House Subcommittee Presses Forward on 21st Century Cures

WASHINGTON — Roughly 95% of rare diseases have no FDA-approved treatment. Congress hopes that by equipping government agencies with better resources, it can accelerate innovation and bring cures to patients faster.

The latest draft of the 21st Century Cures bill would grant $10 billion in mandatory funding to the National Institutes of Health beginning in fiscal year 2016.

“The research committee is ecstatic to see this new provision in the bill, and we are deeply appreciative,” said Kathy Hudson, PhD, deputy director for Science, Outreach, and Policy at the NIH.

The House Energy and Commerce’s Subcommittee on Health invited top brass from the NIH and the FDA to offer their perspectives of the revised legislation at a hearing on Thursday.

Full committee chairman Fred Upton (R-Mich.) and ranking member of the Oversight and Investigations subcommittee Rep. Diana DeGette (D-Colo.) launched the 21’s Century Cure initiative a year ago.

Full committee ranking member Frank Pallone Jr. (D-N.J.), health subcommittee chairman Joe Pitts (R-Pa.), and health subcommittee ranking member Gene Green (D-Texas) joined with Upton and DeGette to release the bill’s second draft.

The five-member bipartisan team noted in a press release, “We’ve done things differently with 21st Century Cures, taking our time to listen and solicit feedback from every corner of the healthcare innovation infrastructure.” They pointed to the 24 roundtable discussions across the country, eight hearings, and several white papers that preceded the hearing.

Rep. Joseph Pitts, (R-Pa.), chairman of the Subcommittee on Health, said, “While increasing accountability, this legislation would invest in the basic research so critical to equipping our nation’s best and brightest with the tools they need to discover the underpinnings of disease.”

In addition to funding NIH, provisions of the bill also support efforts to:

  • Speed development for treatment of life-threatening illness
  • Repurpose drugs found ineffective for one condition and test them in another
  • Develop partnerships among patients, providers, and researchers
  • Promote an interoperable health system
  • Enhance telehealth practices
  • Advance the development of more targeted personalized treatments

Other provisions in the bill explored ways to bring patients’ perspectives into the research process.

Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research, spoke of 20 “facilitated discussions” with patients around the burden of their disease and the effects of treatment. Research teams documented the conversations in a report called “The Voice of the Patient” and on occasion issued guidance based on patient feedback.

“What we learned is we need a much more structured and organized way to incorporate this input into drug development, and we think that what’s laid out in the discussion drafts will really help with that,” Woodcock said.

Other representatives spoke about the importance of having interoperability among electronic health records. Rep. Michael Burgess ( R-Texas) was frustrated that $28 billion had been sunk into trying to make electronic health records talk to each other, but “health data continues to be fragmented.”

Rep. Pitts added, “Imagine a world where your cellphone would not work with a land-line or if my cellphone did not connect with other networks. Ridiculous. Well, that’s the world of electronic health records.”

Hudson sympathized. In moving her mother from Texas to Minnesota she said, “I ended up carrying two boxes of paper medical records with me.”

She added,”I hope that doesn’t happen in the future, and I think we’re moving quickly to solve that problem.”

Hudson noted that interoperable systems are important in patient care but also “vital” for research, and especially important as the Precision Medicine initiative takes shape.

The Public Responds

Mary Woolley, president of Research!America, a nonprofit focused on public education, was pleased to see Upton and DeGette keep their promise to fund medical research.

“While it is important for the final language in the bill to allow for flexibility in the use of these funds in order to maximize their benefit, these additional dollars can empower NIH to sustain and embark on innovative studies that could reduce the prevalence and impact of costly and disabling conditions that continue to threaten individual and population health, our economic security, and global competitiveness,” said Woolley in a press release.

Vijay Das, policy advocate at Public Citizen’s Congress Watch, was less enthusiastic.

Das, in a press release, said that the new proposal is “riddled with dangerous provisions that would put patients at risk.”

“The 21st Century Cures initiative offers a horse trade: Increase funding for the world-renowned National Institutes of Health (NIH) in exchange for providing perks to the pharmaceutical and medical device industries to approve medications and devices faster based on weaker evidence,” Das said.

“Faster approval of new medicines will not spur innovation, but it will sacrifice safety. Passage of this legislation would put lives at risk.”

Woodcock told MedPage Today that while the new bill would speed development to biomarkers “[t]here’s no change to the time of our review process.”

“Most of the provisions here also are not for common diseases that have a lot of alternative therapies. They’re going to be for cancers or rare serious disease where the people have already said they’re willing to trade off some uncertainty to get access to treatment sooner.”

“I think it’s good of the patient groups to remind people that [safety is] an important side of the equation,” Woodcock added. “We don’t see anything in here that’s going to diminish that.”

Full Steam Ahead

Rep. DeGette said she found the level of consensus around the bill “striking and positive.”

