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

An expensive drug for the treatment of 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 5,000 Australians. Unfortunately MM carries one of the lowest long-term survival rates of all cancers. It follows a typically relapsing course and only 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 new generation anti-neoplastic agent which belongs to the class of medications called proteosome inhibitors.⁴ In recent years, 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.¹ Velcade is now approved for treatment of progressive forms of multiple myeloma in patients who have received at least one prior therapy.² Velcade therefore offers potential clinical benefits to patients who have failed alternative therapies. Until recently, treatment with Velcade would put patients out of pocket approximately $20,000 for each dose and up to $60,000 for a full six-month treatment. However, the government has recently agreed to make this drug available at a subsidised rate through the Pharmaceutical Benefits Scheme (item numbers 9117W and 9118X).³ This decision has been welcomed by the Myeloma Foundation of Australia, health professionals and patients alike. Patients can now reap the benfitis 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). 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.³ Health practitioners are encouraged to refer to the PBS for full authority details. This new PBS listing allows an expensive anti-neoplastic medication to become available to the general public at an affordable price, thus overcoming issues of inequity of access and past ethical dilemmas regarding informing patients about unaffordable therapies. 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. 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
2. MIMS online- Prescribing Information, Velcade (Bortezomib), MIMS Australia Pty Ltd 2003.
3. Schedule of Pharmaceutical Benefits [online], Australian Government, Department of Health and Aging, 2007. Available from URL: http://pbs.gov.au/html/home
4. Virtual Medical Centre, Velcade, [online] 2007 [cited 125th November 2007]. Available from URL: /drugs.asp?drugid=3181