2016 in Review for Drug and Device Regulations: An 11th Hour Reckoning
Posted 20 December 2016 | By
Turn back the clock to late October and those first 10 months of 2016 look like a relatively commonplace year for the pharma and medical device industries and their US regulatory counterpart, the US Food and Drug Administration (FDA) (on the EU side, Brexit was already causing headaches for the European Medicines Agency (EMA) as early as June).
On the positive side:
- Agreements on the reauthorizations for each of the user fee bills that will help fund FDA for the next five years (GDUFA, PDUFA, MDUFA and BsUFA) made headway
- A steady stream of draft and final guidance was released (labeling, generic drug labeling, data integrity and manufacturing, compassionate use, compounding, REMS, contract manufacturing agreements, software as a medical device, device reporting requirements, 510(k) modifications, co-developing in vitro companion diagnostics and therapeutics, collecting race and ethnicity data for trials, adaptive designs for device trials and next generation sequencing, among others)
- More high-profile rulemakings (combination products, citizen petitions, prioritizing generic drug applications for sole-source products, pharmaceutical patents and drug compounding) despite a shortage of agency staff
- Continued reductions of the generic drug application backlog, which Janet Woodcock, director of the Center for Drug Evaluation and Research, explained to senators in January and later in July
- Three new biosimilar approvals (for Pfizer, Amgen and Sandoz) (read a new Focus explainer on biosimilars here)
- New collaborations between FDA and EMA on rare diseases and patient engagement (and a possible deal in early 2017 to mutually recognize good manufacturing practice inspections and increase harmonization with other regulators, as well as other work to promote the development of new antibacterials)
- New ways to further engage with patients
- A new FDA database to track the safety of new immunotherapy products making their way to market
On the downside: The new drug approval rate was off significantly from a year earlier (though some of the 2015 approvals came earlier than expected and the number of complete response letters (CRLs) due to manufacturing issues increased), widespread disagreement over the approval in September of Sarepta’s Exondys 51 (eteplirsen) creating friction at the agency with questions about what kind of precedent the approval might set, a spike in the number of CRLs for generic drugmakers, delayed regulations for lab-developed tests, biosimilar interchangeability guidance, generic drug labeling and off-label promotion.
And between 8 November and now, that landscape has shifted dramatically.
A mammoth, nearly 1,000-page 21st Century Cures bill, which lingered early in the year as a series of Senate bills on top of the House-passed version from 2015, then stalled as recently as July to the brink of collapse (over how it would be paid for and questions on mandatory funding for the National Institutes of Health). In late November, the bill was suddenly revived, quickly pulled through with major bipartisan and lobbying support and signed by President Barack Obama in the lame duck session.
Advocates hail the new law as a modernization of FDA’s approval process (new efforts linked to real world evidence, which FDA also addressed in March, and patient engagement are included in the bill), with new funding to fight the opioid epidemic, FDA (and much-needed changes to the agency’s ability to hire talent) and NIH. Critics, however, say the law is nothing more than an early Christmas gift for the pharma, biotech and device industries, softening the approval regulations to a point unseen since before the Kefauver-Harris amendments were enacted in 1962, though many of these concerns will play out as the bill is implemented in 2017 and beyond.
Meanwhile, President-elect Donald Trump has yet to offer his choice for FDA commissioner, though further agency reforms beyond this new law and some kind of solution or plan to curb rampant pharmaceutical price gouging feel inevitable, even as questions on how, why and what will change are still up in the air.
Brexit and the EU
Uncertainty in the US is also being mirrored across the pond, and 2017 will likely prove to be a crucial year in determining the direction of both regions.
In June, the UK voted to leave the EU, meaning the EMA could lose valuable British resources and manpower (and vice versa), and almost certainly lose the EMA’s headquarters in London while creating a climate of uncertainty.
On the bright side, the EU finally reached an agreement on updates to the overarching regulations for medical devices and in vitro diagnostics that haven’t been updated since the 1990s. The new regulations are likely to enter into force next summer (expect more to come from Focus and RAPS on these regulations and their implications in 2017).
In addition, the EU regulator set a high bar for other regulators around the world on transparency, with the creation of a new online database of clinical trial data. And following the death of a patient in France in a Phase I trial in February, EMA revised its 2007 guidance on first-in-human trials.