1. Specialty pharmacy Market overview
By: John Sugrue

2. What is a specialty drug?
May have special handling and/or storage requirements
Usually not oral medications
Could need healthcare provider administration
May require regular monitoring (electrolytes, drug levels, etc…)
Often requires a prior authorization through patient’s insurance

3. Common Specialty Conditions
Cancer
Anemia
Multiple Sclerosis
Hepatitis
Cystic Fibrosis
Psoriasis
Rheumatoid Arthritis
Hemophilia

4. Orphan Drug Act
Orphan Drug – meets the needs of a disease in which less than 200,000 people are afflicted.
Provides incentives for drug companies to develop drugs for rare conditions
Less than 10 specialty drugs were developed between and 1983
Since then, over 400 specialty drugs have been FDA approved

6. Top 3
56%
Top 4
65%
Diplomat 2%

8. Top 3
56%
Top 4
59%
Diplomat 3%

10. Top 3
57%
Top 4
64%
Diplomat 3%

11. Growth
Since 2010, specialty pharmacy estimated revenue has grown from $39.2 billion to $98.3 billion (↑ 251%)
Diplomat has grown from $600 million to $3.4 billion (↑ 567%) in the same time frame
Report from IMS Health – within the past 10 years, spending has gone from $70 to $425 billion.

13. Q2 results for Diplomat Released on 8/9/16
Total revenue was $1.09 billion (35% increase from last year’s 2nd quarter)
3% increase in prescriptions dispensed
CEO Phil Hagerman states that this growth is more than it appears.
Moved away from high volume, low revenue compounding at the end of last year.

14. New FDA Drug Approvals - 2015
Most novel new drugs approved since 1992
45 new drugs and 9 biologics
16 of these were considered to be in a new class
Of these new approvals, nearly half are considered specialty drugs.
25 for rare diseases
13 oncology drugs
4 hemophilia drugs
1 for von Willebrand Disease

15. Hemophilia Drug Approvals - 2015
4/29/15 – Ixinity (Recombinant Factor IX) – Indicated for Hemophilia B
Other brand names already available: BenefIX, Alprolix, Rixubis
9/15/15 – Nuwiq (simoctocog alfa) – Indicated for Hemophilia A
First derived from a human cell line
Other brand names: Advate, Recombinate, Xyntha etc...

16. Hemophilia Drug Approvals - 2015
10/20/15 – Coagadex (Coagulation factor X) – Indicated for factor X deficiency
First and only product indicated for this condition.
11/13/15 – Adynovate (PEGylated Factor VIII) – Indicated for Hemophilia A
Attachment to polyethylene glycol extends the half-life (requires less frequent dosing)
12/8/15 – Vonvendi (recombinant von Willebrand factor)

17. Hemophilia Drug Approvals - 2016
3/4/16 – Idelvion (recombinant factor IX linked with albumin fusion protein)
Longer acting factor IX
3/16/16 – Kovaltry (octocog alfa) – Indicated for Hemophilia A
Another option for factor VIII replacement
5/27/16 – Afstyla (recombinant factor VIII single chain)
Longer acting factor VIII

18. ACE910 (emicizumab) Recombinant humanized bispecific antibody
Binds to activated factor IX and X and mimics the action of factor VIII
Subcutaneous drug that has a half life of 4-5 weeks
Pharmacokinetic simulation suggested during weekly prophylaxis, peak and trough levels would only differ by ~5%.
Out of the 64 patients in dose-escalation study, 2 were positive for anti-ACE910 antibodies
Uchida N, Sambe T, Yoneyama K, et al. Blood Mar 31; 127(13): 1633–1641.

19. ACE910 (emicizumab)
Results from a study in patients with severe hemophilia A:
18 Japanese patients with severe hemophilia A enrolled
All received once weekly SQ injection of ACE910 (12 weeks)
13/18 patients had no bleeding events
3 of whom had frequent bleeds before trial
Less factor replacement was needed to cure a bleeding event
No antibodies were developed
Shima M, Hanabusa H, Taki M, et al. N Engl J Med 2016 May 26; 374:

20. Questions?

21. References
Developing Products for Rare Diseases & Conditions [Internet]. Silver Spring, MD [2016 Sep 13]. Available from:
Fein A. Pharmacy Market Share for Specialty Drugs, 2010 [Internet]. Pembroke Consulting [2016 Sep 13]. Available from:
Fein A. The Top 10 Specialty Pharmacies of 2014 [Internet]. Pembroke Consulting [2016 Sep 13]. Available from:
Fein A. The Top 10 Specialty Pharmacies of 2015 [Internet]. Pembroke Consulting [2016 Sep 13]. Available from:
Bringing the Specialty Pharmacy Market into the Billions [Internet]. WRB Communications [2016 Sep 13]. Available from:
Fein A. Our 2020 Outlook for Specialty Pharmacy Revenues [Internet]. Pembroke Consulting [2016 Sep 13]. Available from:
Diplomat reports Q2 results, including 35% total revenue gain [Internet]. Drug Store News [2016 Sep 13]. Available from:
Diplomat Publishes New Report on Specialty Drugs Approved in 2015 and Expected for 2016 [Internet]. Flint, MI. PRNewswire [2016 Sep 13]. Available from: html.
Uchida N, Sambe T, Yoneyama K, et al. A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects. Blood Mar 31; 127(13): 1633–1641.
Shima M, Hanabusa H, Taki M, et al. Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med 2016 May 26; 374: