Biomedical reform legislation is third on the Senate HELP Committee’s priorities for this Congress, but given the “fast-moving train,” the chamber’s leadership appears more interested in zeroing in on a few key transformations rather than producing the kind of “kitchen sink” reform the House Energy and Commerce Committee seems to be pursuing.

Unfortunately for FDA Commissioner Margaret Hamburg and NIH Director Francis Collins, the priorities they listed for the Health, Education, Labor and Pensions Committee may not be the most politically viable options.

They are mostly pleading for more government funding, while in the House, explicitly de-regulating industry through faster product approval paradigms has been a major focus of draft legislation ([A#00150202012]).

During a March 10 Senate hearing, though, Hamburg worked to build a case that a stronger regulatory agency actually aids and speeds innovation.

HELP Committee Chair Lamar Alexander, R-Tenn., said HELP is working “to reduce the cost and the amount of time it takes to go from discovery of a medicine or treatment or a medical device and take it all the way through to the medicine cabinet or doctor’s office.”

The goal is number three on the list of priorities he set with Ranking Member Patty Murray, D-Wash., behind education reform for both primary and higher education.

Alexander said the biomedical reform effort “ought to succeed” because it is non-partisan, adding that the House of Representatives is on a parallel track and President Barack Obama is working on a precision medicine initiative ([A#00150209016]).

The scope will be broader than FDA and speeding up the agency’s reviews. “Sometimes it takes two, four, six years to get to the FDA’s front door,” Alexander noted.

But Alexander’s approach to biomedical reform doesn’t seem to be the deep dive that Rep. Fred Upton, R-Mich., has taken in leading the House’s effort to modernize and speed biomedical innovation. Upton conducted more than six months of information gathering sessions from a broad array of stakeholders before releasing a nearly 400-page discussion document ([A#00150202014]).

“We don’t want to waste our time. We can’t do everything,” Alexander said. “So if this train is moving through the station in the next 12 months and if our goal is to help get from discovery to the medicine to the doctor’s office, what are the two or three things that we ought to spend our time on?”

This is chance for NIH and FDA “to let us help you get the obstacles out of the way” that may be hindering the agencies from getting their jobs done, he added. “Some of them relate to money … some relate just to the pile-up of administrative regulations,” Alexander said.

Senate Bill Timeline Behind House

Some of Alexander’s desire to home in on key initiatives rapidly may be due to the fact that the Senate is playing a bit of catch-up to the House’s 21^st Century Cures, which got almost a year’s jump on the Senate effort, and that Alexander seems to be trying to stick close to Upton’s legislative timeline Also see "Congress Dives Into Biomedical Innovation: Will It Drown In A Deep Sea Of Competing Needs?" - Pink Sheet, 12 May, 2014..

The Senate HELP committee issued a white paper in late January to kick off its biomedical reform effort and announced the formation of a working group in early February. It is aiming to produce a bill by the end of the year. But even that pace could put the brakes on some of Upton’s momentum in the House.

Upton, who is term limited as Energy & Commerce’s Chair, is pushing for biomedical reform before the next FDA user fee reauthorization bill in 2017 when he would no longer hold the gavel. Upton’s goal is to have his legislation through the House by Memorial Day and to the President’s desk early next year.

For now, stakeholder feedback on the House discussion document continues to roll in (see related story,[A#00150316016]).

“As a former governor, Alexander thinks it is useful to tell people to focus on specific and precise priorities to drive the conversation forward. Since this is just the start of the process, it’s premature to determine what the scope of the legislation will be,” an Alexander spokesperson said in response to a question about the chairman’s more targeted focus compared to the 21^st Century Cures Initiative.

Real-World Observational Data Eyed By FDA

Greater investment in regulatory science and FDA’s ability to engage with companies earlier in the research and development process are “two critical and related needs,” that would help advance FDA’s activities and support biomedical research and product development, Hamburg told Alexander.

Hamburg has long championed regulatory science as a way for FDA to develop the knowledge tools and approaches it needs to assess product efficacy, safety, quality and performance, as she told Alexander. Getting Congressional funding has been a challenge, though the 2012 FDA and Safety and Innovation Act did provide some user fee funding for the efforts Also see "FDA Has Regulatory Science Plan; Now It Needs Funding" - Pink Sheet, 6 October, 2010..

“It’s been an underappreciated, underdeveloped and underinvested area of our overall biomedical product enterprise, and it’s proving to be really essential as we are trying to take that last set of steps from research and development into a product that will really make a difference in people’s lives,” Hamburg told the HELP Committee.

FDA told “The Pink Sheet” it needs greater investments in translational science, noting that the scientific community struggles with adequate treatments and cures for certain diseases like Alzheimer’s or multiple sclerosis because it does not yet understand how the diseases work, making it difficult to find the right drug target and treatment.

FDA said investments in regulatory science should include funding for precision medicine and methodologies to better leverage real-world observational data, as well as public-private partnerships on complex issues such as evidentiary standards for biomarkers.

‘Not Much’ Congress Can Do On Industry Engagement

Hamburg said it’s important for FDA to “be able to engage in a consistent way earlier as research plans are being shaped so that the right studies are done so that the return on investments of monies in research all along the way are really driving towards a product that works.”

The “breakthrough” therapy program, for example, has been very successful, but it did not come with additional resources, Hamburg noted, and while FDA wants to continue the program, “it does come with cost.”

Early engagement, especially in the pre-IND stage can take critical time and cost off product development because FDA “can really say ‘you don’t need to do that study,’ ‘do this study,’ ‘use this approach because it will get to the answers that will really make a difference in our approval process,’” Hamburg said.

“I think that’s been enormously exciting but it does signal changes for the future in how FDA organizes itself and how we work with the broader research and industry community,” she said.

