Report on New Patented Drugs - Macugen

Under its transparency initiative, the PMPRB publishes the results of the reviews of new patented drugs by Board Staff, for purposes of applying the PMPRB's Excessive Price Guidelines (Guidelines), for all new active substances introduced after January 1, 2002.

Brand Name: Macugen

Generic Name: (pegaptanib sodium injection)

DIN: 02267225 (0.3 mg/90 ?L syringe)

Patentee: Pfizer Canada Inc.

Indication - as per product monograph:
For the treatment of subfoveal choroidal neovascularization (CNV) secondary to age-related macular degeneration.

Date of Issuance of First Patent(s) Pertaining to the Medicine: February 14, 2006

Notice of Compliance: May 2, 2005

Date of First Sale: August 26, 2005

ATC Class: S01XA17
Sensory Organs; Ophthalmologicals; Other Ophthalmologicals

Application of the Guidelines

Summary

The introductory price of Macugen was found to be within the PMPRB's Guidelines because the price in Canada did not exceed the median of the prices of the same drug product in those countries listed in the Patented Medicines Regulations, 1994 (Regulations) in which it was sold.

Scientific Review

Macugen is a new active substance and the PMPRB's Human Drug Advisory Panel (HDAP) recommended that it be classified as a category 2 new medicine as it provides a substantial improvement in the treatment of subfoveal choroidal neovascularization (CNV) secondary to age-related macular degeneration, where current standard of care provides insufficient therapy options for patients who have minimally classic or occult lesions with no classic morphology. This recommendation is based on two randomized phase III clinical trials (reference #7), in which patients treated with Macugen exhibited favourable results in all subtypes (classic or occult) of wet (CNV).

Loss of visual acuity is a major cause of incapacity and considerably reduces quality of life. The possibility to stop the progression of the disease while treating a greater spectrum of patients with Macugen, combined with the relative ease of administration and medically manageable side effects, remains very relevant in this patient population.

The HDAP did not identify any comparators for the conduct of a Therapeutic Class Comparison (TCC) test for Macugen as there are no comparators that can be considered clinically equivalent.

Price Review

Under the Guidelines, the introductory price of a new category 2 drug product will be presumed to be excessive if it exceeds the prices of all of the comparable drug products based on a TCC test, or the median of the international prices identified in an International Price Comparison (IPC) test. As no comparable drug products could be identified for purposes of conducting a TCC test, the introductory price of Macugen was considered within the Guidelines as it did not exceed the median of the international prices identified in the IPC test. Macugen was sold in only one other country (United States) of the seven countries listed in the Regulations at the time of introduction to the Canadian market.

Introduction Period (August to December 2005)

Source: 1. No publicly available price at introduction (2005) or in 2006. PPS Pharma January 2007.
2. Publicly available price as per the Regulations.

The Guidelines provide that when a medicine is sold in fewer than five countries at the time of its introduction, the introductory price will be treated as the interim benchmark price. Board Staff will review at the end of three years or when the medicine is sold in at least five countries, whichever comes first.

Where comparators and dosage regimens are referred to in the Summary Reports, they have been selected by the PMPRB Staff and the HDAP for the purpose of carrying out the PMPRB's regulatory mandate, which is to review the prices of patented medicines sold in Canada to ensure that such prices are not excessive. The publication of these reports is also part of the PMPRB's commitment to make its price review process more transparent.

The information contained in the PMPRB's Summary Reports should not be relied upon for any purpose other than its stated purpose and is not to be interpreted as an endorsement, recommendation or approval of any drug nor is it intended to be relied upon as a substitute for seeking appropriate advice from a qualified health care practitioner.

References - Macugen

1. Age -Related Macular Degeneration: American Academy of Ophthalmology. http://www.aao.org/aao/education/library/ppp/upload/Age-Related-Macular-Degeneration.pdf, accessed September 22, 2005.

2. Amoaku WM. Pegaptanib in exudative age-related macular degeneration. Drugs 2005:65(11):1579.

3. Appraisal consultation document: Photodynamic therapy for age-related macular degeneration. NICE. Available at: www.nice.org.uk/page.aspx?o=30199.

4. Asif M, Siddiqui A, Keating GM. Pegaptanib: In exudative age-related macular degeneration. Drugs 2005;65(11):1571-7.

5. Bressler NM. Treatment of Age-Related Macular Degeneration with Photodynamic Therapy (TAP) Study Group. Photodynamic therapy of subfoveal choroidal neovascularization in age-related macular degeneration with verteporfin: two year results of 2 randomized clinical trials- TAP report 2. Arch Ophthalmol 2001;119:198-207.

6. Ferris FL. A new treatment of ocular neovascularization.
N Engl J Med 2004;351(27):2863-4.

7. Gragoudas ES, Adamis AP, Cunningham ET, et al. Pegaptanib for neovascular age-related macular degeneration. N Engl J Med 2004;351(27):2805-16.

8. Health Canada, Notice of Decision for Macugen, June 2005.

9. Larouche K and Rochon S. Evaluation of photodynamic therapy for the treatment of exudative age-related macular degeneration (AMD) with subfoveal neovascularization. A technology assessment prepared for the Agence d'évaluation des technologies et des modes d'intervention en sante. Montreal: AETMIS, 2004,xiv-101 p.

10. Moshfegi A and Puliafito C. Pegaptanib sodium for the treatment of neovascular age-related macular degeneration.
Expert Opin. Investig. Drugs (2005)14(5):671-682.

11. Ng EW, Adamis A. Targeting angiogenesis, the underlying disorder in neovascular age-related macular degeneration.
Can J Ophthalmol 2005;40:352-68.

12. Pauleikhoff D, Bornfeld N, Gabel VP, Holz F, Roider H. The position of the Retinological Society, the German Opthalmological Society and the Professional Association of Ophthalmologists - Comments on the current therapy of neovascular AMD. Klin Monatsbl Augenheilkd 2005;222:381-8.

13. Product monograph of Macugen (pegaptanib sodium injection).
Pfizer Canada Inc., Kirkland, QC, April 25, 2005.

14. Rakic JM, Blaise P, Foidart JM. Pegaptanib and age-related macular degeneration. N Engl J Med 2005;352(16):1720-1.

15. Schachat A. New treatments for age-related macular degeneration. Ophthalmology, April 2005;112(4):531-2.

16. Schwartz S. Pegaptanib in exudative age-related macular degeneration. Drugs 2005;65(11):1578-9.

17. Verteporfin therapy of subfoveal choroidal neovascularization in age-related macular degeneration: Two-year results of a randomized clinical trial including lesions with occult with no classic choroidal neovascularization-verteporfin in photodynamic therapy report 2.
Am J Ophthalmol 2001;131:541-560.

18. Visudyne Product Monograph: CPS 2005 Electronic version.