“Underserved” therapeutic areas can cover a wide variety of disease. This panel saw the greatest potential in working closely with patient groups to develop new therapies for rare diseases, where there are over 7,000 but only 200 therapies. China offers a massive market to bring transformative medicines to patients, especially in cancer, where there are four million cases a year, and liver disease, where half the world’s patients are there. Sometimes a clever scheme such as “volume guarantee” is needed to resolve supply and demand imbalances, for example in the provision of long lasting implantable contraceptives for women in developing countries.
For the BIO-Europe Spring® 2019 Plenary session in Vienna, Amy Schulman, moderator, and partner at Polaris Partners opened discussions by asking the panel to define what is meant by an “underserved” therapeutic area? Is it defined by geography, patient population, reimbursement, or something else?
Roche Pharma Partnering’s head of business development for pharma research and early development, Michael Crowley, thought more in terms of whether there is an opportunity in, for example, a rare disease for a partnership to revolutionize the standard of care. Roche has partnered with 4D Molecular Therapeutics to develop gene therapies for eye diseases, just one of ten molecules across nine rare diseases the company has in development.