The increased knowledge in cancer biology and the introduction of new drugs have contributed to an improved survival and, in many cases, also to increased quality of life. However, not everyone benefits.
In fact, the positive effects are only seen in a smaller proportion of patients and sometimes with troublesome side effects that may be lifelong. We mean that patients are worth better than many times receiving ineffective treatment that is not appropriate for them. Only then can you start talking about a personalized medicine. We also propose that the limited knowledge in personalized health care of today could be an opportunity for Swedish life science by using our registries with improved involvement of patients and better use of Patient Reported Outcome Measures (PROMs), in combination with all the efforts that now are seen at the preclinical level.
“We must dare to discuss the difficult ethical question of if it is reasonable that a large number of patients are exposed to an ineffective treatment and are at risk of serious adverse events to find the small proportion of patients who will benefit.”
It must be clearly stated that this is not an issue about whether or not severely ill patients should have access to new drugs that can be potentially life-saving. We live in a welfare state and can afford it. But at the same time, we must dare to discuss the difficult ethical question of if it is reasonable that a large number of patients are exposed to an ineffective treatment and are at risk of serious adverse events to find the small proportion of patients who will benefit.
“It is a paradox that the research and pharmaceutical industries have failed to develop markers that can predict who will benefit from a certain given treatment to the same extent as they have developed new drugs.”
Despite the fact that the development of targeted therapies and drugs that strengthen the immune system gives great hope, it is a paradox that the research and pharmaceutical industries have failed to develop markers that can predict who will benefit from a certain given treatment to the same extent as they have developed new drugs.
At the same time, we see that the new drugs are considerably more expensive than older medicines. Therefore, it is not unreasonable that society (state and county councils) has an improved accountability to ensure that both patients and society get the maximum benefit from these treatments, with the goal of a considerably higher efficacy than today.
The high price has become a challenge for healthcare and society. In order to manage and control costs, both society and industry have built up resource-consuming administrative organizations nationally, as well as in the 21 individual Swedish county councils. This scheme also contributes to difference in access to new drugs. Large resources are used that could be used better.
“Here is where we also see a great potential for better and direct use of the experiences of patients and their relatives by including measurement.”
At the same time, the desire to quickly address new treatment options in severe diseases has led to the introduction of several new drugs based on minor studies that make assessing results uncertain. Furthermore, when follow-up data of patients treated outside clinical trials in routine care is missing, the possibility of learning the real value of these new treatments is hampered. Here is where we also see a great potential for better and direct use of the experiences of patients and their relatives by including measurement, like PROMs.
Only a limited proportion of patients have a clear benefit
Now, with the support of a new generic cancer drug registry, we can show that a large proportion of the patients in the catchment area of Stockholm County Council (SLL) treated with a new immunotherapy treatment receive a demanding treatment that has no positive effects. The register shows that half of 690 treated patients did not respond to treatment at all and about 30 percent have a two year survival. Similar patterns can in fact be seen in the pivotal published studies.
According to publicly known figures, SLL has paid 214 million (SEK) for these treatments – whether effective or not – since the introduction of the three currently evaluated drugs. This cost does not include all the resources required in hospitals with frequent visits, drug preparation and treatment of side effects. Large amounts of resources go to actions that are without any positive effects. Similar conditions are seen for most cancer drugs, with only a limited proportion of patients having a clear benefit. All of the data will soon be disclosed in a scientific journal.
This limited study with a regional registry (currently being introduced nationally within the framework of the Regional Cancer Center) shows that our unique registry in Sweden has a great potential to contribute to a knowledge development that benefits the individual affected patient, but also will benefit society and ultimately even the researching pharmaceutical industry.
A Swedish model
The road ahead for us in Sweden could be based on a strong citizen and patient perspective, where society (with an independent academy and health care) assumes greater responsibility for better individualized treatment. This can be done through better and transparent use of our quality registers, our regional and national healthcare collective registers. In this respect, there is also a need for legislation to review and enable broader quality monitoring without compromising patient integrity.
“It is important to create conditions that can assure that Sweden is a future country of interest for the pharmaceutical industry.”
Globally, everything points to the fact that the pharmaceutical industry will continue to grow and competition between countries for corporate investment will increase. Therefore, it is important to create conditions that can assure that Sweden is a future country of interest for the pharmaceutical industry.
The potential for optimal development of a successful follow-up of new cancer drugs would therefore help the pharmaceutical industry to continue to be one of Sweden’s most important industries and, in the future, contribute to community development.
Against this background, one understands the need to quickly find predictive markers and patient-reported measures that can indicate which patients will benefit from a given treatment and those who will not. The county councils and universities, as well as the pharmaceutical industry, should have a clear mission to assist healthcare and patients with such knowledge.
“Industry should also improve their own collaboration and use common markers and methods in their clinical trials.”
Industry should also improve their own collaboration and use common markers and methods in their clinical trials to provide an optimal interpretation of results leading to better conditions for individualized treatment. This work can of course be done in conjunction with interested companies, while we must be able to handle differences between the citizens’ preferences and the commercial operators’ needs for returns in their own company.
Sustainable patient care
It is obvious that there is a huge need to create long-term sustainable research and development that includes both models for funding and analysis that can identify which patients benefit from a particular treatment.
“By comparison, how many would step on a train if less than half of the passengers arrived at the destination?”
However, it cannot solely be the responsibility of society (the buyer) to find resources. The seller (the pharmaceutical industry) must also improve their contribution.
As is the case for other companies in our society, the pharma companies are also responsible for ensuring that their products are efficient and safe for anyone to use. By comparison, how many would step on a train if less than half of the passengers arrived at the destination?
Roger Henriksson, Professor, Chief Physician, Norrlands University Hospital, Chairman of the Expert Council for Therapy of Cancer Diseases, and member of the Executive Board of the Pharmaceutical committee, SLL
Kjell Bergfeldt, Senior consultant, Member of the Board of Swedish Oncology Association, Chief of Operations and Chief Physician Skandionskliniken, Uppsala
Johan Falkenius, Pharmaceutical representative physician, Dept of Oncology/Tema Cancer, Karolinska University Hospital and Member Expert Council for Therapy of Cancer Diseases, SLL
Eskil Degsell, Patient Representative, Regional Cancer Center Stockholm Gotland and Patient Representative for Brain Tumour Pathway at Karolinska University Hospital.
Sven-Åke Lööv, Member of the Expert Council of Therapy of Cancer Diseases, SLL and Member of the National Council for New Cancer Therapies