Hospital Pharmacist
Vol 10 No 1 p8
January 2003

Letters (Controversy surrounding high-cost drug use in oncology)

From Mr de Lemos

Jones et al provided a timely review (PDF* 75K) on the dilemma faced by the oncology community in the era of more and more costly drugs (Hosp Pharm, October, 2002, pp275-77).

I would like to comment on two of their recommendations. First, the authors suggested that once a drug has been licensed, it should be freely accessible to prescribers. This probably stems from a misinterpretation of the distinctive roles of regulatory review and funding policy.

Regulatory approval to market a drug only requires demonstrated safety and efficacy, while funding policy generally needs to know if that treatment is medically reasonable or necessary.^1.2

The relative therapeutic merit of new oncology drugs is not always easy to assess,³ but there are several scenarios when a drug may be safe and efficacious but still deemed not to be medically necessary. For example, the effectiveness of one drug compared with currently available therapies may be relatively small, or the drug's price may be much higher than the comparator drug, but only marginally more effective.⁴ The more difficult situation in oncology is that surrogate endpoints of potential, but unvalidated, clinical significance are used in lieu of survival data. This difficulty is compounded by the fact that many new oncology drug applications are based on phase II, non-comparator trials.⁵ It would be more helpful if the great urgency of introducing new drugs is matched by the same urgency to validate the commonly used surrogate endpoints.⁶

Secondly, the authors also suggested that when new drugs are licensed, national guidelines, like those from the National Institute for Clinical Excellence, should be issued concurrently. This is not possible for the simple reason that the timing of when a new drug would be approved is commercially confidential information, as any approval would lead to an increase in the value of the manufacturer's stock. A better way to solve this problem is to demand that all new drug applications include information needed for decision-making by both the regulatory and the funding review bodies.

Perhaps it is time that concerted efforts are made to ensure that drugs are not only tested for their efficacy, but also for their effectiveness in the real world.

Mário de Lemos
Provincial Drug Information Co-ordinator
Systemic Therapy Program
British Columbia Cancer Agency
Vancouver
Canada

  * PDF files on PJ Online require Acrobat Reader 4 or later.