Académique Documents
Professionnel Documents
Culture Documents
Discussion Guide
Confidentiality We conform to all national and international legislation on data protection and comply with the Market Research Codes of Conduct. Your personal data will be used exclusively for the purpose of this research, and will not be disclosed to the sponsor or any third party without your consent. You have the right to rectify this personal data, or have it deleted. Your responses will be collated with those of other respondents and presented to the sponsor in an aggregate or anonymous form. The interview will be audio recorded to help us in our analysis. If necessary, the audio record or its transcripts may be passed on to the sponsor in an anonymous form, only for their internal use. You have the right to withhold information or withdraw from the interview at any time.
We are required to pass on to our client any details of suspected adverse reactions mentioned during the study. Although what you say will be treated in confidence, if you mention a suspected adverse reaction in a patient during the discussion, we will need to report this even if you have already reported this directly to the company or the regulatory authorities. In such a situation, you will be asked whether you are willing to waive the confidentiality given to you under the Market Research Codes of conduct specifically in relation to that suspected adverse reaction. You will still have the option to remain anonymous, if you so wish. Everything else you say during the interview will continue to remain confidential. In case of any clarifications, please contact _____
Page 2 of 10
My name is XXX. Thank you very much for agreeing to participate in this study conducted by SmartAnalyst Inc. and sponsored by a pharmaceutical company. The focus of todays discussion is to understand treatment dynamics related to two facets of hemophilia, specifically Acquired Hemophilia and Hemophilia B. The objective is to identify the unmet needs in both diseases as well as to understand how treatment decisions will be made given the evolving treatment landscape. In particular we will focus on some new products and the value that you perceive they may bring to these conditions. Given your experience in the field, we are very keen to know your opinion, so please do not hesitate to give us your thoughts freely. If you are ready, we can start the discussion.
PHYSICIAN BACKGROUND
Designation/position: Institutional affiliation:
2. What is the process for identification and diagnosis of a patient with Acquired Hemophilia? a. Where does the patient typically present? b. What steps are taken upon review of the symptoms? c. What lab tests are required to confirm diagnosis?
3. Who are the various health care professionals involved in the diagnosis of Acquired Hemophilia? a. When is a hematologist typically called in? Are there situations where a hematologist is not called? Please describe. b. What role does the hematologist play versus others in diagnosing the patient?
Page 3 of 10
c.
What role does the hematologist play versus others in making treatment decisions for the patient?
4. How often are patients with Acquired Hemophilia misdiagnosed? a. What are the primary reasons for misdiagnosis? b. How soon is the diagnosis corrected? c. What percentage of patients are untreated as a result of misdiagnosis?
d. What percent of patients that are initially misdiagnosed eventually are treated for Acquired Hemophilia?
5. What percent of patients presenting with Acquired Hemophilia require hemostatic treatment? a. What level of bleeding must be observed in order to initiate hemostatic treatment? b. c. What physician specialty typically initiates treatment? When is treatment usually initiated? (e.g., in ED, following hospital admission, following hematology consult)
d. What percent of patients with acquired hemophilia resolve spontaneously vs those that require hemostatic treatment?
6. What is the primary treatment approach for patients diagnosed with Acquired Hemophilia? a. What medications are considered first line for immunosuppression? b. What medications are used first line for controlling the bleeding? c. What medications are used second line for controlling the bleeding?
d. Overall, how satisfied are you with current treatments for Acquired Hemophilia? What, if any, do you consider to be significant unmet needs?
7. For what percentage of your Acquired Hemophilia patients requiring hemostatic treatment do you recommend NovoSeven? a. How do you determine if NovoSeven is appropriate per patient?
Page 4 of 10
8. What do you consider to be the benefits and risks of NovoSeven in this patient population? (Probe for efficacy, thromboembolic effects, inability to use standard assay)
9. Do you/have you ever prescribed a porcine FVIII product for a patient with Acquired Hemophilia? a. If yes, what product(s)? b. What is/was your experience with the porcine FVIII? (Probe for efficacy and safety) c. Did any patient have pre-existing antibodies to porcine FVIII? What percentage?
10. Do you/have you ever prescribed a porcine FVIII product for any condition other than Acquired Hemophilia? a. If yes, what product(s) and which condition(s)? b. How effective was/is the porcine FVIII in treating the condition? What advantages or disadvantages did you associate with this product? c. What are the characteristics of the patients where the product worked best?
11. Are you aware of any porcine FVIII products currently in development for Acquire Hemophilia? a. If yes, which one(s)? b. What have you heard thus far?
OBI-1 is a recombinant form of porcine FVIII in Phase 3 clinical development for the treatment of Acquired Hemophilia.). Since OBI-1 shows low cross reactivity to some anti-human FVIII antibodies, it is expected that OBI-1 may provide therapeutic benefits to some patients who are not able to use human FVIII.
12. What do you consider to be the possible benefits and risks of a recombinant porcine FVIII product like OBI-1 versus NovoSeven in Acquired Hemophilia patients?
Page 5 of 10
13. For what percentage of your Acquired Hemophilia patients requiring hemostatic treatment would you likely prescribe OBI-1 under the following scenarios? Please describe your decision-making criteria for each.
