Addressing Challenges in Rare Disease Drug Development - Together

01.27.14 | By

Dr. Richard Moscicki, Deputy Center Director for Science Operations at the Center for Drug Evaluation and Research at the Food and Drug Administration (FDA), noted in his remarks at the FDA public workshop on Complex Issues in Developing Drug and Biological Products for Rare Diseases held earlier this month that more than a third of the novel drugs approved in CY13 treat rare disease.

Several Important Pieces of the R&D Puzzle

01.17.14 | By

Like a complex puzzle, the research and development (R&D) process for new medicines requires many pieces to work concurrently to ensure success. From the patients who inspire us to the experts behind the research, every part is critical to a prosperous pipeline for patients suffering from some of the world’s most debilitating diseases.

A New Window Into the Lives of Biopharmaceutical Scientists

01.17.14 | By

As we continue our discussion of STEM education this week, we’re providing a window into the daily lives of some of our member companies’ biopharmaceutical researchers and getting their advice for future STEM educated individuals in our “I am Research, Progress, Hope” series.

Rare Disease Community Loses a Champion

01.15.14 | By

The rare disease community lost a champion last week when Sam Berns passed away at age 17 due to complications from progeria, an extremely rare genetic disease which causes accelerated aging. Last October, Sam shared his inspiring philosophy for a happy life in his talk at TEDxMidAtlantic.

PhRMA Statement on Release of Sequestered FY2013 PDUFA Fees in Omnibus Spending Package

WASHINGTON, D.C. (January 14, 2014) – Pharmaceutical Research and Manufacturers of America (PhRMA) Vice President of Scientific and Regulatory Affairs Sascha Haverfield, PhD, issued the following statement on the release of sequestered prescription drug user fees included in the omnibus spending package filed yesterday:

Understanding Patient Perspective Critical in Benefit-Risk Assessment

01.06.14 | By

The assessment of the benefit-risk balance of a medicine is a key component of the drug development and regulatory review process. This assessment is also an important determination that each patient and doctor must make when choosing a treatment strategy.

Upholding Ethical Standards

12.28.13 | By

The best health stems from the best information, and this includes biopharmaceutical information-sharing with physicians and patients. As the creators of many of the medicines we all rely upon, the companies have the most up to date knowledge on safety and benefit/risk, making conversations with prescribing physicians imperative to better care.

TPP and Intellectual Property

12.20.13 | By

What provides two-thirds of the Trans-Pacific Partnership (TPP) agreement’s total economic benefits? The answer is intellectual property-intensive manufacturing industries.

Cost of Cancer Medicines

12.19.13 | By

Rather than trying to uncover what’s at play behind a woman’s struggle to pay for the costs of her leukemia medicine, last night’s ABC World News with Diane Sawyer barely scratched the surface of a complex issue. The result: a one-sided, overly simplistic view of the cost of cancer medicines in the United States.

A Conversation on Healthcare in 2013

12.17.13 | By

When we launched Conversations in June of this year, our goal was to blend formal contributors with an ongoing opportunity for experts and thought leaders to weigh in on a broad range of high-level policy topics. The forum has been a tremendous success. So far this year, we have seen 19 unique questions with over 90 responses from a variety of thought leaders and stakeholders, both inside and outside of the healthcare space.

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