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Myelodysplastic Syndrome Discussion

Myelodysplastic Syndrome Discussion. 16 January 2006. Prepared by MedPanel, Inc. Myelodysplastic Syndrome Discussion. Outline. Project Objectives MedPanel Methodology & Panelists Major Takeaways Detailed Findings Conclusions. Project Objectives MedPanel Methodology & Panelists

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Myelodysplastic Syndrome Discussion

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  1. Myelodysplastic Syndrome Discussion 16 January 2006 Prepared by MedPanel, Inc.

  2. Myelodysplastic Syndrome Discussion Outline • Project Objectives • MedPanel Methodology & Panelists • Major Takeaways • Detailed Findings • Conclusions

  3. Project Objectives MedPanel Methodology & Panelists Major Takeaways Detailed Findings Conclusions

  4. Project Objectives • Convene online focus group of hematologist-oncologists (heme-oncs) experienced in treating MDS • Gather feedback from this group regarding recently introduced & upcoming products • Vidaza® (azacitidine), FDA-approved 5/04 • Dacogen™ (decitabine), pending FDA approval • Revlimid® (lenalidomide), pending FDA approval • Explore alternative indications and/or patient populations/sub-types • Determine impact of products on current marketplace (immediate and 6-month perspective)

  5. Project Objectives MedPanel Methodology & Panelists Major Takeaways Detailed Findings Conclusions

  6. MedPanel MethodologyPanelist Recruitment We recruited based on a specific set of screening criteria developed Proprietary Database Screening Criteria • Hematologist-oncologists (n=6) • All panelists must be: • In practice 2-25 years • Panel average: 12 years • Spending > 75% time in clinical practice • Panel average:93% • Treating a > 10 MDS patients per month • Panel average:22 patients/month Outside the Database Simultaneous discussion guide development & panelist recruitment • Asynchronous, threaded discussion • Panelists respond anonymously • Questions posed by physician moderator to expand, probe, or clarify responses as necessary

  7. MedPanel MethodologyPanelist Details

  8. Project Objectives MedPanel Methodology & Panelists Major Takeaways Detailed Findings Conclusions

  9. Major TakeawaysTreatment of MDS Patients Treatment by patient risk-group • Low-risk MDS patients are typically offered supportive care with transfusions of pRBC or platelets • Intermediate-risk MDS patients receive supportive care with transfusions with or without chemotherapy • Most high-risk MDS patients are treated with Vidaza monotherapy • Patient age, PS, and IPSS-defined risk category help to determine which treatments are given • Decreased progression of disease and improvement in toxicity measures are the most important product attributes for treatment of MDS • Other important attributes include control of symptoms and improvement in quality of life and efficacy measures • Most panelists expressed minimal satisfaction with the performance of current MDS treatments on the important product attributes • The major unmet needs for treatment of MDS are the low treatment response rates and the lack of a cure Key product attributes & needs

  10. Major TakeawaysRole of Vidaza & Dacogen Vidaza • Currently administered to in the outpatient setting at 75 mg/m2 per day for seven consecutive days every 28 days for 4 to 8 cycles or as long as beneficial • Discontinued in up to 50% of patients most often due to unmanageable side effects, usually after 2-3 cycles • Major strengths include FDA approval, outpatient administration, prolonged survival, and improvement in quality of life • Weaknesses include high cost, low response rates, no significant improvement in overall survival, myelosuppression, inconvenient administration, and short duration of response • Major strength is the high treatment response rate • Compared to Revlimid, its main advantage is that it is indicated for all MDS patients, not a subset • Major weakness is its toxicity (myelosuppression) • Compared to Revlimid its main disadvantages are that Dacogen is more toxic and is delivered intravenously Dacogen

  11. Major TakeawaysRole of Dacogen & Revlimid Dacogen use & drivers • Dacogen is likely to be used primarily in high-risk MDS patients in the outpatient setting at a daily dose of 10 mg/m2 administered subcutaneously • The major barrier to the adoption of Dacogen is lack of FDA approval; a better dosing schedule would facilitate the acceptance and use of Dacogen • Major strengths are its efficacy in patients with 5q- syndrome and oral administration • Major weaknesses include patient compliance, reimbursements issues, and added toxicity • Barriers to the adoption of Revlimid include: cost and reimbursement issues, side effects, and lack of clinical evidence • While oral Revlimid would be used primarily in patients with 5q- syndrome, the majority of panelists would also consider it for patients with non 5q- disease • Additional evidence of activity in patients with 5q- syndrome, low cost, patient assistance programs, and availability of samples would facilitate Revlimid’s acceptance and use Revlimid

