Drug Discovery, Development & Delivery
The Danish Approaches for Personalised Medicine – For the Benefit of Patients The term tailor-made or personalised medicine covers a form of drug treatment in which the use of drugs is adapted to the individual patient's very special disease situation and biological characteristics. The desire is to achieve a unique treatment result that best meets the individual patient's genetic profile. The Danish healthcare system offers unique possibilities for realising the potential of personalised medicine. Based on a common national information infrastructure, the new genome centre in Denmark will ensure the entire setup for collecting samples from patients for genome sequencing and analysing data for research or treatment purposes. The data and research will be used to identify medicines that have the best effect on different diseases and patient groups. Together with data from existing national registries and databases, as well as the national biobanks, Denmark has established a solid national framework to develop tailored personalised medicines to benefit patients. Not All Medicine Benefits All Patient Groups Humans are not alike, which is why we often react differently to the same medicine. One of the great benefits of personalised medicine is that the quality of treatment will increase significantly. It will be possible to give patients the treatment that gives the greatest possible effect the first time, while reducing the risk of side-effects. This will improve the patient's quality of life. With personalised medicine, it is possible to achieve more health for the money, as patients receive correct medication the first time. Unnecessary money is not spent on costly medications that are not effective or perhaps even harmful to the patient. A large proportion of the medicines offered in healthcare today do not work or are directly harmful to the patient. It is both stressful for the individual patient and a socio-economic challenge. This is a global problem that is also manifesting itself in the United States, among other places. Figure 1 is from a US 42 INTERNATIONAL PHARMACEUTICAL INDUSTRY
study conducted by the US Food and Drug Administration (FDA) and illustrates the proportion of patients who do not benefit from the medication they receive for their disease. Why Personalised Medicine The term "personalised medicine" was first used in April 1999 in an article in the Oncologist: "New era of Personalised Medicine - Targeting Drugs for Each Unique Genetic Profile"1. Since then, and especially in the last few years, the number of scientific articles specifically dealing with personal medicine has grown steadily, so that today hundreds of articles on the subject are published every year. Figures from the United States compiled by the Food and Drug Administration (FDA) show that 12 out of the 48 new drugs approved in 2019 were in the personal medicine class – that is, 25 per cent of all new drug approvals2. While the total percentage of personalised medicines approved by FDA decreased from both 2018 (42%) and 2017 (36%), the FDA approved many new indications for existing, approved personalised medicines, providing new treatment options for many patients. And furthermore, several biosimilars for personalised medicines were also approved. The development in personal medicine is therefore not just a future scenario – it is reality here and now. Diseases develop differently from patient to patient, who in turn respond differently to the same medication. Some need one tablet, while others need two tablets for it to have an effect. Some people benefit from a standard treatment – while others simply experience side-effects. One perspective when using personal medicine is that medicines are only given to those patients who are known to have an effect on the medicine – this is already seen today in connection with the use of certain cancer medicines. Another perspective is that one can predict via molecular diagnostic tests whether a patient will have the desired effect or experience serious side-effects with a given drug treatment. One step is
therefore to have knowledge of a drug that is particularly effective in relation to patients with a particular genetic profile. Another step is to have a test that can determine if the specific patient has a genetic profile that matches the use of the desired drug. Therefore, drug and concomitant diagnostic tests are usually closely related when talking about personal treatment – illustrated in Figure 2. The use of personalised medicine was previously most prevalent in the treatment of cancers. This makes sense, since cancer is one of the therapeutic areas that has the most ineffective drugs for treatments (Figure 1). However, today the treatment principle is being transferred to many other disease areas. In 2019, there were a significant number of approvals for the treatment of non-cancer disease. Seven of the 12 new personalised medicines drugs, approved by the FDA in 2019, were for indications outside the field of oncology. This includes the approval of ZolgensmaTM (onasemnogene abeparvovec-xioi) for the treatment of spinal muscular atrophy. This gene therapy corrects bi-allelic mutations in the SMN1 gene2. The Danish Approach to Personalised Medicine Many countries, like Denmark, are in the process of developing strategies for personalised medicine. One of the first initiatives in the Danish Personalised Medicine Strategy was the establishment of a National Genome Center in May 2018. The National Genome Center provides genomics-based analysis and insights related to diagnostics of relevant patient cases at Danish hospitals. The establishment of the National Genome Center means that Denmark will be able to raise the overall effectiveness of personal medicine across the entire healthcare system to a level that would otherwise take a long time to achieve within individual hospitals. The Danish effort is unique in three areas. Denmark focuses on personal medicine benefiting patients immediately. In addition, the country has public control over the use of information from all Danish patients3. And finally, Spring 2021 Volume 13 Issue 1
