BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases

from BIOtechNow by biotechnow@bio.org (Biotechnology Industry Organization)

 

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

Cancer Stem Cell Therapy: Real Or Just Hype?

Cancer Stem Cell Therapy: Real Or Just Hype?

from BIOtechNow by biotechnow@bio.org (Biotechnology Industry Organization)

Also known as tumor-initiating cells, cancer stem cells (CSC) have unique capability endemic to stem cells of self-renewal and differentiation, thereby causing tumor relapse and/or metastatis. While traditional cancer treatments focus on tumor shrinkage, it’s a distinct possibility that these agents do not effectively destroy all of the CSCs, causing the tumor to reappear.

The CSC theory is relatively new, so the treatment pipeline is still in its early stages.

Nathan Sadeghi-Nejad, a contributor to Forbes and TheStreet, moderated a panel of scientific experts at the forefront of developing treatments targeting CSCs during the 2012 BIO Investor Forum. In an article for Forbes, he provides his thoughts on how CSCs will affect oncology drug development:
“Bulls believe CSC-based therapies will revolutionize oncology drug development and dramatically improve patient outcomes. Bears argue the data are inconclusive or that expectations are unreasonably high. I think the truth lies somewhere in the middle…”

BIO Testifies on Importance of Timely Passage of BsUFA and PDUFA

BIO Testifies on Importance of Timely Passage of BsUFA and PDUFA

Washington, D.C. (March 29, 2012) – Sara Radcliffe, Executive Vice President of Health at the Biotechnology Industry Organization (BIO), testified today on biosimilar user fees (BsUFA) and the Prescription Drug User Fee Act (PDUFA) before the Senate Health, Education, Labor and Pensions Committee in a hearing entitled, “FDA User Fee Agreements: Strengthening FDA and the Medical Products Industry for the Benefit of Patients.”

In her testimony, Radcliffe expressed support for the establishment of a biosimilars user fee program as part of the Food and Drug Administration’s (FDA) implementation of a well-constructed, science-based pathway for the approval of biosimilar products that protects patient safety and preserves incentives for innovative new therapies.

“A transparent, predictable, and balanced regulatory framework for the review and approval of biosimilars, accompanied by reasonable performance goals and a dedicated, independent funding stream, will ensure that FDA can facilitate the development and evaluation of biosimilars products,” Radcliffe testified.

click link for full article: BIO Testifies on Importance of Timely Passage of BsUFA and PDUFA