Higher chances of healing through genetic medicine

Genetic medicine is the term used to describe active substances which are either of genetic origin in terms of their biological structure or which target genetics in terms of their active profile. The proportion of portfolio companies of BB Biotech that deal with this growth topic are increasing steadily. A first position was established in 2018, when the effectiveness of the gene therapy approach for the treatment of X chromosomal myotubular myopathy of the US company Audentes became apparent.

A more precise diagnosis is the basis for deciding who qualifies for treatment. Advances in molecular diagnostics mean that more and more biomarkers are available for use in such tests. Biomarkers are biological indicators that can be used to investigate cell metabolism or analyze changes in the genome. The stated aim in cancer treatment is to find out which patients have particular gene mutations that trigger the growth of tumor cells.

Based on the test results, the treatment likely to respond best to specific individual characteristics will be selected. For patients, this means better chances of a cure and fewer unwanted side effects. The first genetic medicines ready to enter the market were for the treatment of cancer. Oncology continues to account for the lion's share of market-ready products and clinical candidates. New genetic medicines are increasingly being used in other therapeutic areas, however, primarily in the management of rare inherited diseases or in the treatment of metabolic disorders, autoimmune disorders and cardiovascular diseases.


The new markets of tomorrow

The implications for drug development are: Patient populations will be smaller but the medical benefit will be all the greater. Biotech companies are ideally placed to benefit from advances in genetic medicine. With more and more products receiving breakthrough designation because they are truly innovative, regulatory procedures leading to approval are easier and shorter. At the same time, the financial effort decreases because of the smaller numbers of patients required for clinical studies. Another positive effect is that biotech companies can cater to the smaller target groups for the products with their own sales team at reasonable cost.

Because of the perfectly customized effect, these products fetch higher prices. Industry experts expect cancer therapies in particular to generate significantly higher revenues over the next few years. Four gene therapy products have been approved in the USA and in the EU so far. Approximately 2 800 substances are undergoing clinical trials worldwide. The treatment usually involves a prolonged inpatient stay and has the potential to bring about a full cure, so it has its price. The leader of the pack is Zolgensma, a one-time-only gene therapy to treat spinal muscular atrophy that can bring about a permanent cure, which comes with a price tag per treatment of USD 2.1 mn.

To justify their prices, companies will have to demonstrate exceptional medical benefit of their products – the benchmark increasingly being a temporary or permanent cure. The political debate over health care cost containment needs to be addressed at the same time. Especially when it comes to gene therapies for monogenic disorders, the pay-for-performance model is likely to carry the day. Full reimbursement is linked to the success of treatment, however this may be defined. Payment by milestone is one variant.