Today’s prevailing wisdom is that R&D costs are on the rise while ROI on those investments are down. The Tufts Center for the Study of Drug Development puts the total costs of new drug around $2.7B (in 2017 dollars) while another study1 highlights the fact that the ROI on R&D investment has fallen to 3.7% which is the lowest figure in the past six years. As such, the pharmaceutical industry’s focus on improving productivity is a critical issue.
This week on The Pharmcast, we turn our attention to the topic of rare disease, and more specifically: how to engage and educate rare disease patients to advance development. It's estimated that nearly 30 million Americans, or nearly 1 in 10 people, are living with a rare disease - and almost 95% of rare diseases don't have any FDA-approved treatment.
One of the unique challenges and perhaps keys to rare disease development is patient engagement. Patient populations are small by definition. Roughly 50% of patients are children, and benefit-risk equations are completely different from those of more traditional diseases. To learn more about engaging rare disease patients, we sit down with Dr. Lorna Speid, to discuss her stories of rare disease drug development, outline the unique challenges of engaging the rare disease population, and her advice to developers.
I’ve just returned from a cold, blustery and wet London where I attended the annual Pharma Access Leaders Forum.
But the meeting was hot.
In fact, piping hot when you consider the tectonic changes being felt in the world of healthcare technology assessment (HTA) and their implications on both patient access and innovation. And the linkages couldn’t be more profound.
Wither HTA in the EU? A key red thread through a series of potent discussions was real world evidence (aka, “outcomes data”). Head honcho HTA officials from across Europe (including England and Scotland – both still in Europe as of last report) returned again and again to the value of outcomes not just for the evolving world of Risk Sharing Agreements, but for the acceleration of reimbursement science.