ALL of the FDA EXPEDITING measures NNVC's drugs may qualify for:
and recent statistics on the FDAs greatly accelerated rate of approvals:
Absolutely! It should qualify for both and additional FDA expediting measures as well.
The FULL RANGE of FDA EXPEDITING measures:
For example some of NNVCs drugs should qualify fot the FDA's flexible clinical development program:
"Flexible Clinical Development Programs
Several of the drugs for serious diseases without satisfactory alternatives were approved on the basis of non-traditional clinical trial designs or drug development programs. For example, some of these drugs were approved on the basis of a single compelling study or studies using very small patient populations "
Note that the combination of very low toxicity and very high efficacy (both of which NNVCs drugs have exhibited) tends is exactly the combination that can get approved on the basis of a single compelling study. THAT PLUS that they study is inherently much shorter than the usual (which has to wait many months or often years for clinical effects) because influenza is a very short disease and effects are fully apparent in a few weeks -- could make for extraordinarily short clinical trials.
Cross posted from my post on the SGMO board at another website:
Note especially the chart on the bottom of page 5 which shows the US has been approving more new drugs on the world market than all other countries COMBINED in 8 of the last 14 years.
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As in FY 2011, the 35 approvals of novel drugs in FY 2012 were notable for their efficiency and timeliness. Most of the drugs were approved for U.S. patients before they were available in other countries, and the efficiency of the drug review process continued to grow. For example:
• Of the 32 novel drug approvals that FDA was able to compare to approvals in other countries, 75% (24/32) were first approved in the U.S.;
• 77% of the 35 novel drugs were approved on the first cycle of review, i.e., without the need for additional information that would lengthen the approval time; and
• 34 out of the 35 drugs (97%) met the target dates agreed to under the Prescription Drug User Fee Act (PDUFA),1 surpassing the PDUFA goal of reviewing and acting on 90% of new molecular entities (NMEs)—novel drugs—by their target dates.
FDA expedited the review and approval of over half of these new medicines by using its several review authorities for important new drugs, including Fast Track, Priority Review, and Accelerated Approval. For example, of the 12 drugs that received a Fast Track designation, 75% were approved on the first cycle of review, and of the 10 Fast Track drugs for which FDA was able to make comparisons to approvals in other countries, 100% were approved in the U.S first.
..
FDA believes that Americans should have access to safe and effective therapies as early as possible. As in FY 2011, the 35 approvals of novel drugs in FY 2012 were notable for the efficiency and timeliness of their reviews. Most were approved after a single review cycle and before their approval anywhere else in the world. FDA also continued its high rate of approving drugs on or before the target dates agreed to under the Prescription Drug User Fee Act (PDUFA). All but one of the 35 novel drugs (97%) met PDUFA target dates,2 surpassing the PDUFA goal of reviewing and acting on 90% of NMEs by their target dates.
Over the past decade, roughly half of the new active substances launched anywhere on the world market were first approved in the United States, and the percentage of first introductions in the U.S. is increasing. In 2011, 64% of new active substances were first launched in the U.S., approaching an all-time high for U.S. drug introductions.4
Looking only at the 35 NMEs that were approved in the U.S. in FY 2012, the great majority were approved earlier than in other countries.
Of the 32 novel drugs for which FDA was able to make comparisons to approvals in other countries, 24 (75%) were approved by FDA before any other regulatory
agency in the world, including the European Medicines Agency (EMA), the European Union’s drug approval authority.
Of the 33 novel drugs approved in FY 2011 that were PDUFA products, 32 (97%) met their PDUFA target dates for review. This surpassed the PDUFA goal for FY 2012 of reviewing and acting on 90% of NMEs by their target dates. (Two of the 35 novel approvals were non-PDUFA products, but were reviewed by FDA under PDUFA timeframes; both met their target dates.)
The median approval time for applications received in FY 1993 was 19.0 months, compared to 9.9 months for applications received in FY 2011 (the most recent fiscal year for which we have enough data to provide estimates5). ).
HOW FDA EXPEDITES DRUG REVIEW
FDA uses a range of tools to expedite the development, review, and approval of the most promising new therapies. These tools include Fast Track, Priority Review, and Accelerated Approval.6 Eighteen of the 35 novel drugs (51%) were reviewed under at least one of the Fast Track, Priority Review, or Accelerated Approval programs. FDA also allowed flexible clinical development programs, where appropriate, for drugs for unmet medical needs, such as for orphan
drugs. In addition, FDA is beginning to use the "Breakthrough Therapies" provision that was added to FDA’s authority this year in the Food and Drug Administration Safety and Innovation Act (FDASIA),7 but it was not available to expedite any of the drugs approved in FY 2012.
