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The FDA, in an effort to bring promising new therapies to patients as quickly as possible, has introduced a spate of shortcuts to speed up the approval process. Those programs are working as intended, new research finds, but drug companies are often loath to fulfill their obligations.

The big idea behind the FDA’s accelerated drug approval program is that regulators will OK a promising drug based on clues that it will improve patient lives, so long as pharma companies later carry out larger trials to confirm those hints of efficacy. But looking at four years of data, a team of researchers found that only 50 percent of those trials actually took place within three years of approval.

Furthermore, 44 percent of such trials were not the placebo-controlled variety considered to be the gold standard but rather relied on the same surrogate measures used to win a quick approval, leaving each drug’s true value unclear. This was particularly striking for cancer drugs, accounting for 80 percent of studied approvals, which were cleared based on how well they shrank tumors, not how long they kept patients alive.

Further Reading:

• “GAO Report: FDA Expedites Drug Approvals, But Its Postapproval Oversight Stinks!”; http://sco.lt/89j3Zp
• "FDA is Lax in Enforcing Law Regarding Prescription Drug Postmarketing Studies"; http://bit.ly/1PoqAsY

The number of new drugs approved for sale in United States and Europe has bounced back this year [so far], suggesting a marked slowdown in 2016 was an aberration rather than a sign of flagging research and development productivity.

The U.S. Food and Drug Administration has already cleared 21 new prescription medicines for sale against 22 in the whole of 2016 (read “New Drugs Approved by FDA in 2016 is Half the Number Approved in 2015”; http://sco.lt/55JaUb), and just nine at this stage last year.

The European Medicines Agency has recommended 42 compared with a 2016 total of 81, and 31 in the first five months of last year. Unlike the FDA, the EMA includes generic or non-patented drugs in its list.

John LaMattina, a former research head for Pfizer and a board member at PureTech Health, is unsurprised by the rebound and believes concerns raised at the end of 2016 about deteriorating drug pipelines were "far too dire".

Hilary Thomas, chief medical adviser at KPMG, said U.S. regulators in particular were showing an innovative approach that was helping to accelerate approvals - as highlighted by a novel decision to clear a cancer drug for the first time based on genetics, not tumor location.

Still, the targeted nature of many new medicines will limit the overall patient population getting the latest wave of novel drugs.

"What the data masks is that while there might be more approvals, the total number of people getting new drugs is probably not going to up hugely because these are more specific, personalized treatments," she said.

The global pharmaceuticals industry is set to win the lowest annual number of new drug approvals this year since 2010 (read “New Drugs Approved by FDA in 2016 is Only 60% the Number Approved by This Time in 2015”; http://sco.lt/9CtktV) and a new report on Tuesday suggests drugmakers' returns on research investment are deteriorating (read “Consultants Advise Pharma to Act More Like Biotech to Revive ROI for New Drugs”; http://sco.lt/8qLTCT).

Only 19 new drugs have been approved in the key U.S. market so far in 2016 and, with less than three weeks to go, it is clear the full-year tally will be well down on 2015 and 2014's bumper haul of 45 and 41 new products respectively.

At the same time the profitability of drug research is being squeezed by steadily rising costs and increasing political pressure over the high prices of many modern medicines.

As a result projected returns on investment in research and development (R&D) for the top 12 pharmaceutical companies have fallen to just 3.7 percent this year from a high of 10.1 percent in 2010, according to consultancy Deloitte.

Five years of exclusivity is provided for drugs which do not contain active molecules that have appeared in previously-approved drugs.

This is one of the longest exclusivity terms provided by the FDA. Only orphan drugs receive a longer term of exclusivity, although the five years granted under the Generating Antibiotic Incentives Now (GAIN) Act may be added in addition to other exclusivities, giving it an effetively longer span.

This chart shows the companies which have received the most new chemical entity exclusivities in the past five years.

Since 2009, drug review time has steadily declined, with particularly fast review times seen in the therapeutic areas of oncology, infectious, and rare diseases. At the same time, the data suggests that, despite consistent decreases in overall average approval times, therapeutic areas such as endocrine, central nervous system and gastrointestinal diseases still have room for improvement. Increased approval times can also be attributed the new 'Breakthrough Therapy Designation' pathway, established as part of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA), which offers 60-day review timelines for qualifying product submissions. 

Key facts and infographic highlights:

• Following a dramatic slowdown, average FDA review times have steadily improved since 2009, dropping from 21 months to 10 months
• Oncology and immunomodulatory (immunomod) and anti-infective products have seen the strongest consistent review performance. Endocrine, gastrointestinal, central nervous system and musculoskeletal products continue to see the most consistent lag relative to the overall average.
• 2015 marked an increase in the diversity of breakthrough approvals since the inception of the 'Breakthrough Therapy Designation.' Historically, breakthrough therapies have been concentrated in oncology, rare diseases and infectious conditions.