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FDAMAPMaking Regulations Easy….
www.FDAMap.comEmail: [email protected]
410-501-577720203 Goshen Rd, Suite 261
Gaithersburg, MD 20879
AS A REGULATOR, FDA’S CURRENT TAKE ON NEW DRUGS INTENDED FOR USE IN RARE DISEASES IS AFFECTING SMALL POPULATIONS OF PATIENTS.
There is a law in the US that supports and encourages the development of new drugs and therapies for patients with rare diseases. To protect these drugs from the competition from generic drugs, the new product gets seven-year period of marketing exclusivity.
DO YOU KNOW THAT THERE ARE OTHER INCENTIVES AS WELL FOR NEWLY DEVELOPED DRUGS FOR RARE DISEASES?
Yes, the other incentives include: user fee waivers tax credits and grants
FORTUNATELY, THESE INCENTIVES COME AS A BREEZE OF FRESH AIR, AS THERE HAS BEEN A SERIOUS LACK OF INVESTMENT IN THIS FIELD SO FAR!
Therefore, the Orphan Drug Act, has remarkably changed the scenario and increased the number of orphan drug approvals per annum.
Orphan drugs approved in 1983 (Just two)
Orphan drugs approved in 2014 (Around 50)
BUT AS FAR AS TRUTH IS CONCERNED, WE NEED TO REALIZE THAT
95% out of the roughly 7,000 rare diseases have currently no proper approved treatments
BUT, DO YOU THINK THAT IF A DRUG IS DESIGNATED AS AN ORPHAN DRUG, IT WOULD LEAD TO AUTOMATIC AND QUICK APPROVAL BY THE FDA?
No, simply being designated as an orphan drug doesn’t warrant FDA to approve it quickly or without any concrete evidence. So, we can safely say that the standard of approval for orphan drugs is technically and legally the same as that of the other drugs.
As a standard rule, to get a new drug approved, there must be 'substantial evidence' of its effectiveness. And it
requires well-controlled and 'adequate’ investigations, according to FDA.
SO, HOW DOES FDA APPROVE ORPHAN DRUGS?
SO WHAT IS THIS FLEXIBILITY ALL ABOUT?
It has been noticed that FDA has shown considerable flexibility while dealing with orphan drug products in its regulatory actions.
CAN YOU PROVE IT WITH SOME RELEVANT
STATS?
According to an industry survey, out of 27 orphan drugs approved by FDA from the year 2010 and 2014, only eight drugs approved by FDA were subjected to general standards of evidence to gain approval. The rest were approved implementing “a flexible approach”, as far regulatory practices are concerned.
FDAMAP WILL TRAIN YOU ON HOW TO PREPARE FOR MEETINGS WITH THE FDA
FOR DRUG DEVELOPMENT AND THE BEST PRACTICES INCLUDING DO’S AND
DON’TS, ETC.
AT FDAMAP, YOU GET A TRAINER, WHO IS WELL VERSED WITH REGULATORY AFFAIRS AND HAS RELEVANT EXPERIENCE DEALING WITH THE FDA AND GETTING PRODUCT APPROVALS.
FDAMap has expert trainers and speakers working for it. These are highly experienced individuals, who are well-known in the pharma industry and have years of international experience in providing regulatory consulting services and training.
www.fdamap.com | 20203 Goshen Rd, Suite 261, Gaithersburg, MD 20879
Email: [email protected] | Phone: 410-501-5777 | (C) Copyright (2015) All Rights Reserved
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