  12. Major TakeawaysOther Key Issues Details on 5q- • Between 10% and 50% of panelists’ MDS patients are low-risk patients with non 5q- disease and up to 50% are high-risk patients with 5q- syndrome • Patients with 5q- syndrome are currently defined by chromosome and cytologic analyses • Reimbursement has a strong impact on therapy choices for the majority of panelists, however, none have had significant reimbursement issues for MDS • Use of Revlimid for MDS treatment will be reimbursement-dependent, whether it is priced at $4000 or $6000 per month. • Aranesp, thalidomide, and arsenic trioxide will impact the MDS pharmaceutical marketplace, while Telintra, tipifarnib, and amifostine are not expected to have an impact • Only one panelist was aware of products in development that would compete with Dacogen and Revlimid • These include: the liposomal form of TLKI-199, SCIO-469, tipifarnib, lonafarnib, and bryostatin-1 Cost & reimbursement Other potential MDS treatments

  13. Major TakeawaysFuture Treatment Patterns Treatment landscape in 6 months • In the next 6 months, the use of Dacogen and Revlimid is expected to increase while Vidaza will begin to decline • Dacogen will meet unmet needs for the treatment of transfusion-dependent patients, while Revlimid will meet unmet needs for the treatment of patients with 5q- syndrome • Dacogen will be used first-line for the majority of MDS patients, except for those with 5q- syndrome where Revlimid would be the treatment of choice • The majority of panelists feel that Revlimid and Dacogen will not completely replace Vidaza although they will limit its use to a second-line treatment • The ideal Vidaza patient will be those unable to tolerate Dacogen

  14. Major TakeawaysFollow-Up Survey Results • Between 20% and 60% of panelists’ MDS patients are high risk • Between 40% and 80% are low risk • In one year, Dacogen use is expected to increase most in high risk patients while Revlimid will increase in both patient categories High Risk Patients Current & Expected Treatment Low Risk Patients Current & Expected Treatment Average Percent of Patients Currently Treated or Expected to be Treated in One Year

  15. Project Objectives MedPanel Methodology & Panelists Major Takeaways Detailed Findings Conclusions

  16. Detailed FindingsCurrent Treatment of MDS Both low- and intermediate-risk MDS patients are usually offered supportive care with transfusions of pRBC (packed red blood cells) or platelets • Other treatment strategies for low-risk patients include: • Growth factors / Cytokines • Procrit or Aranesp • Observation • Other treatment strategies for intermediate-risk patients include: • Growth factors / Cytokines • Chemotherapy (Vidaza monotherapy) Most high-risk MDS patients are treated with Vidaza monotherapy • Other treatment strategies for high-risk patients include: • Supportive care with prn transfusions of pRBC or platelets • Growth factors / Cytokines • Bone marrow transplantation • Intensive chemotherapy (combinations and high doses)

  17. Detailed FindingsImportant Therapy Attributes Decreased progression of disease and improvement in toxicity measures are the most important product attributes for treatment of MDS • Other important product attributes include: • Control of symptoms • Improvement in quality of life measures • Ease of administration • Improvement in overall survival • Minimal risk of cytopenia • Decreased need for transfusion • Minimal risk of infection • Cost The panelists are dissatisfied with current MDS treatments’ performance on important product attributes • “Improving, but a long way to go” • “Effective, but supportive in nature”

  18. Detailed FindingsUnmet Needs The major unmet needs for treatment of MDS are the lack of a cure and the low response rates associated with current treatments • Other unmet needs include: • High costs • High toxicity rates • Continued need for transfusion • Short time to progression • Increased risk of infection • Inconvenient administration requirements

