The scientific advances that we are witnessing mean that we know more about ill health than ever before. The knowledge that we have gained is being translated into new therapies to treat a wide variety of often age-related conditions, including cancer, Alzheimer’s disease, and type 1 diabetes. We are also making progress in the fight against rarer conditions, such as haemophilia.

Improved treatments offer hope and help to patients, carers, and their families. This has a positive impact on the wider society, helping people lead healthier, more productive lives. In fact, industry pipelines are brimming with innovation. CAR-T cell therapy will transform the way that we address blood cancers and disorders, potentially increasing the life expectancy of a patient with acute lymphoblastic leukaemia by over 60 years.

Cell therapy, in which living cells are injected into the patient to treat the causes of their disease, may be used to treat common conditions such as type 1 diabetes. This novel therapy can be used to control blood sugar without the need for constant insulin injections. This will aid in the restoration of normal pancreatic functionality and, as a result, will restrict the onset of serious, long-term health conditions. That is good news for the estimated four million people in Europe who live with type 1 diabetes.

Gene therapies, which repair the direct cause of genetic disease, by introducing genetic material into cells to compensate for abnormal, ill-expressed or missing genes, have the potential to relieve or even cure rare diseases where the patient’s quality of life and prognosis are poor, such as haemophilia B. In fact, gene therapy could mean that patients might avoid a lifetime of prophylactic or bypass therapy.

It is already becoming clear that the launch of new technologies, along with the resulting opportunities created for patients, will pose challenges to healthcare systems. Developing new ways to assess the costs and benefits of these innovations will be essential if their introduction is to be managed effectively. Early planning and action will enable the careful introduction of new treatments in ways that ensure their most effective use, maximise their best affordability and – most importantly – enable all eligible patients to benefit as soon as possible. EFPIA and our members want to work with partners to help prepare for these exciting new advances to ensure that patients with unmet medical needs are able to benefit at the earliest opportunity.

Author:

Nathalie Moll was appointed Director General of the European Federation of Pharmaceutical Industries and Asscociations (EFPIA) in 2017. Previously, she was the Secretary General of EuropaBio, the European Association for Bioindustries. After graduating with Honours in Biochemistry and Biotechnology from St Andrews University, Scotland, and working for a stage at the European Commission, Nathalie has spent 20 years employed in the biotech industry at EU and national level in associations and corporate positions.