Hemophilia Drug Development Summit Takes Aim at Hurdles Facing New and Upcoming Treatments

Key leaders in hemophilia therapy development, representing large pharmaceutical and biotech firms as well as academia and contract research organizations, will gather at the Hemophilia Drug Development conference in Boston on Aug. 14 through 16.

The meeting is designed to be an industry-dedicated networking and discussion forum, and many of the major experts in  hemophilia — pharmaceutical and academic — will participate. About 80 percent of all attendees are expected to be industry representatives, followed closely by academic researchers (8 percent) and “solution and service providers” (9 percent).

Participants will have the opportunity to gain new insights into the long-term impact of new and potentially revolutionary extended half-life products, non-factor products, and gene therapies that are being developed to treat hemophilia.

Discussions will also focus on strategies to overcome the hurdles in the development of, and marketing access to, new hemophilia treatments, facilitating their translation into the clinic.

Several key aspects of drug development will be addressed, including:

• Optimizing patient-centric approaches to ensure clinical trial success;
• Improving the recognition of underlying biological mechanisms involved in next-generation therapies;
• Better understanding of patient motivations to adopt novel therapies;
• Exploring how to improve clinical trial design to obtain clinically meaningful results in an era of new drug development;
• Discussing, in-depth, reimbursement models that work to ensure effective market access.

The summit will have 21 expert speakers, including Robert Peters, senior vice president of research at Bioverativ; Henry Mead, global medical director of hematology at CSL Behring; Mark Skinner, president and CEO of Institute for Policy Advancement; Alison Schecter, global program head of rare diseases at Sanofi Genzyme; and Howard Levy, chief medical officer of Catalyst Biosciences.

Pharmaceutical companies including Spark Therapeutics, Novo Nordisk, uniQure, Bayer, and Shire will also be represented at the conference.

Such discussions are highly relevant to hemophilia patients and caregivers, said Michelle Rice, senior vice president for external affairs with the National Hemophilia Foundation. That’s because those touched by hemophilia are both interested and motivated to embrace new and promising treatments that impact everyday life.

“These new therapies have the potential to greatly improve the quality of life for those eligible to utilize them,” said Rice, who will speak on a panel looking at ways of demonstrating treatment benefits while linking them to costs.

“[I]magine being a patient with severe hemophilia and an inhibitor and having to infuse yourself 15–30 times a month; im-agine how that impacts your ability to engage in work, school or social activities, Rice said. “Now imagine being able to infuse subcutaneously once a week or even once a month…what an amazing sense of freedom!”

But, she continued, “This is a community that has experienced more than their share of issues related to product safety. … Advocacy efforts” are, for this reason, crucial to ensuring access and acceptance.

Participants will also have the opportunity to register for two pre-conference workshops set for Aug. 14.

• Workshop A will be led by Liselotte Jansson, chief research officer at Apitope, and will be address immune reactions against hemophilia therapies and strategies to overcome them.
• Workshop B will be led by Irina Matytsina, international medical director of global development at Novo Nordisk, and will discuss clinical trial design and strategies to optimize their outcome.

Early registration discounts are offered through July 13; more information is available here. Pricing differs according to type of registration (industry or academic, for instance), and savings of up to $600 off regular prices are possible.

Event organizers say attendees will have the opportunity be part of “cross-industry discussions” that could pave the way for advancing “innovative hemophilia therapies that will transform the lives of patients.”

The meeting’s venue is The Westin Boston Waterfront, 425 Summer St., Boston. Registration details can be found online, by email at [email protected], or by calling +1-617-455-4188.