Hereditary Angioedema: treatment Awareness

A Survey of Fifty Allergists

November 2007

STUDY DESCRIPTION

OBJECTIVES

Evaluate treatments in development for hereditary angioedema (HAE)

• Determine current prevalence of HAE patients and HAE attacks in the United States
• Identify physician awareness of treatments in development for HAE
• Examine the factors that drive allergists to prescribe medications for HAE
• Evaluate each treatment on a number of factors including:

• Onset of action
• Duration of effect
• Ease of administration
• Side effect profile

• Gain insight into the scenarios in which physicians would use each HAE treatment
• Anticipate the impact of reimbursement on uptake of the treatments

Companies and Products Mentioned in This Report 

Key Findings

• Responding allergists have seen a median of 3 HAE patients over past year. The average patient is 23 years of age and experiences three HAE attacks per year.
• DX-88 Ranked Lowest in Awareness. The majority of the allergists indicate awareness of HAE is higher now than 5 years ago. Of the treatments in developments for HAE, the physicians are most aware of rC1-INH (Rhucin) and least aware of DX-88. However, overall awareness of the treatments in development is mediocre.
• Onset of action most important attribute for HAE treatments.  Duration of effect was ranked as the second most important attribute. C1-INH (Cinryze) and C1-INH (freeze dried, Berinert-P) received the highest and second highest ratings respectively for their efficacy, duration of effect, onset of action, and side-effect profiles. Icatibant had the highest mean rating for ease of administration.
• Rhucin rated as having the most superior product profile. 40% of the respondents indicate they believe Rhucin has the most superior profile. The rest of the respondents were evenly divided between the other four treatments. Respondents reported that Rhucin is most suited for laryngeal attacks, Berinert-P is most suited for abdominal attacks and DX-88 is most suited for cutaneous attacks.
• Icatibant and Rhucin used most frequently in initial treatment. After viewing the treatment profiles, respondents suggest they would most frequently use Icatibant and Rhucin for the most severe cases of HAE. Selection of DX-88 for the most severe cases of HAE jumped from 10% to 26% following display of the product profiles. The discrepancy between responses prior to and following the profiles supports the data suggesting that awareness of DX-88 is very low.
• Discrepancy between treatment ratings and anticipated use. Although the respondents gave the highest attribute ratings to Cinryze and Berinert-P, they anticipate using Rhucin most frequently and indicated it has the most superior profile. This discrepancy may be due to two factors. First, the respondents indicated they are most familiar with Rhucin which could bias them toward using it more frequently. Second, Rhucin was selected most frequently as having the superior profile after respondents were presented with the product profiles for each treatment, whereas the attribute ratings were made prior to the profiles being displayed.
• Physicians expected reimbursement to be a challenge, but the least important prescribing factor. 44% believe reimbursement will be a substantial challenge and 52% anticipate it will be somewhat of a challenge for patients if the treatments become available in the US. The respondents indicated price has almost no bearing on their HAE treatment choice.

Inclusion Criteria and Respondent Demographics

quantitative survey detailed findings by question

SECTION ONE: Hereditary angioedema (hae) patient population and awareness

Question 1             

How many individual HAE patients have you seen in the past 12 months?
Please do not include multiple visits by a single patient.

HAE Patients Seen in Past 12 Months (n = 49*):

Note: One respondent indicated they have seen fewer patients than they treated over the last 12 months, so their response was deleted

Question 2

Please estimate the average age at which your HAE patients are diagnosed with the disease.

Average Age of HAE Diagnosis (n = 50):

Question 3

How many attacks does your average HAE patient experience per year?

Average HAE Attacks per Year (n = 50):

Question 4

In your opinion, what is the minimum number of HAE attacks that would constitute a case of severe HAE? Please select one.

Question 5

How does the level of awareness of HAE now compare to 5 years ago? 

Question 6

Have you enrolled patients in a clinical trial for HAE?

Question 7

Please indicate the HAE treatments for which you enrolled patients in a clinical trial. Please select all that apply.

