Advocating for the Rare Disease Community

11.07.13 | By

Have you heard of  Batten disease? Or  of Pompe disease? Niemann-Pick Disease? These are just a few of the 7,000 rare diseases that over 30 million Americans live with every day. Another rare disease that you may not be familiar with is Mitochondrial disease. Unfortunately, Art Estopian is too familiar with the disease. Art’s son, Arturo was diagnosed with thymidine kinase 2, mitochondrial disease, an inherited condition that causes progressive muscle weakness.

Sharing Takeaways from Alzheimer's Thought Leaders

11.05.13 | By

Today, Alzheimer’s affects more than five million people in America. According to the Alzheimer’s Association, that number will triple in the next 40 years, and by 2050, caring for patients with Alzheimer’s and other forms of dementia is expected to cost the United States more than $1 trillion annually. Given the tremendous looming human and financial costs, more and more attention and resources are being devoted to finding new ways to treat those with Alzheimer’s disease and, ultimately, find a cure.

Evolution of Comparative Effectiveness Research

11.05.13 | By

Last week, I took part in an interesting multi-stakeholder roundtable convened by NEHI and the California Healthcare Institute (CHI).

Conversations Wrap Up: Pharmaceutical Supply Chain

11.05.13 | By

The U.S. pharmaceutical supply chain is the most secure in the world in part because of strong industry and government collaboration.  One benefit is that American patients can have confidence that the prescription medications they purchase from a reputable pharmacy are true to the label.

Genetic Alliance & PhRMA Announce Initiative for Patient-Focused Drug Development

Washington, D.C. (November 4, 2013) — Today, Genetic Alliance and the Pharmaceutical Research and Manufacturers of America (PhRMA) announced an initiative to explore the use of a technology-enabled, crowd-sourcing approach to patient engagement as a complement to ongoing patient-focused drug development efforts under the Prescription Drug User Fee Act (PDUFA V).

Insights and Innovation From the Personalized Medicine Community

11.04.13 | By

Personalized Medicine was an emerging field of medicine in 2005 when we first held what was to become an annual event, our personalized medicine conference. The importance of personalized medicine was given a boost by the then U.S. Secretary of Health and Human Services, Michael Leavitt, who said in January 2005, “I believe we are moving into a remarkable and powerful new era in medicine and particularly in prescription drugs.

Expanding Knowledge to Enhance Patient Wellness

11.02.13 | By

The old adage that 'you learn something new every day' doesn’t apply to the biopharmaceutical industry; for those researching and developing new medicine, it would be more accurate to say you learn something new every few minutes. Our member companies continue to push the limits of research, and that process keeps our work consistently exciting.

Use of SI Units in Regulatory Submission

Washington, D.C. (October 31, 2013) — Pharmaceutical Research and Manufacturers of America (PhRMA) Vice President of Scientific and Regulatory Affairs Sascha Haverfield, PhD, issued the following statement on the recent announcement by the Food and Drug Administration (FDA) regarding the use of Systeme International (SI) units in regulatory submissions:

From moving trucks to hitting “send”, biopharmaceutical companies need a stable and reliable system to submit New Drug Applications

11.01.13 | By

Less than 15 years ago, submitting an application to the Food and Drug Administration (FDA) for the approval of a new medicine generally involved loading docks, moving trucks, and enough paper to obliterate a small tree farm.  Today, those data once supplied on hundreds of thousands of paper pages are now a structured collection of electronic files submitted to the FDA through a secure portal. 

Fostering Opportunities in the Transatlantic Trade and Investment Partnership

10.31.13 | By

Yesterday, the Senate Finance Committee held its first hearing on the Transatlantic Trade and Investment Partnership (TTIP), a comprehensive free trade agreement that the U.S. and European Union have begun negotiating. America’s research-based biopharmaceutical companies believe that the TTIP negotiations provide an important opportunity to spur a new era of biomedical innovation in countries that are already global leaders on this front.


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