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Glybera Becomes EU’s first Gene Therapy in Landmark Ruling

The road towards personalised medicine becomes smoother as regulators give the green light to the Europe’s first approved gene therapy.

In a landmark decision, the European Commission approved UniQure’s drug Glybera, a single-use gene therapy treatment for patients with the rare, inherited disease lipoprotein lipase deficiency (LPLD). This significance of this decision cannot be emphasised enough as this is the first gene therapy approved for use in Western Europe, although China approved the world’s first gene therapy in 2003. Additionally, the US has still not approved any gene therapies. 

Results from clinical trials showed that this gene therapy provided a higher benefit for LPLD patients in terms of safety, diet and quality of life than the traditional protein replacement therapy. This means that although Glybera will reportedly cost £1 million per patient, its efficacy and safety standards will make it more cost effective in the long run. In addition, it is hoped that Glybera will make the approval of other gene therapies for Parkinson’s disease, muscular dystrophy, haemophilia and retinal degeneration easier as the healthcare community continues to gain a better understanding of this type of treatment.

Therefore, in the long – term, the trial and error approach in prescribing could be avoided as healthcare providers would offer patients a personalised gene therapy. With an improved knowledge of genetics, doctors could make better use of their consultation time and predict their patient’s future health patterns.

Furthermore, patients will be empowered to predict and manage their own health outcomes.  They will gain a better understanding of their disease and body through their individual genome- based data. Self-management of one’s own disease was supported by a recent Pew Internet and California HealthCare Foundation report which stated that 60% of US adults track their exercise routine, weight or diet. Moreover, one third of caregivers also monitor these health indicators for their loved ones. Additionally, 34% of self – trackers said that data collection informed a health decision while 46% said that self- tracking changed their approach to healthcare on the whole.            

Personalized medicine can also help the pharmaceutical industry, in accordance with a new value-based business model, develop differentiated products that reduce system-wide costs and creates a more efficient production and distribution cycle for medicines in the future. An ageing population increased the prevalence of chronic diseases meaning that patients respond to the same medication in different ways depending on their genetic make- up. Pharmaceutical research can no longer depend on using a variety of different compounds on individuals in the hope that one will offer more benefits than side effects to the patient.

Personalised medicine will allow pharmaceutical companies to create safer, more effective treatments for patients. However, at the Gastein Health Forum, Austria in October this year, policymakers stated that development in the personalised medicine sector was prevented by the lack of standardised rules for data storage and protection. Currently, a pan-EU Biobanking and Bimolecular Resources Research Infrastructure (BBMRI) hopes to make it easier to access samples of human blood, DNA, tissue, cells or related data and will help pharmaceutical companies create new treatments at a rapid pace . However, these samples which will help the healthcare as well as the pharmaceutical industry understand the relationship between genes, lifestyle, the environment and diseases, are currently underused as regulation and ethical considerations lag behind.

All in all, Glybera, as Europe’s first approved gene therapy, will make it easier for more genetic therapies to be approved in the western world. However, in order to benefit from the personalised medicine sector, the pharmaceutical industry should change their current business model so that they start catering to a select number of patients which would ensure long-term rewards. Although there are current issues with data protection and storage in the EU, pharmaceutical companies could foster partnerships other healthcare or genetic sequencing companies so that they are constantly developing their ‘niche buster’ drug range. In any case, personalised medicine will be an evolutionary rather than a revolutionary process which is dependent on all healthcare stakeholders focusing on the body as a whole rather than a disconnected set of organs, for the first time.

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Patient-Centered Clinical Trials

Sep 4, 2014 - Sep 5, 2014, Boston

Unleash the Potential of Patient-Focused Clinical Research: Redefine Recruitment, Enrollment and Study Management