Fast Track
Fast Track, which was developed by FDA, and codified into law in 2007, is a process designed to facilitate the development and expedite the review of drugs to treat serious or life-threatening diseases that will fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious diseases, including AIDS, Alzheimer’s disease, cancer, epilepsy, and diabetes. Filling an unmet medical need is defined as providing a therapy where none exists or one that may be potentially superior to an existing therapy. [[ALL of the IVPR MGD applications should qualify : a one shot cure, 100% effective, constant protein levels (vs high variatons with other methods, etc]]
Once a drug receives Fast Track designation, FDA offers the sponsor early and frequent communications to facilitate an efficient development program. The frequency of communications ensures that questions and issues are resolved in a timely manner, often leading to earlier drug approval. Fast Track drug sponsors are also eligible for "rolling review" of applications, allowing earlier submission and initiation of review. More than a third (12/35) of the 35 drugs were given a Fast Track designation. Of the 12 drugs that received a Fast Track designation, 9 (75%) were approved in the first review cycle. Of the 10 Fast Track drugs for which FDA was able to make comparisons to approvals in other countries, 100% were approved in the U.S first.
Priority Review
In 1992, under PDUFA, FDA agreed to specific goals for improving drug review times and created a two-tiered system of review times— Priority Review and Standard Review. Priority review designation is given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.[[ALL of the IVPR MGD applications should qualify : a one shot cure, 100% effective, constant protein levels (vs high variatons with other methods, etc]]
FDA aims to review priority drugs more quickly, in six months versus 10 months for standard drugs. For example, in January 2012, FDA gave a priority review to Kalydeco, a breakthrough drug to treat patients with cystic fibrosis (CF) and who have a specific genetic defect. Kalydeco is the first medicine that targets the underlying cause of CF rather than its symptoms or complications. It was reviewed and approved by FDA in just over three months. Twelve of the 35 FY 2012 drugs received priority review. Of those 12, 11 (92%) were approved on the first cycle, and 10 (83%) were approved in the U.S. before any other country.
Accelerated Approval
The Accelerated Approval process, first created by FDA in 1992 and later codified in statute, allows approval of drugs that treat serious or life-threatening diseases and that may fill an unmet medical need, based on a surrogate endpoint that is reasonably likely to predict clinical benefit but is not fully validated to do so.
[[Because with the IVPR applicatons for the MGDs, the therapeutic protein is EXACTLY the missing protein that is the root cause of the condition, acceptance of protein level as endpoint is certain in most cases (there may be a very few exceptions where damage is cumulative and appears to be permanent)]]
In some cases, approval is based on an effect on a clinical endpoint other than survival or irreversible morbidity. A surrogate endpoint is a marker—a laboratory measurement, or physical sign—that is used in clinical trials as an indirect or substitute measurement for a clinically meaningful outcome, such as survival or symptom improvement. For example, viral load is a surrogate endpoint for approval of drugs for the treatment of HIV/AIDS. The use of a surrogate endpoint can considerably shorten the time to approval, allowing more rapid patient access to promising new treatments for serious or life-threatening diseases. Accelerated Approval is given on the condition that sponsors conduct post-marketing clinical trials to verify the anticipated clinical benefit. If these trials fail to demonstrate the anticipated benefits, approval can be revoked.
More than 80 new products have been approved under Accelerated Approval since the program was established, including 29 drugs to treat cancer, 32 to treat HIV, and 20 to treat other conditions such as pulmonary arterial hypertension, >>>>Fabry disease<<<< and transfusion-dependent anemia. Two of the 35 NMEs approved in FY 2012 were approved under Accelerated Approval.
Flexible Clinical Development Programs
Several of the drugs for serious diseases without satisfactory alternatives were approved on the basis of non-traditional clinical trial designs or drug development programs. For example,>>>>>> some of these drugs were approved on the basis of a single compelling study or studies using very small patient populations<<<<
Reliance on flexible drug development programs is of particular value in approving drugs for patients with rare diseases—those affecting fewer than 200,000 people in the U.S. Orphan drug therapies for rare diseases represent the most rapidly expanding area of drug development, yet they can be among the most difficult to study. Because of the small numbers of patients who suffer from each disease, FDA often allows non-traditional approaches to establishing safety and effectiveness.
Breakthrough Therapies
FDASIA gave FDA a new tool to expedite the development of therapies that show substantial promise in early clinical trials. This new authority arose out of discussions between FDA, the National Institutes of Health (NIH), industry, academia, and patient groups on how to create a novel pathway for development of breakthrough therapies. A drug company may seek designation of a drug as a "breakthrough therapy" if it is for a serious and life-threatening disease and preliminary clinical evidence shows the drug may offer substantial improvement over existing therapies.
.[[ALL of the IVPR MGD applications should qualify : all serious conditions, a one shot cure, 100% effective, constant protein levels (vs high variatons with other methods, etc]]
Once FDA designates a drug as a breakthrough therapy, it will provide advice and interaction throughout the development process to streamline the drug’s clinical trials and review. Under FDASIA, FDA has 18 months to issue guidance on how it plans to implement the breakthrough therapy provision, but companies may begin to seek the designation right away. The breakthrough therapy provision was not available to expedite any of the drugs approved in FY 2012.
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