Director DayOne Initiative, Senior Project Manager ICT, Public Relations
Tel. +41 61 295 50 16thomas. brenzikofer@baselarea. swiss
Can the treatment of rare disorders be a role model for healthcare innovation in precision medicine? This was the question at this years’ last edition of the DayOne Experts event series. The answer was, simply put: Yes.
As we move forward to a better understanding of diseases, driven by ever more data analysis, common disorders are likely to split up in different manifestations of the same complex disease each affecting a smaller number of patients which are then to be treated with more targeted therapies.
This trend can be explained with the long tail. The Theory states that in a world where products find consumers through digital platforms with unlimited shelf space, retailers make their profits by selling smaller volumes of more differentiated products rather than selling one single product in high volumes.
As healthcare in precision medicine moves toward the long tail, the question is: What would a multi-niche-platform business in healthcare look like? Success stories in the treatment of rare disorders could unveil some insights into this topic as they had to exactly do this: cope with small numbers.
The speakers and panelists represented the whole healthcare spectrum from patients to science and market access. They made clear which challenges are to be faced if healthcare innovation will move in the direction of small numbers:
1) Having an active patient community on board from the beginning is key. Only patients know what it means to live with a condition and therefore which outcome measurements are relevant to show efficacy of a given treatment
2) The development and approval of a therapy has to be more agile as the close involvement of active patients will probably mean outcome measurements may have to be adapted on the way. This also means that regulation has to become far more flexible than it is today.
3) The industry will have to come up with new business models to facilitate market access. Outcome based reimbursement will become the norm and technologies to measure these outcomes a key enabler.
A big thank you all the speakers and panelist and pa for sharing their graeat stories and insights:
Rocco Falchetto, Swiss Society for Porphyria
Richard Welford, Idorsia
Alexander Solyom, Enzyvant
Jennifer Cain Birkmose, Roche
Jasmin Barman-Aksoezen, Stadtspital Triemli, Zurich
Morteza Yazdani, Shire
We will be back in february starting the DayOne Experts Series 2019 on „Aging and Longevity“