The pharmaceutical care landscape is presently characterised by a number of challenges, namely an increase in regulatory requirements and R&D costs, coupled by a paradoxical decrease in the number of new active ingredients reaching the market.
The pharmaceutical industry must operate within a climate of increased public demand for higher medical value – safer, more effective medicinal treatments that positively impact public health at a reasonable and affordable price – within a sustainable business model that provides investors and shareholders with continued return on investment.
These quandaries were debated at the leading EU health policy conference, the European Health Forum Gastein in Austria last month as part of the wider discussion on EU regulatory framework improvements within the context of health care distribution in a globalised, ageing society.
As the pharmaceutical industry becomes ever-more specialised, the customisation of medicinal treatment to the individual patient represents a major leap in the industry’s approach to healthcare, and has followed the great medical advances in genetics, genomics, pathology and molecular science. These scientific disciplines have led to better treatment decisions and targeted therapies through the development of more precise diagnostic tools. The scope of personalised medicine lies in tailoring medicinal treatments and diagnostics to specific patient sub-groups.
Although there is no uniform definition of the term “personalised medicine”, it is often described as the tailor-made provision of “the right treatment to the right person at the right time”, creating a paradigm shift that has given rise to new opportunity and challenge.
Personalised medicine has an important role to play in the diagnosis, prevention and treatment of rare, hereditary chronic life-limiting disease. It is recognised that personalised healthcare delivers added medical and economic value by offering better clinical outcomes, reducing both the incidence of ineffective treatment and adverse drug reactions which inflate healthcare costs. This is facilitated by the inclusion of biomarkers or diagnostic tests in drug dossiers. Personalised medicinal treatments also create better value for pharmaceutical companies that deliver them as they are more likely to be accepted and reimbursed by national health care systems.
Patient stratification and the consequential segmentation of drug markets is set to increase the benefit-risk ratio for the targeted patient sub-group, while boosting market penetration for the specific indication. It is hoped that development and registration of personalised medicines will also be shorter. Thus the initial lower unit sales resulting from targeting a narrower indication will be commercially compensated for.
Personalised medicine goals are to be achieved by industry through investment in new pharmaceutical production technologies, the streamlining of pharmaceutical and diagnostic expertise, and analogous development of the medicinal product and diagnostic test through the fostering of multi-disciplinary R&D processes.
European pharmaceutical policy is centred on the simultaneous pursuit of “Solidarity and Innovation” to maximise public health within the limits of the available resources within an ethical framework, while also improving quality, access and availability, and controlling costs. In this regard, the EU institutions have long recognised that a strong pharmaceutical industry is needed to strengthen the internal market and provide its citizens with high quality, affordable medicines. The achievement of these aims necessitates a strong legislative framework and comprehensive scientific guidance.
The modernisation of the Pharmacovigilance regulatory framework and the upcoming counterfeit medicines legislation as part of the EU legislative “Pharmaceutical package” are both set to improve patient safety, while a new proposal for the Clinical Trials Directive is currently underway, following extensive consultation with stakeholders on addressing the concerns that impact performance and research. The EU has made headway in multinational clinical trial assessments, and in its acknowledgment of the global dimension of clinical trials and R&D is currently developing the health technology assessment.
While the quality of EU licensed medicinal products is guaranteed, the European Commission has acknowledged that efficacy is not always consistent in all patients and that this can be remedied by increasing commitment and investment in personalised medicine. The spiralling costs of new medicinal therapies however pose a heavy burden on healthcare budgets and an added challenge in promoting the manufacture of tailor-made innovative medicines at an affordable price.
The study of pharmacogenomics and pharmacogenetics are fundamental to regulatory approval and clinical use of personalised medicinal products. The research goal is the identification of the genetic profile of patients who are more likely to suffer from adverse effects or to have an optimal response to a drug – thereby optimising the use of effective, targeted medicines at public health level. Some genetic biomarkers in fact have already been validated by regulatory authorities and thus form part of the drug development process.
However the demonstrated clinical utility of a deeper understanding of genomics has also been cloaked in scepticism, the major concerns being how the raw data can be annotated into useful information that affects physicians’ diagnosis and translated into the development of new medicinal therapies, as this is considered to be many years away from completion. Multi-disciplinary cross-border collaboration and a strong, global clinical bioinformatics infrastructure are required to store, handle and integrate data and share best practices.
It is evident that new approaches are needed to ensure innovative drug development delivered at added medical value to patients and public health budgets. The modernisation of regulatory and licensing procedures for simultaneous registration and marketing of the drug and the diagnostic test, and the standardisation of biomarker tests, are necessary requirements.
The lack of harmonisation of the regulatory approval process and reimbursement schemes, and the administrative hurdles and expenses in marketing advanced therapies are also said to limit economic opportunity. It would therefore appear that the promise of personalised medicine is yet to be delivered – and the window of opportunity is wide open.
azammit@fff-legal.com
Dr Zammit is an associate at Fenech Farrugia Fiott Legal, specialising in pharmaceutical and medical law within the firm’s corporate and commercial law practice area. She is also legal advisor to the healthcare business section of the Chamber of Commerce, Industry and Enterprise.