This forecast attempts to quantify the impact of gene therapy and other non-factor treatments will have on the US hemophilia market over the next 10 years. It is based on in-depth interviews with hemophilia treatment centers and other hemophilia stakeholders along with a survey of over 200 US-based hemophilia patients. The report includes:

  • Uptake of gene therapy in hemophilia A and B across 8 different patient groups from market introduction in 2021 through 2028, including timing, patient popuations and interest level across the various groups
  • Forecast of coagulation factor VIII and IX as well as novel non-factor treatments from 2018 to 2028, in dollars and units (factor VIII and IX) or patients (non-factor treatments),
  • Overview of the development of gene therapy for hemophilia A and B, which companies are likely to commercialize their product and the expected timing of launch
  • Background on the technologies used by various gene therapy companies and how they differ to one another as applied to hemophilia
  • Snapshot at patient and Treatment Centers’ expectations for gene therapy in hemophilia in early 2018

 

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EXECUTIVE SUMMARY

Hemophilia A Forecast

Hemophilia B Forecast

1) INTRODUCTION

2) BACKGROUND ON GENE THERAPY TREATMENT IN HEMOPHILIA

2.1) History of Gene Therapy in Hemophilia

2.1.1) Recent Clinical Trial Results in Hemophilia B

2.1.2) Recent Clinical Trial Results in Hemophilia A

2.2) Gene Therapy Variations Under Investigation Today

2.2.1) AAV-based Viral Vectors

2.2.2) Lentivirus

2.2.3) CRISPER-Cas9

2.3) Issues with Current Gene Therapy Trials and Future Possibilities

2.3.1) Pre-existing antibodies (PEA) to AAV or lentivirus vectors

2.3.2) Inhibitor to factor VIII or IX

2.3.3) Weight and Health Issues

2.3.4) Liver Function Considerations

2.3.5) Manufacturing Challenges

3) CURRENT VIEWS AND PERCEPTIONS OF GENE THERAPY TODAY

3.1) Results from HTC interviews

3.1.1) Expectations in 2021 – Beginning of Gene Therapy Commercialization23

3.1.2) Expectations in 2024 – Midway into Gene Therapy Transition

3.1.3) Expectations in 2026 – Expectations for Well Established Gene Therapy Treatment

3.2) Results from Patient Survey

3.2.1) Demographics of Hemophilia Patients and Caregivers

3.2.2) Current Product Usage

3.2.3) Community Involvement

3.2.4) Attitudes Towards New Products

3.2.5) Thoughts on Attitudes, Benefits and Risks of Gene Therapy

3.2.6) Interest in Gene Therapy Today

3.2.7) Patient Expectations in 2021 – Beginning of Gene Therapy Commercialization

3.2.8) Patient Expectations in 2024 – Midway into Gene Therapy Transition

3.2.9) Patient Expectations in 2026 – Expectations for Well Established Gene Therapy

4) ASSUMPTIONS ABOUT GENE THERAPY UPTAKE AND PRICING 

4.1) Timing Expectations for Gene Therapy Product Approval

4.2) Forecast Patient Numbers and Percentages

4.2.1) Adult Severe Patients Without Factor Inhibitors or Pre-Existing Antibodies – First Patient Subgroup to be Eligible for Gene Therapy

4.2.2) Teen Patients Age 12-17 who are Severe

4.2.3) Moderate Severity Patients Age 12 years old and Older

4.2.4) Patients with Inhibitors to Factor Proteins and Pre-existing Antibodies to Vectors

4.2.5) Patients Age 7-11 who are Moderate or Severe

4.2.6) Mild Patients who are on Prophylaxis

4.2.7) Infants and Children Age 0-6 years old

4.2.8) Repeat Dosing of Patients After Failure

4.3) Thoughts on Payment Models for Gene Therapy

4.3.1) Private Insurance Reimbursement

4.3.2) Government Reimbursement

4.3.3) First AAV Gene Therapy Product Pricing and Reimbursement

4.4) Pricing and Payment Assumptions for Hemophilia Gene Therapy

5) HEMOPHILIA A GENE THERAPY FORECAST TO 2028

5.1) Topline Analysis of Gene Therapy Forecast

5.2) Patient Uptake by Different Subgroups

6) HEMOPHILIA A MARKET FORECAST FOR ALL PRODUCTS

6.1) Effect of non-Factor Products on the Hemophilia A market

6.2) Factor VIII Products Market from 2018 to 2028

6.2.1) Who will be using factor VIII products after 2028?

7) HEMOPHILIA B GENE THERAPY FORECAST TO 2028

7.1) Topline Analysis of Gene Therapy Forecast

7.2) Patient Uptake by Different Subgroups

8) HEMOPHILIA B MARKET FORECAST FOR ALL PRODUCTS

8.1) Effect of non-Factor Products on the Hemophilia B market

8.2) Factor IX Products Market from 2018 to 2028

8.2.1) Who will be using factor IX products after 2028?

9) METHODOLOGY

10) ANNEX – PATIENT QUESTIONNAIRE RESULTS SUMMARY