Seminar 3 – New drugs – how much are they worth?

In cancer therapy, we assisted to a gradual shift from cytotoxic drugs to more or less selective, high cost targeted therapeutic agents. These new agents frequently achieve only marginal benefits, and this matter is under increasing scrutiny. Thus, important questions were raised regarding allocation decisions and value issues in the reimbursement of cancer drugs by insurance companies or health care systems, depending on local organization. Moreover, based on research into the biology of human tumors, we now appreciate that cancers are very heterogeneous not only in terms of morphology, histology, and clinical outcome but also at the molecular level. Among a given type of cancer there is great genetic variation with a variety of high- and low-frequency mutations, including mutations that are responsible for driving the initiation, progression, or maintenance of the tumor. This makes suboptimal the ‘one-size-fits all’ approach or a single drug regimen for patients with the same tumor type or histology, and, necessarily, will change the therapeutic approach by clinicians. Based on these premises, and due to economic constraints of health care systems, and, at the same time, in several cases, an increased uncertainty on the cost benefit profile of new technologies at the time of marketing (e.g., poor efficacy and safety data), several countries are experimenting risk sharing mechanisms. Through these mechanisms, in case of patient negative outcome, the pharmaceutical company reimburses part of the cost of the drug to the hospital/ local trust. On the other hand, in case of clinical success, the hospital covers the overall cost.

In this scenario, questions arise at the local level on whether these mechanisms represent the best choice (e.g., are real "cost saving" mechanisms), or whether an a priori more restricted selection would constitute a better alternative for the overall system. Outcome research plays a pivotal role in answering to those questions which were left unanswered at the time of registration.

The hospital pharmacist plays a key role for the success of the overall process, since he/she is directly involved in the evaluation, dispensation and outcome monitoring of new technologies.

Teaching goals:

• discuss issues on lack/scarcity of efficacy/ effectiveness and safety data at the time of drug registration
• discuss new methodologies in clinical trials in cancer therapy (e.g., appropriate patient selection, novel clinical trial designs and biomarker-driven trials).
• present different methodologies of risk sharing between industry and payers for new drugs (medical devices)
• discuss the importance of linking these mechanisms with outcome research (e.g., the example of Italy)
• present the potential involvement of hospital pharmacists in the management and evaluation of these systems

Learning objectives:

After the presentation the participant should:

• become familiar with new approaches for cancer treatments
• become familiar with major pitfalls in evaluating new technologies
• become familiar with risk sharing mechanisms in different countries
• be able to evaluate the impact of these mechanisms on drug use in their context
• be prepared to apply and evaluate in the real world these mechanism in their setting