from BIOtechNow
The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases. With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline. In anticipation of an even more compelling opportunity, many companies are taking a step beyond and focusing on ultra rare diseases.
Last week’s 11th Annual BIO Investor Forum in San Francisco hosted a panel, Raring to Go: The Race to Treat Ultra-Rare Diseases, which provided an in-depth analysis of this thriving market opportunity by featuring companies specializing in the ultra rare disease market alongside clinical and policy / regulatory experts in the field.
Moderated by Ellen Licking, Senior Writer & Analyst, Real Endpoints, the panel opened with a focus on the legislation and the regulatory environment. Licking pointed out that there has been a recent uptick in interest on rare diseases which has led to an increase in deal-making. With the recent passage of FDASIA, there have been new opportunities identified for expediting review and approval of rare disease drugs, including accelerated approval, expanded reviewer training and priority review voucher programs to include pediatric rare diseases.
for complete article: BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases
Last week’s 11th Annual BIO Investor Forum in San Francisco hosted a panel, Raring to Go: The Race to Treat Ultra-Rare Diseases, which provided an in-depth analysis of this thriving market opportunity by featuring companies specializing in the ultra rare disease market alongside clinical and policy / regulatory experts in the field.
Moderated by Ellen Licking, Senior Writer & Analyst, Real Endpoints, the panel opened with a focus on the legislation and the regulatory environment. Licking pointed out that there has been a recent uptick in interest on rare diseases which has led to an increase in deal-making. With the recent passage of FDASIA, there have been new opportunities identified for expediting review and approval of rare disease drugs, including accelerated approval, expanded reviewer training and priority review voucher programs to include pediatric rare diseases.
for complete article: BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases
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