She acknowledged that several members had commented on the new burdens the FDA would be tasked with as a result of provisions in the bill. Rep. Pallone counted more than 15 new guidance documents as well as separate approval processes for antibacterial and antifungal drugs for serious and life-threatening diseases.

Woodcock made clear that fulfilling these statutory requirements would create delays in reviewing applications, if the FDA was not given additional resources. “Currently, our drug review program is really going full speed. We’re making all our deadlines and we’d like to keep it that way.”

DeGette said, “We still need to find a way to fund the FDA for the things that we’re asking you to do and we know that. So, we’re going to do all of that.”

The next step is to have the bill scored and marked up, she said.

Senate Passes 21st Century Cures Act

WASHINGTON — The Senate passed the 21st Century Cures Act, sweeping legislation that aims to bring treatments more quickly from the lab bench to patients’ bedsides on Wednesday afternoon in a vote of 94-5.

“As a result of a lot of strong bipartisan work, we are sending a bill now to the president’s desk that will invest in tackling our hardest to treat diseases, put real dollars behind the fight against the opioid epidemic and make badly needed changes to mental health care in our country,” said Sen. Patty Murray (D- Wash.), Ranking Member of the Senate Health, Education, Labor and Pensions (HELP) Committee, just before the vote.

“I’ve heard often from those whose loved ones are suffering from Alzheimer’s, addiction, and other debilitating diseases,” wrote President Obama in a press release shortly after the vote, citing Vice President Joe Biden’s own tragic loss of his son Beau Biden.

“Their heartbreak is real, and so we have a responsibility to respond with real solutions,” Obama wrote, adding that he looked forward to signing Cures as soon as it reaches his desk.

The “Cures” bill authorizes a total of $6.3 billion for funding basic science, streamlining the FDA’s review process, and addressing the opioids epidemic. The bill also incorporates a handful of mental health reforms, aimed at improving care coordination, strengthening mental health parity laws, and promoting evidence-based treatments and therapies.

Murray thanked HELP Committee Chairman Lamar Alexander (R-Tenn.), who offered his own appreciation to his colleague and to those on both sides of the aisle.

Earlier this week Alexander called the bill’s provisions to invest in regenerative medicines “a game-changer” for stroke patients, those with heart disease or retinal disease and dubbed the entire package “another Christmas miracle” — referencing President Obama’s nickname for the 2015 rewrite of the “No Child Left Behind” education bill.

But not everyone was cheering: Public Citizen called the bill an early Christmas present for the pharmaceutical industry.

But the watchdog group didn’t sound only sour notes: in a prepared statement Michael Carome, MD, director of the group’s health research arm, claimed credit for helping to eliminate “provisions that would have 1) opened a gaping hole in the Physician Payments Sunshine Act for educational gifts made by industry to physicians; 2) increased medication prices and cost taxpayers an estimated $12 billion over 10 years; 3) encouraged hospitals to overuse the newest antibiotics, thereby contributing to the harmful spread of antibiotic resistance; and 4) allowed medical device manufacturers to make changes to high-risk medical devices without U.S. Food and Drug Administration oversight.”

Battling Opioids

Several Senators focused on the bill’s response to the opioids epidemic, emphasizing the current lack of resources and the slim capacity of many treatment facilities.

“When people with substance use disorder are turned away this means they remain on the streets, desperate, often committing crimes to support their addiction and at constant risk of a lethal overdose … Make no mistake this legislation will save lives,” said Sen. Jeanne Shaheen (D- N.H.) speaking from the floor on Tuesday.

Her colleague, Sen. Harry Reid (D- Nev.) said that the bill was “weak” in parts and “we could have done better,” but was also excited to see dollars for opioids.

“There should be far more, and it should be given a different way than we have it here, but it’s money,” he said. “And we have people … dying as a result of this scourge that’s sweeping America … So, that part of [the bill] is excellent.”

But the harshest critics of “Cures,”as the bill has been dubbed were unmoved by this sentiment.

Those opposing the bill included Sen. Bernie Sanders (I- Vt.), Sen. Jeff Merkley (D- Ore.), Sen. Ron Wyden (D- Ore.), Sen. Elizabeth Warren (D- Mass.), and Sen. Mike Lee (R- Utah)

The final breakdown for the bill is as follows:

  • $4.8 billion for the National Institutes of Health
  • $1 billion in state grants to help respond to the opioid crisis
  • $500 million in additional support for the FDA

Of the money allocated to the NIH over a 10-year period, the bill earmarks $1.8 billion for Vice President Joe Biden’s “Cancer Moonshot” project, — renamed “The Beau Biden Cancer Moonshot” — $1.6 billion for the BRAIN initiative, and $1.4 billion for President Obama’s Precision Medicine Initiative.

The Beau Biden Cancer Moonshot

The FDA’s funding would be geared towards bolstering research and staff, and like its sister agency, funds would be delivered over a decade, whereas the opioid grants will be administered over 2 years.

