Heinz Zwierzina M.D., CDDF Managing Director tells us all about biomarkers and patients’ access to personalised oncology drugs in Europe in this insightful article
It is becoming evident that the more we know about microbiology, the more complex drug development becomes. Histopathologically well-defined tumour entities become multiple different diseases classified by genetic alterations. These may differ from one cancer type to another and the ones we commonly see in, for example, lung cancer aren’t always the ones we see in breast or colorectal cancer. Another issue is that usually, there are only a very small number of patients with a particular mutation. Thus, we will need to adapt the way we design clinical trials to show whether a specifically targeted drug is effective in a very small patient population.
Biomarkers can be objectively measured and evaluated as indicators of normal or pathogenic biological processes or of pharmacologic response to therapeutic interventions. As such, biomarkers hold great potential to predict clinical outcomes and define a personalised treatment strategy. Given the complexity of the neoplastic process, in most instances, a single marker cannot offer the necessary sensitivity and specificity. Therefore, research is now focussed on the development of multiplexed assays that screen multiple genes and proteins at the same time.
The need for collaboration among all stakeholders
There is a huge clinical need for minimally invasive tests to determine the subgroups of patients with a high probability for (non-)response to therapy. Moving to the future, it will be very important to move from sporadic testing of patients to ‘real-time oncology’, using novel biopsy methods (such as liquid biopsy) to meet the challenges of repeated biopsies, and also leverage big data analyses, as well as artificially supported intelligence and machine learning. This should integrate the continuous assessment of key genomic, epigenetic and proteomic analysis, describing the disease, the micro-environment and the interaction, along with measurement of real-time impact for any modification in treatment. This should help understand the sources of variability in a medicine’s benefit/risk profile.
Regulatory challenges, the hurdles to achieve reimbursement, and access to clinical data are all barriers that will need to be further addressed by all stakeholders to achieve the widespread use of biomarkers in clinical practice in future. Although small compared to the costs of oncology drug development, the development, validation and commercialisation of biomarker assays are expensive. Therefore, in order to realise the potential value from biomarkers, early dialogue between industry, regulators and payers is very important.
The Cancer Drug Development Forum (CDDF) recognises the complex issues of integrating biomarker development into innovative oncology drug development. It organises and encourages multi-stakeholder meetings and workshops among academia, the pharmaceutical industry, regulatory authorities, health technology assessors, patient organisations, as well as payers to address these challenges.
Health Technology Assessment (HTA) and patient access
From the healthcare system perspective, access to biomarkers that define the patients that may or may not respond to therapy will be crucial to improving treatment standards and efficiency of care. However, the reimbursement of biomarker testing varies from country to country. For example, the K-ras testing is reimbursed at significantly varying rates across the EU Member States.
The key challenges for biomarker testing in Europe are:
- Accessibility of biomarker testing varies from country to country, but also within cities and regions. Biomarker diagnostics are often performed at larger hospitals only, access to which may add additional barriers to timely diagnosis.
- Administrative barriers lead to delays and longer waiting periods for biomarker test results, which vary from a few days to a month in some countries. There is no established organisation between hospitals to perform a test and share the results of these tests effectively.
At a European level, there is a need to promote research and development in biomarkers, adapt regulatory frameworks to the specificities of new health technologies, and harmonise HTA across the Member States. At the national level, there is a need to train healthcare professionals and patient advocates on the importance of biomarker testing, and to adapt reimbursement frameworks to the specificities of new health technologies. Lack of consistent HTA processes across Europe create a barrier to entry for novel diagnostics because they require a significant additional investment from diagnostics companies to navigate the payer landscape.
The way ahead
The close collaboration between the Cancer Drug Development Forum (CDDF) and the European Cancer Patient Coalition (ECPC) aims to raise awareness and educate patients/policymakers regarding biomarkers and personalised medicine. Biomarker testing holds value for facilitating faster diagnosis, a targeted personalised treatment plan, and avoid wasting resources on ineffective treatments.
Innovative access strategies are required to support rapid reimbursement and adoption of biomarkers in clinical practice. Finally, only a multistakeholder approach can ensure the attractiveness of Europe as a market for the development and launch of oncology biomarker tests.
Heinz Zwierzina M.D.
Innsbruck Medical University
CDDF Managing Director
The Cancer Drug Development Forum (CDDF)
Tel: +32 2 775 02 15