  19. Detailed FindingsMeeting Unmet Needs New products will meet some important unmet needs in select patient segments • Vidaza: • Transfusion-dependent patients • Dacogen: • Transfusion-dependent patients • Revlimid: • Patients with 5q- syndrome • Patients with low/Int-1 IPSS scores

  20. Detailed FindingsStrengths & Weaknesses of Vidaza Strengths of Vidaza include: • FDA approved • Outpatient administration • Prolonged median survival time • Decreased risk of AML transformation • Decreased need for transfusion • Improvement in quality of life measures Weaknesses of Vidaza include: • High cost • Low response rates • 7 day treatment requirements • No significant improvement in overall survival • Myelosuppression • Not curative treatment • Inconvenient administration • Short duration of response

  21. Detailed FindingsUses of Vidaza Vidaza is currently administered to MDS patients in the outpatient setting at 75 mg/m2 per day for seven consecutive days every 28 days for 4 to 8 cycles • Vidaza could also be used for the following patients: • High-risk patients • Patients refractory to growth factor and cytokine treatment • Patients progressing to intermediate- and high-risk status • Patients in lower risk groups requiring multiple transfusions Vidaza is administered for as many cycles as the patient benefits and is discontinued due to unmanageable side effects • Reasons for continuing Vidaza treatment include: • “I give as long as improvement seen even if more than 4 cycles.” • “Complete response may require more than four cycles.” • “May take more than 4 cycles to see efficacy.” • “…or to best RR.”

  22. Detailed FindingsStrengths & Weaknesses of Dacogen The major strength of Dacogen is its high treatment response rate • Other strengths include: • Strong cytogenetic response • Prolonged median survival time • 5 day or once per week dosing schedule The major weakness of Dacogen is its toxicity, particularly myelosuppression (pancytopenia) • Other weaknesses include: • Inpatient administration • Lack of FDA approval • Lack of clearly defined treatment regimen (dosing and administration)

  23. Detailed FindingsBarriers & Facilitators for Dacogen The major barrier to the adoption of Dacogen is lack of FDA approval • Other barriers include: • Toxicity concerns • Reimbursement issues • Inconvenient dosing schedule • Lack of clearly defined treatment recommendations A better dosing schedule would facilitate acceptance and use of Dacogen • Other facilitators include: • Clinical evidence of efficacy (from large-phase III clinical trials) • Improved toxicity profile

  24. Detailed FindingsUses of Dacogen Dacogen is likely to be used primarily in high-risk MDS patients in the outpatient setting at a daily dose of 10 mg/m2 administered subcutaneously • Dacogen also could be used in the following patients: • Patients with intermediate 1-2 IPSS scores • Patients refractory to growth factor treatment • Revlimid non-responders • Vidaza non-responders

  25. Detailed FindingsStrengths & Weaknesses of Revlimid Strengths of Revlimid include: • High response rates in patients with 5q- syndrome • Oral administration • Decreased toxicity and equivalent efficacy to thalidomide Weaknesses of Revlimid include: • Low patient compliance • Reimbursement issues • Toxicity • Neutropenia • Neuropathy • Constipation • Fatigue • Hypercoagulation • Bone marrow toxicity

  26. Detailed FindingsBarriers & Facilitators of Revlimid Barriers to the adoption of Revlimid include: • Cost • Lack of FDA approval • Lack of clinical evidence • Reimbursement issues • Myelosuppression in cytopenic patients • Hypercoagulation • Small target patient population • Low patient compliance Acceptance and use of Revlimid would be facilitated by: • FDA approval • Strong evidence of activity in patients with 5q- syndrome • Oral administration • Low cost • Availability of samples • Patient assistance programs • Additional clinical data

  27. Detailed FindingsUses of Revlimid Oral Revlimid would be used primarily in patients with 5q- syndrome • Revlimid also could be used in the following patients: • Patients with low/intermediate-1 IPSS scores • High-risk patients • Patients who refuse intravenous or subcutaneous treatment • Patients requiring second- or third-line of treatment Most panelists would use Revlimid in patients with non 5q- disease • “…till data says not to since there are no ‘good’ drugs available and Revlimid appears to be non-toxic.” • “…as there is still some response in Low/Int-1 IPSS scores patients…” • “…should be well-tolerated in these patients.”

