In this slideshare, we have discussed the current approach and regulatory practices adopted by FDA with reference to drugs for rare diseases. FDA has been found to be a little flexible in review and approval of this category of drugs. FDAMap's forthcoming workshop "How to Prepare for an FDA Meeting" - http://bit.ly/1ENjEmN, to be held in Washington DC on June 4-June 5, 2015 will guide you on many such regulatory issues.

1. FDAMAP
Making Regulations Easy….
www.FDAMap.com
Email: Info@FDAMap.com
410-501-5777
20203 Goshen Rd, Suite 261
Gaithersburg, MD 20879

2. 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.

3. 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

4. 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)

5. 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

6. 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.

7. 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?

8. 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.

9. 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.

10. 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.

11. 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.

12. www.fdamap.com | 20203 Goshen Rd, Suite 261, Gaithersburg,
MD 20879
Email: info@FDAMap.com | Phone: 410-501-5777 | (C)
Copyright (2015) All Rights Reserved
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