New Hope for Patients with Myeloma as Velcade is Added to the PBS

A novel therapy for advanced and progressive stages of multiple myeloma has become available on the Australian Pharmaceutical Benefits Scheme from November 1st 2007. This is fantastic news for sufferers of this rare and debilitating haematological cancer, whom normally face a poor prognosis without treatment.

Multiple myeloma (MM) is a rare malignancy affecting plasma cells within the bone marrow. Destruction of plasma cells inhibits their ability to produce antibodies and function properly within the body’s immune system. This leads to excessive production of abnormal plasma cells and intact monoclonal immunoglobulins or Bence-Jones proteins. Multiple myeloma causes several clinical consequences including bone pain, hypercalcaemia, renal failure, infection and anaemia. Although uncommon overall, myeloma remains the second most common haematological malignancy following non-Hodgkin’s lymphoma, and affects over 5000 Australians. It accounts for approximately 1% of all cancers and 2% of all cancer deaths. Unfortunately MM carries one of the lowest long-term survival rates of all cancers. It follows a typically relapsing course and less than one third of patients survive the cancer longer than five years.However, recent advances in treatment have helped decrease the occurrence and progression of disease. In particular, combination therapies involving Velcade have shown promising results above those of single agents. Velcade (Bortezomib) is a novel anti-neoplastic agent which acts by inhibiting a new target, the proteosome within cells.^3,6 After demonstrating promise in clinical trials, Velcade underwent an accelerated approval program to become available to Australians, following collaboration between the National Cancer Institute, the Food and Drug Administration (FDA) and the pharmaceutical company.² Initially the drug treatment was expensive but hundreds of patients have since received treatment free of charge through the Janssen-Cilag Compassionate Use Program, Expanded Access Program and a heavily subsidised Assistance Program.Now Velcade is approved for treatment of progressive forms of multiple myeloma in patients who have received at least one prior therapy.² Follow-up of a recent phase III randomised multi-centre clinical trial further confirmed the benefits of this treatment in patients with relapsed or refractory myeloma.^1,4 Compared to high-dose dexamethasone (a traditional myeloma therapy), Velcade proved statistically superior in terms of time to progression, response rates, time to response, duration of response and overall survival.¹ Velcade therefore offers hope and clinical benefits to patients who may have failed previous therapies. The announcement of Velcade’s listing on the Pharmaceutical Benefits Scheme (item numbers 9117W and 9118X) is great news.⁵ A medication worth up to $7,000 per cycle and up to $42,000 for an average treatment cost (based on the average number of cycles administered in the large phase III study) is now available to consumers at a subsidised price.⁴ Patients can reap the benefits of prolonged survival (one year extended life expectancy) for only a minimal cost of $30.70 per month (and down to $5 for Health Care Card holders).⁵ This decision has been welcomed by the Myeloma Foundation of Australia, health professionals and patients alike. BORTEZOMIB – Powder for injection 3.5 mg is accessible via an authority script as “initial monotherapy or in combination with a corticosteroid, for the treatment of multiple myeloma in a patient with a WHO performance status of 2 or less, who has progressive disease, who has received at least 1 prior therapy (other than thalidomide), who has undergone or is ineligible for a primary stem cell transplant and who has experienced treatment failure after a trial of at least four (4) weeks of thalidomide at a dose of at least 100 mg daily.” Continued authority scripts are dependent on the demonstration of clinical response to bortezomib treatment.⁵ Health practitioners are encouraged to refer to the PBS for full authority details. This new PBS listing allows a novel and efficacious anti-neoplastic medication to become available to the general public at an affordable price. Furthermore it ensures equitable access to a novel treatment to ensure the clinical needs of myeloma patients are met. Access to affordable therapies is vital to the improvement of quality of life for myeloma sufferers and their families. In addition it offers patients enhanced survival time and added hope for the future. References

1. Corso Alessandro, An update of the APEX study, Haematological Reports 2006; 2(5): 2. Available [online] at URL: http://www.haematologicareports.org/2006/5/2.pdf
2. FDA Approves Velcade for Multiple Myeloma Treatment, [online] 2003 [cited 12th November 2007]. Available from URL: http://www.fda.gov/bbs/topics/NEWS/2003/NEW00905.html
3. MIMS online – Prescribing Information, Velcade (Bortezomib), MIMS Australia Pty Ltd 2003.
4. Richardson PG, Sonneveld P, Schuster MW, et al. Bortezomib or high-dose dexamethasone for relapsed multiple myeloma. N Engl J Med 2005; 352: 2487-98.
5. Schedule of Pharmaceutical Benefits [online], Australian Government, Department of Health and Aging, 2007. Available from URL: http://pbs.gov.au/html/home
6. Virtual Medical Centre, Velcade, [online] 2007 [cited 125th November 2007]. Available from URL: /drugs.asp?drugid=3181