  28. Detailed FindingsDacogen vs. Revlimid The main advantage of Dacogen compared to Revlimid is that Dacogen is indicated for all patients with MDS • Other advantages include: • Increased patient compliance with intravenous administration • Decreased reimbursement issues with intravenous administration • No hypercoagulation • Higher response rate The main disadvantages of Dacogen compared to Revlimid are increased toxicity and intravenous administration • Other disadvantages include increased myelosuppression

  29. Detailed FindingsPatient Subtypes Familiarity with published clinical data from the New England Journal of Medicine as well as data submitted to the FDA influence the panelists’ use of Revlimid • Use in low-risk patients with non 5q- disease • “…depending upon patient motivation, availability and insurance.” • Use in high-risk patients with 5q- syndrome • “I will use as first line.” • “…up front in early stage disease.” Patients with 5q- syndrome are defined by chromosome analysis and cytology • Between 10% and 50% of the panelists’ MDS patients are low risk with non 5q- disease • Between 0 and 50% of the panelists’ MDS patients are high risk with 5q- syndrome

  30. Detailed FindingsReimbursement & Cost Issues Physicians have not encountered any reimbursement issues to date for MDS treatments, however, reimbursement does strongly impact their therapy choices Representative comments include: • “The cost threshold is not my concern, it is for the patient and their payers to decide.” • “I do not give any chemo in the office if there is a loss. I will still give the proper chemo but in the hospital clinic.” • “There is no specific number, the criteria for use, no matter what the cost is. If no reimbursement, then no treatment in the office. If reimbursed and it is the best for the patient, then it would be given no matter what the cost.” • “Of course cheaper the drug then its better for hospital…if patient does not have to pay out of pocket cost then cost would have minimal impact as long as this drug is indicated clearly and used appropriately.” • “In a situation where cure is not possible w/multiple options I discuss all w/the patient. Some will decide on the best copay.”

  31. Detailed FindingsNovel MDS Treatments New and existing agents will impact the adoption and use of Dacogen and Revlimid • Novel agents • Liposomal formulation of TLK-199 • SCIO-469 • Tipifarnib • Lonafornib • Bryostatin-1 • Existing agents • Aranesp • Thalidomide • Arsenic

  32. Detailed FindingsFuture Treatment of MDS In the next 6 months, there will be increased use of Dacogen for the treatment of MDS • Other anticipated changes include: • Increased use of Vidaza, Revlimid, and Dacogen as opposed to supportive care • Increased patient selectivity when utilizing new drugs • More treatment options • New combinations of novel and existing drugs • Higher treatment response rates • Increased use of Vidaza in overall MDS patient population Revlimid/Dacogen probably will not completely replace Vidaza • The majority of panelists believe Vidaza will not be replaced • “I do not think either Revlimid or Dacogen will replace Vidaza completely but Vidaza can still be used as 2nd, 3rd, or lower line when the others fail to work.” • A small number believe Vidaza may be replaced within 2 years

  33. Detailed FindingsIdeal Use of Dacogen & Revlimid Dacogen will be used for the majority of MDS patients, not including those with 5q- syndrome • Other candidates include: • High-risk patients • Patients who have relapsed or are refractory to other treatment Patients with 5q- syndrome are the ideal candidates for Revlimid treatment • Other candidates include patients with non 5q- disease as second-, third-, and fourth-line treatment

  34. Project Objectives MedPanel Methodology & Panelists Major Takeaways Detailed Findings Conclusions

  35. Conclusions • Decreased progression of disease and increased tolerability are the most important attributes when selecting a treatment for MDS • Most panelists are not satisfied with the products currently available to treat MDS due to low treatment response rates and high toxicity rates • Although there are currently no reimbursement issues with MDS treatment, reimbursement is essential and requires FDA approval • Additional clinical evidence and/or FDA approval would increase the use and adoption of both Dacogen and Revlimid • Dacogen will meet unmet needs for the treatment of transfusion-dependent patients; Revlimid will meet unmet needs for patients with 5q- syndrome • In the near future, the use of Dacogen for the treatment of MDS is expected to increase, especially in high-risk patients • Revlimid use also is expected to increase, especially among 5q- patients • Vidaza use will continue, although it may be reserved for second- or later-line treatment

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