Finding the true value of cancer drugs

In a world where prices for cancer drugs are skyrocketing, and affordability is problematic even in high-income countries, it´s necessary to rethink the concept of clinical value and find mechanisms to review and re-evaluate over time. Those responsible for payment and reimbursement of medicines have to make tough decisions about which basket they should put their limited amount of money in. For those decisions to be fair to all stakeholders, they need to be grounded in better evidence”, says Dr. Lars Lööf.

Considerable research activity has resulted in many new approved drugs for cancer therapy, along with countless products currently in the pipeline. When introduced on the market, they often come at very high prices, although the evidence for their true clinical value in relation to the price tag is immature at the time of marketing approval. Thus, the price tag may not correspond to their actual value to patients or to the healthcare systems that are buying them; the drugs may be more effective than the products currently available, while others works no better than older ones. In many cases, it is simply too early to say.

It is difficult to fully evaluate the true value of a new drug based on results from pre-marketing clinical trials as the conditions may differ when the drug is used in clinical routines. The real outcome (e.g. long-term survival, life-quality, adverse effects etc.) may not be established until the drug has been on the market and in use in the health-care systems for years. Many new drugs today pass through “fast tracks” in the authorization process for marketing approval. Thus, there is an urgency to provide access to new treatments under controlled conditions to increase the knowledge and understand the full potential and value of new cancer drugs when on the market", says Professor Lööf.

In the workshop True Clinical Value and Price-Setting for New Cancer Drugs at Uppsala Health Summit Professor Lööf and his colleagues hope to stimulate a discussion around changing focus from marketing approval to a life-style perspective of new cancer drugs. The goal is to achieve a knowledge-base and sustainable foundation for evaluation of the true clinical value, prioritization, health-economic evaluations and price-setting. For example, what should you pay for a cancer drug at a certain point of time after marketing approval is being used for different types of indications and by many more patients, compared to when it was first released for just one type of rare indication? And are the current prices of the new complex treatments reasonable, considering that there are not enough patient-reported outcomes yet?

We welcome a broad group of different stakeholders to our workshop who would like to explore the idea further of establishing the true clinical value for new cancer drugs. This involves representatives from industry, clinicians, health-economists and patient organizations. We would like to have a an outcome-oriented conversation, while also considering the complexity of the issue. We have to respect the industry perspective; of course they will attempt to increase their revenue and they must be able to protect their ability to invest in research and development. But for drug development to be sustainable in the long-term, and for health systems to be able to make fair and equal decisions around treatments, we have to find alternative ways and international cooperation to continuously collect knowledge during the life-cycle of new drugs, to fully understand and determine their value”, says Professor Lööf.

In the workshop several questions will be brought to the table: Can new options in the regulatory processes be created that support and enable continuous collection of evidence after marketing approval? How can we achieve an efficient, fair and equal access to new drugs? How can we consider the perspectives of children and elderly people, in order to achieve information on, for example, the influence of age-specific factors of life quality, tolerance and safety of a specific new cancer drug?

The three-hour workshop will not give all the solutions, of course, but I hope it will raise a couple of suggestions concerning potential steps forward to be taken in coming years - who must be involved, who have to take the first steps, and are there platforms today that we can build on for multi stake-holder discussions”, says Lars Lööf.