In 2016 the FDA’s Center for Drug Evaluation and Research (CDER) approved a total of 22 novel drugs, some of them being of exceptional value and importance in terms of medical care advance. Processes of drug reviewal and approval also deserve to be mentioned, along with the regulatory tools CDER uses to help speed some of the most valuable drugs to the market and to the patients who are in desperate need for those solutions.
The greater part of the hundreds of applications CDER approves every year is variations – new dosage forms or cost-saving generics of previously approved products. These new variations offer a number of benefits such as bigger choice for consumers, a competitive marketplace, larger access and improved affordability.
And just a small section of the annual new approvals consists of novel drugs that are really innovative products, some of them helping advance therapy and diagnostics to an altogether new level.
The most groundbreaking CDER novel approvals of 2016 include products offering new solutions to such problems as:
- spinal muscular atrophy (first drug ever)
- Duchenne muscular dystrophy (first drug ever)
- delusions and hallucinations in some people with Parkinson’s
- primary biliary cirrhosis (rare chronic liver disease)
- hepatitis C (two new medications)
- ovarian cancer
- soft tissue sarcoma
- bladder cancer
- chronic lymphocytic leukemia
- two new diagnostic agents for detection of some cancer forms.
Along with rigorous premarket scrutiny of products in terms of meeting safety standards, CDER practices postmarket safety surveillance. This is done with the aim to observe how the approved products perform when being used by a larger population of patients.
Value and Impact of Drugs Classified as Novel
The 22 novel drugs of 2016 approved by CDER include:
- New therapeutic biologics under BLAs (Biologic License applications)
- MNEs (new molecular entities) with unique chemical structures never approved before.
The innovative products classified by CDER as novel drugs cater to previously unmet therapeutic needs, provide innovative treatment or have some other significant impact on the quality of public health and patient care.
In 2016 CDER approved a smaller number of novel drugs than it did in previous years even though the amount of applications remained almost the same.
Classified by CDER as Fist-in-Class were eight out of 22 novel drugs: Defitelio, Exondys 51, Venclexta, Xiidra, Ocaliva, Spinraza, Zinbryta, Zinplava. Mechanisms of action of such drugs often differ from those of existing ones.
Nine of the 22 novel drugs were approved to treat “orphan” or rare diseases with an incidence of 200,000 or lower among Americans. As a rule, there are few if any drugs designed to treat rare diseases. Significant medications in this area are:
Spinraza, a drug designed to treat spinal muscular atrophy
Exondys 51, designed to treat Duchenne muscular dystrophy.
Among novel drugs of 2016 there are also other remarkable products for the treatment of different cancer types, like:
- soft tissue sarcoma – a product called Lartruvo
- ovarian cancer – Rubraca
- urothelial carcinoma (a common type of bladder cancer) – Tecentriq
- chronic lymphocytic leukemia in certain patients – Venclexta.
Included in the list of novel drugs were also two diagnostic products:
- Axumin, an imaging agent helping to detect prostate cancer
- Netspot, an imaging agent helping to detect neuroendocrine tumors.
Methods Used to Expedite Novel Drugs to Market
With the aim to speed up the processes of development, approval, and introduction of novel drugs to the market, in 2016, CDER employed the following regulatory approaches.
The Fast Track approach is applied to drugs that feature the potential of addressing medical needs that no other medication has met before. This approach speeds up the processes of development and review of new drugs, say, by allowing portions of a drug application to be reviewed by CDER before submission of the complete application, or by intensifying communication of developers with FDA.
CDER processed by Fast Track approach 8 out of 22 novel drugs approved in 2016: Epclusa, Exondys 51, Anthim, Spinraza, Zinplava, Defitelio, Lartruvo, and Ocaliva.
This approach is designed to make a potential new therapy’s development time shorter. It is applied to breakthrough drugs that have evidence (clinical study results) of demonstrating improvement in at least one significant area and reaching such results in a more efficient way than any other existing medication. Seven of the 22 novel drugs were categorized by CDER to be handled as Breakthrough therapies. The advantages of this status include all benefits of the Fast Track program along with a more intensive FDA guidance throughout the development process of the given efficient drug.
Expedited with Breakthrough status were Nuplazid, Rubraca, Epclusa, Lartruvo, Tecentriq, Zepatier, and Venclexta.
The Priority Review approach implies that the drug will be reviewed within 6 rather than the standard 10 months period.
A drug becomes eligible for Priority Review in case if CDER concludes that it is capable of providing a substantial advance in medical care.
CDER designated eligible for this approach 15 of the 2016 novel drugs: Epclusa, Exondys 51, Axumin, Defitelio, Lartruvo, Netspot, Nuplazid, Tecentriq, Venclexta, Ocaliva, Rubraca, Spinraza, Xiidra, Zepatier, and Zinplava.
The method of Accelerated Approval is used to speed up the availability of medication to patients who need it. This possibility is granted to drugs for severe or life-threatening illnesses which offer a unique action or benefit(s) over existing treatments. After being evaluated eligible for Accelerated Approval, the drug is subject to additional testing to confirm claimed benefits.
CDER granted Accelerated Approval to six of the 2016 novel drugs: Exondys 51, Lartruvo, Ocaliva, Venclexta, Rubraca, and Tecentriq.
Almost all novel drugs (21 of them) were approved by CDER on the “first cycle”, with no requests of additional information leading to approval set back and undergoing another review cycle.
It is worth mentioning also that in 2016, 19 of the 22 novel drugs were approved in the U.S. earlier that in any other country.