orphan drugs

Takeaways from the NORD Rare Summit: Zebras Among Us

I look forward to the NORD Rare Summit each year.   It is a time to reflect on the work all of us do in the rare disease community−whatever area we work in, be it drug development, clinical research, regulatory compliance or patient advocacy. It’s a wonderful opportunity to collaborate as a community, truly learn from one another, and most importantly return to our work with a renewed focus and passion for advancing treatments for rare disease patients, knowing that the patient is central to all our work.

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FDA User Fee Act Is Progressing

Over the last two weeks, the Senate and the House passed their versions of the Food and Drug Administration Safety and Innovation Act. Conference committees will now work together to come up with a compromise, which could potentially be signed by President Obama this summer. The bill is nearly 400 pages long and covers such areas as user fees for novel, generic, and biosimilar medications, device regulation improvements, and drug shortage issues.

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Bigger Can Be Better

The World Orphan Drug Congress being held this week presents a unique opportunity for manufacturers, advocacy groups,  and patient access providers to meet and discuss how we can all work together to take really good care of small, special patient populations.

One interesting discussion has been about whether small or large providers can best serve the needs of orphan drug manufacturers and their patients.

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