On Tuesday, the powerful U.S. House Energy and Commerce Committee proposed an ambitious bipartisan proposal to reform major aspects of the FDA and the American drug and medical technology regulatory regimen. If it wins final passage and President Barack Obama's signature, it would become the most significant healthcare reform law in America since the Affordable Care Act.
The draft bill, presented under the 21st Century Cure initiative, would make far-reaching changes aimed at streamlining the approval of game-changing drugs, enhancing collaboration and communication between the biopharma industry and government agencies, extending exclusivity to antibiotics and other important therapeutic classes, and injecting patients' voices into the clinical development and approval process.
There's a lot to chew on in the preliminary proposal—about 400 pages' worth, in fact, and BioPharma Dive will continue to track and analyze the bill in the weeks and months to come. In the meantime, here are the biggest top-line reforms and most important things to keep in mind about the legislation—and what they could mean for the industry (you can read a one-pager of the bill here and a section-by-section summary here, or follow the discussion on Twitter with the hashtags #Cures2015 and #Path2Cures):
1. It tries to get patients involved in the clinical process
Right off the bat, the first section of the draft bill is labeled "Putting Patients First By Incorporating Their Perspective Into The Regulatory Process And Addressing Unmet Medical Needs."
Under the bill, the "FDA would be required to establish a structured framework for the meaningful incorporation of patient experience data into the regulatory decision-making process, including the assessment of desired benefits and tolerable risks associated with new treatments." Furthermore, one proposed provision would establish a process that would make it possible for the FDA to consider surrogate endpoints, which has long been a goal of rare disease advocates.
2. Proposed reforms aim to expedite drug approvals and clinical trials, enhance exclusivity
The main purpose of 21st Century Cures is to "modernize" the FDA and regulatory pathway in order to get drugs to patients and the general public faster. This is something that the biopharma industry has been calling on for years—but what, exactly, does that look like?
Mostly, it comes down to extending exclusivity and expediting breakthrough approvals. For instance, the bill puts special emphasis on antibiotic development (this is also the provision that enjoys some of the most bipartisan support) by incorporating the Antibiotic Development to Advance Patient Treatment (ADAPT) Act, which would support "greater collaboration between industry and FDA around adaptive clinical trials and labeling changes." It would also establish a transferable exclusivity program supported by the White House for antibiotics developers.
But antibiotics aren't the only therapeutic class that would win more exclusivity under the proposal. Several other provisions would grant two more years' exclusivity to new therapeutic entities that present "significant improvements to existing molecules" (meaning less adverse events, a better delivery method, etc.) and six months' extended exclusivity for orphan drug-designated products.
The draft bill would also enhance funding for the NIH's National Center for Advancing Translational Science (NCATS) in order to help the agency better perform tasks such as finding new uses for older drugs, especially for orphan diseases and unmet medical needs.
Other ambitious undertakings presented in the bill: streamlining the clinical trials process by cutting down on unnecessary bureaucratic tape and paperwork; putting more emphasis on maintaining post-approval studies; incorporating Bayesian statistics and adaptive trial designs; and much, much more.
3. There's a stab at social media marketing and communication
The industry is still fairly uneasy about social media use. And for good reason—the regulatory landscape is still rather murky.
21st Century Cures makes a stab at addressing this uncertainty, promising to clarify FDA social media guidance. For instance, must information from a hyperlink contained in a tweet be considered part of the original communication (yes, it does)?
The committee is also reportedly crafting a proposal to "clarify and rationalize" outdated rules on how the industry and physicians can communicate information about their own products.
4. The politics are tricky—but not impossible
There's bound to be controversy and partisan sniping when it comes to crafting reform of this magnitude. And the stakes are even higher considering that the legislation would directly affect the health and safety of millions of Americans, including some of the most sick and vulnerable people in the country.
However, 21st Century Cures (or at least many of its major provisions) is the rare initiative that has enjoyed bipartisan support in an ever-gridlocked Congress. As the draft summary takes pains to note, the reforms proposed in the bill come from top Democrats and Republicans alike, including Sens. Michael Bennett (D-CO) and Orrin Hatch (R-UT), Rep. Joe Pitts (R-PA), Rep. Marsha Blackburn (R-TN), Rep. Diana DeGette (D-CO), and even the White House.
But an ambitious mishmash of ideas, bipartisan or not, does little to ensure comity or final passage in today's tumultuous legislative landscape. Last minute hang-ups over controversial provisions, or the specifics of the bill's funding mechanisms, could easily derail the whole effort (see: every year that Congress has failed to pass a permanent "doc fix").
And then there's the fact that Congress is, for the first time during Barack Obama's presidency, now fully controlled by Republicans. That means that Senate health committee leadership has flipped from Democrats to the GOP, and Republican staffers are the ones who get to write the actual language of the bill. This could lead to some chest-thumping and scuffles over turf (not to mention legislative minutiae).
But for now, the biotech, pharmaceutical, and life sciences industry has a lot to consider.