 2007
    • Figure 9: Unmet needs in endometriosis as identified by key opinion leaders, 2007
    • Figure 10: Patient diagnosis referral pathway in endometriosis, according to key opinion leaders, 2007
    • Figure 11: Side-effect versus efficacy profile of key treatments on the endometriosis market according to interviewed EU and US opinion leaders, 2007
    • Figure 12: An endometriosis patient treatment pathway dependent on treatment goal
    • Figure 13: Frequency of products in the pipeline, by development stage, 2007
    • Figure 14: Breakdown of pipeline products by delivery method, where known, in 2007