Other HAE treatment trials mentioned: “Can’t recall… it was in 2000 and 2001”

*Only respondents who answered yes in question 6 responded to this question

SECTION two: hae treatment evaluation

Question 8

On a scale from 1 to 5, please indicate your level of awareness of each of the following treatments in development for HAE. 1=Not at all Aware and 5=Very Aware

Ratings of Awareness for Each Treatment (n = 50)

Question 9

Please rank the following attributes of a HAE drug from 1 to 5 in terms of its degree of importance in choosing a particular treatment. 1=Most Important Attribute, 2=Second Most Important Attribute, and so on.

Attribute Importance Rankings (n = 50)

Question 10

Given what you know of the profiles of these products in development, which would you use initially in non-severe HAE patients?

Question 11

Which of these drugs would you choose as a treatment for the most severe cases of HAE?

Question 12

Would you use any of these treatments prophylactically for HAE? Please select all that apply.

Question 13

Using a scale from 1 to 7, please rate your impression of Icatibant on each of the given attributes. 1=Very Poor Impression, 4=Neutral Impression, and 7=Very Positive Impression

Please select NA if you feel the attribute is not applicable or if your knowledge of Icatibant is too limited to respond.

Icatibant Impression Ratings (n = 41*)

*Only respondents who indicated they are familiar with Icatibant in Q8 answered this question

Question 14

Using a scale from 1 to 7, please rate your impression of DX-88 on each of the given attributes. 1=Very Poor Impression, 4=Neutral Impression, and 7=Very Positive Impression

Please select NA if you feel the attribute is not applicable or if your knowledge of DX-88 is too limited to respond.

DX-88 Impression Ratings (n = 34*)

*Only respondents who indicated they are familiar with DX-88 in Q8 answered this question

Question 15

Using a scale from 1 to 7, please rate your impression of rC1-INH (Rhucin) on each of the given attributes. 1=Very Poor Impression, 4=Neutral Impression, and 7=Very Positive Impression

Please select NA if you feel the attribute is not applicable or if your knowledge of rC1-INH (Rhucin) is too limited to respond.

rC1-INH (Rhucin) Impression Ratings (n = 46*)

*Only respondents who indicated they are familiar with rC1-INH (Rhucin) in Q8 answered this question

Question 16

Using a scale from 1 to 7, please rate your impression of C1-INH (Cinryze) on each of the given attributes. 1=Very Poor Impression, 4=Neutral Impression, and 7=Very Positive Impression

Please select NA if you feel the attribute is not applicable or if your knowledge of C1-INH (Cinryze) is too limited to respond.

C1-INH (Cinryze) Impression Ratings (n = 42*)

*Only respondents who indicated they are familiar with C1-INH (Cinryze) in Q8 answered this question

Question 17

Using a scale from 1 to 7, please rate your impression of C1-INH (freeze dried, Berinert-P) on each of the given attributes. 1=Very Poor Impression, 4=Neutral Impression, and 7=Very Positive Impression

Please select NA if you feel the attribute is not applicable or if your knowledge of C1-INH (freeze dried, Berinert-P) is too limited to respond.

C1-INH (freeze dried, Berinert-P) Impression Ratings (n = 42*)

*Only respondents who indicated they are familiar with C1-INH (freeze dried, Berinert-P) in Q8 answered this question

SECTION three: hae treatment profiles

Question 18

Based on the information above, which treatment would you use initially in non-severe HAE patients?

Question 19

Based on the information above, which of these drugs would you choose as a treatment for the most severe cases of HAE?

Question 20

Based on the information above, would you use any of these treatments prophylactically for HAE? Please select all that apply.

Question 21

Are any of the treatments listed below particularly suited to specific types of HAE attacks? Please select all of the types of attacks that apply for each treatment

Question 22

Overall, which product do you believe has the most superior profile?

Question 23

Do you anticipate reimbursement will be a challenge for patients if these therapies become available in the US?