Definition Effective and partially effective outcomes in 7883% when used as first line treatment Effective and partially effective outcomes in greater than 78-83% when used as first line treatment
% of Patients at Peak
14. What would you expect in terms of pricing for a product like OBI-1 for Acquired Hemophilia? What role, if any, will price play in your prescribing decisions for OBI-1?
15. What percentage of Acquired Hemophilia patients relapse after initial treatment? a. Of those that relapse, what percent require retreatment with hemostatic agents? b. If retreatment is required, what medications are typically used? c. If patient was formerly treated with NovoSeven, do/would you use it again? Why or why not?
d. If the patient was formerly treated with OBI-1, do you anticipate you would use it again? Why or why not?
16. Do you believe that a recombinant porcine FVIII like OBI-1 may also provide a therapeutic benefit for Hemophilia A patients with inhibitors? Why or why not? a. What would be the characteristics of an inhibitor patient for which you would prescribe a recombinant porcine FVIII product? b. What are the benefits/risks of a recombinant porcine FVIII in this patient population versus other available therapies? c. Would you consider using the recombinant porcine FVIII in the acute setting only, or could you conceive of outpatient usage? Please elaborate.
17. Through our conversation thus far, have we touched upon what you consider to be the biggest unmet need in Acquired Hemophilia today? If not, please describe.
Page 6 of 10
18. What suggestions would you make to the manufacturer responsible for launching OBI-1 in order to address the opportunities and challenges in the current Acquired Hemophilia market?
Hemophilia B 19. How many patients with Hemophilia B do you treat in a typical year?
20. What percentage of your patients are on an on demand vs. prophylactic regimen? a. How does this percentage compare to your Hem A population? b. What are the primary factors influencing prophylaxis adoption in Hem B patients?
21. What percentage of your Hem B patients are on the following products? Product BeneFIX pdFVIII Other 22. What is the most common dosing regimen with BeneFIX for your prophylaxis patients? Why? Product 3x/week 2x/week 1x/week Other 23. How satisfied are you with Benefix for the treatment of Factor IX deficiency? (Please explain) % of Prophylaxis Patients % of On Demand Patients % of Prophylaxis Patients
24. BeneFIX is currently in clinical trials to demonstrate the benefits of a 1x/week dosing regimen. Assuming that the outcome of the trial is positive and labeling is secured, what do you anticipate the impact of this new information to have on BeneFIX prescribing?
25. Are you aware of any new products currently in clinical development for the treatment of Hem B? If yes, please briefly describe your impressions/knowledge of each product.
Page 7 of 10
Product rFIX-Fc (Biogen) N9-gp (Novo) rFIX-FP (CSL) Bax-326 (Baxter) IB-1001 (Inspiration) AMT-060 (UniQure) Baxter/Chatham
Category Long-acting Long-acting Long-acting Short-acting/ Biosimilar Short-acting/ Biosimilar Gene therapy Gene therapy
Mentioned (Unaided)
Comments
26. Where do you anticipate new compounds will gain acceptance for the treatment of Factor IX deficiency? a. b. c. Newly diagnosed Patients Only Patients Switching from BenefIX Only Both newly diagnosed and switch patients
27. What do you anticipate will be the impact of the entry of the long-acting agents on the Hem B category? a. What percentage of your prophy patients will switch to a long-acting product? b. What percentage of your on demand patients will switch to a prophy regimen with a long-acting product? c. Which long-acting product is most likely to capture the greatest market share? Why?
28. What do you anticipate will be the impact of the entry of new short-acting/biosimilar agents on the Hem B category? a. What percentage of your overall patient population will not convert to long-acting products? Why is that? b. Of this patient population, what percent would you anticipate to be on demand vs prophy patients?
Bax-326 and IB-1001 are both short acting intravenous recombinant factor IX (rFIX) protein being investigated for the treatment and prophylaxis of bleeding episodes for patients over 12 years of age with hemophilia B.
Page 8 of 10
29. What do you believe to be the differentiating factors of Bax-326 vs BeneFIX? What positive or negative aspects of Bax 326 have you heard?
30. What do you believe to be the differentiating factors of IB-1001 vs BeneFIX? What positive or negative aspects of IB-1001 have you heard? If issue of Clinical Hold and antibody development in US is mentioned, probe to understand potential impact on product value proposition and prescribing.
31. Please describe a patient profile in which you would considering using Bax-326 or IB-1001? Please explain.
32. What will be the primary drivers of market share capture for a new biosimilar product like Bax-326 or IB-1001?
33. For the following scenarios, please assume that BeneFIX, Bax-326 and IB-1001 have equivalent profiles in terms of efficacy and safety. What percentage of your non-long acting patients (____% as stated in 28a above) would you prescribe per product at peak? (3 values should equal 100%)
Scenario Bax-326 launches 6-12 months ahead of IB-1001 and both are priced equally at discount to BeneFIX Bax-326 launches 6-12 months ahead of IB-1001 but IB-1001 is priced at a deeper discount than Bax-326
% BeneFIX
% Bax326
% IBI-1001
Rationale
34. What suggestions would you make to the manufacturer responsible for launching IB-1001 in order to address the opportunities and challenges in the current Hemophilia B market?
Page 9 of 10
Page 10 of 10