However, in follow-up after the hearing, FDA acknowledged there is “not much” its needs on these two areas from a legislative viewpoint, noting for example that an important outcome of FDA and industry user fees negotiations has been FDA’s “growing ability to provide considerable expertise in clinical trial design to aid drug developers,” through meetings that occur in the pre-investigational new drug (pre-IND) stage to later times in the drug development process. Users fees have helped provide the additional resources to make this interaction possible.

FDA research has found that sponsors who communicate early and frequently with FDA can significantly reduce their overall drug development times, the agency noted Also see "Many Drug Approval Delays, Denials Could Be Prevented, FDA Study Shows" - Pink Sheet, 21 January, 2014..

FDA said Hamburg’s statement about early engagement was also meant to highlight the value of the entire biomedical ecosystem being engaged and communicating throughout the entire product development process including patients and payers.

As for regulatory science, FDA also said current laws provide a lot of flexibility.

FDA Isn’t The ‘Principal Obstacle’ – Hamburg

The fact that Hamburg chose to highlight two needs that FDA says don’t require additional legislative authority reinforced the argument she laid out in her opening statement – that FDA is not the “principal obstacle to the development of innovative treatment,” whose “authorities and procedures must be fundamentally restricted.”

Hamburg cautioned against solutions that would lower the safety and effectiveness barriers for approval of medical products, arguing that lowering the bar won’t create the type of innovation that will save lives and money.

“Unless we correctly diagnose why cures are still lacking for many diseases, we are unlikely to find the solutions that actually deliver those cures,” Hamburg told the HELP Committee.

She refuted three misconceptions: that FDA’s approval of new drugs lags behind other countries; that FDA is rigid and inflexible in its approach to requesting and using data for approval of new drugs; and that FDA is to blame for a drop in biotechnology investment in the U.S.

According to the National Venture Capital Association, biotechnology investment dollars rose 29% in 2014 to $6 billion, Hamburg said, in large part because of the “improved regulatory climate in recent years at FDA,” she argued.

Hamburg insisted that regulations can actually help spur medical progress.

“Remember the great leaps forward of evidence-based medicine of the last 50 years have come in part because of the high standards for product approval Congress put in place after a series of disaster involving unsafe and ineffective medical products. Those standards have also boosted the confidence that Americans placed in medical products, that the world places in the American biomedical product industry,” she said.

Lowering approval standards, Hamburg said later in the meeting, would be bad for the “preeminence” of the pharmaceutical, biotechnology and medical devices industries “in terms of their ability to actually deliver products to people who need them.”

NIH Needs Flexibility On Travel, Grants – Collins

NIH Director Collins told Alexander he had a “long list” of requests but said if Alexander wanted him to name two, his first request was to lessen the burdensome oversight of travel for scientists at both FDA and NIH.

While it “sounds pedestrian” Collins said the burdens placed on NIH and FDA scientists due to “misadventures by other agencies, who convene conferences in places like Las Vegas” in reference to a General Services Administrative administration conference that caused tighter restrictions on federal employees’ travel, has cost NIH about $16 million and the time of hundreds of employees to oversee a process “which as far as I can see has relatively little if any added value.”

“Scientists going to conferences is a critical part of how we move forward, how new ideas emerge and it is very much being inhibited by this very heavy-handed oversight, and we could much benefit from your help.”

Sequestration has also hampered FDA travel Also see "Sequester Travel: Halved FDA Budget Limits Opportunities For Scientists, Impacts Senior Officials Too" - Pink Sheet, 16 September, 2013..

Collins also asked that NIH be allowed to carry over appropriations money it does not spend in a given fiscal year so it could be “more flexible with taxpayer money,” without costing another dime. He referenced the Department of Energy which has a policy in place that allows for flexibility.

NIH, in contrast, has to obligate – or award through a grant or contract – most of its appropriations in any given fiscal year, that year, or forfeit that money.

Partisan Divide On Funding?

Requests for more funding by Hamburg and Collins spurred some partisan tensions.

Like the House 21^st Century Cures Initiative, the Senate’s biomedical reform effort has been billed as bipartisan. Yet as with the House, some political and ideological differences emerged at the HELP committee’s hearing.

Democratic senators such as Murray, Elizabeth Warren, Mass.; Robert Casey, Pa.; and Barbara Mikulski, Md., made supportive comments regarding the importance of adequate resources so FDA and NIH can do their jobs and sympathized with the unpredictability and lack of certainty caused at both agencies by the sequester.

“Nearly everyone in Congress says they support funding [NIH], Warren said, “but talk is cheap and Congress has decimated the NIH’s budget singlehandedly choking off support for projects that could lead to the next major breakthrough in Alzheimer’s and many other diseases.”

While Republican senators were less overt about opposition to funding increases than some of their counterparts on the House side had been in early 21^st Century Cures meetings, they were often critical of funding decisions.

For instance, Bill Cassidy, La., suggested NIH was spending too much time funding PhDs and not enough time funding MDs/PhDs who do translational research and he suggested NIH needs to reprioritize its budget as some diseases get better treatment, noting that HIV and Alzheimer’s comprise about the same proportion of NIH’s budget despite there being many successful treatments for HIV and few options for Alzheimer’s patients.

Alexander, who also sits on the appropriations committee, said he will be working on the funding cuts due to sequestration but added that there is the question of whether currently allocated funding is being used in the best way possibly, making reference to a claim that NIH investigators are spending 42% of their time on administrative tasks. “There is the question of reallocation,” Alexander said.

Alexander also indicated the Hamburg, who will be leaving FDA at the end of March, isn’t off the hook for the biomedical reform effort, saying the Senate HELP Committee will be back in touch with her despite her departure.

FDA’s Top Two Reform Priorities Aren’t Really Legislative Material

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