As Biden presided over a vote for cloture on Monday night, Congress offered an amendment to rename the Cancer Moonshot Initiative in memory of his son, Joseph “Beau” Biden III, who died from brain cancer in 2015.

“I think it fitting to dedicate this bill’s critical cancer initiatives in honor of someone who’d be so proud of the presiding officer [Biden] today: his son Beau,” said Senate Majority Leader Mitch McConnell (R-Kentucky).

That amendment passed without debate.

Winners and Losers

However, a second proposed amendment brought by Sen. Bernie Sanders (I -Vt.) did, receive pushback.

“I have been fighting the greed of the prescription drug industry for decades and as far as I can tell the pharmaceutical industry always wins. They win but the American people lose,” he said in a floor speech on Tuesday.

His legislation would allow Medicare to negotiate with drug companies and that would enable Americans to import their medications from other countries. Sanders stressed that both provisions had been endorsed by President-elect Trump during his campaign.

“Think about what you can do to pave the way for Mr. Trump coming in,” he said attempting to bait Senate Republicans who did not bite.

Full Speed Ahead

“One way to be sure and not get the work done we’re doing today is to add another topic,” said Sen. Roy Blunt (R- Missouri) who objected to Sanders’s amendment. “If everything’s a priority, nothing’s a priority.”

Sanders also criticized Cures for cutting $1 billion from Medicare and Medicaid programs and another $3.5 billion from the Affordable Care Act’s Prevention and Public Health Fund and for not giving enough funding to the NIH. Sanders said that if the bill passed it would still only grant the agency $7 billion less than it received in 2004, accounting for inflation.

The depletion of the ACA’s controversial fund also irritated the American Academy of Family Physicians, the group sent a letter to House leadership last week highlighting its disappointment, according to a press statement. The AAFP also noted that “the legislation stops well short of appropriately funding the important mental health and addiction provisions that are included.”

Mental Health

Echoing, Murray, Sen. John Cornyn (R-Texas) praised the bill’s mental health provisions noting that many families struggle to help their adult children who have mental health problems.

“Often there’s additional tools that need to be available to family members when they find that their loved one is getting sicker and sicker and not being compliant with their medication and potentially becoming a danger to themselves or to the community at large,” he said speaking from the Senate floor.

Cornyn noted that legislation will improve states and local government’s access to tools to help evaluate the healthcare needs of those in prison, so they can be helped. The bill also encourages the development of crisis intervention teams.

Though she voted for the bill, Sen. Debbie Stabenow (D- Mich.) urged Congress to “complete the job,” by delivering full-funding to community mental health centers.

The American Psychiatric Association applauded Cures saying it would improve the access and quality of care for people with serious mental illness, and those with substance use disorders.

“The bill will toughen enforcement of existing parity laws, helping to ensure that mental health care services are covered just like other health care services,” said Maria Oquendo, MD, PhD, president of the APA in a press release.

While Murray argued that another advantage of passing Cures now is to “lock-in” important investments ahead of the next administration, much of the actual funding in the bill will require Congress to appropriate or unlock dollars from various sources each year, such as sales from the federal Strategic Petroleum Reserve.

Approximately 420 organizations lobbied for the Cures bill, according to the The Center for Responsive Politics, including Blue Cross/Blue Shield, Roche, Amgen, and the Pharmaceutical Research & Manufacturers of America, the industry’s major trade association.

What Else is in the 21st Century Cures Act?

WASHINGTON — When most people think about the 21st Century Cures Act, they think about curing cancer and Alzheimer’s disease, and curbing the nation’s opioid epidemic. But the nearly 1,000-page healthcare spending bill, which President Obama signed in mid-December, also aims to reform the nation’s fragmented mental health system, improve access to electronic health data, and ensure that underrepresented individuals are included in important health research.

Mental Health

Approximately 13 million people in the U.S. have a serious mental illness or substance use disorder, according to the American Psychiatric Association (APA), which applauded the 21st Century Cures Act, calling its reforms to mental health a “huge step forward.”

The Cures Act included efforts to promote evidence-based treatments, strengthen mental health parity, and bolster the mental health workforce. The bill also established a grant program focused on early intervention for those showing warning signs of a potentially serious mental illness.

Earlier this year, Congress took aim at the Substance Abuse and Mental Health Services Administration (SAMHSA), saying that the agency had failed patients with serious mental health problems.

One almost immediate change the bill makes is to establish a new assistant secretary for Mental Health and Substance Abuse to oversee SAMHSA, explained Andrew Sperling, director of legislative advocacy for the National Alliance on Mental Illness.

Speaking in a phone interview with MedPage Today that included a public relations official, Sperling said he anticipates that Rep. Tom Price (R- Ga. ), the newly appointed Secretary of Health and Human Services, will have “a great deal of influence” in selecting that individual.

Better Integration, Better Care

The 21st Century Cures Act also designates a new Interdepartmental Serious Mental Illness Coordinating Committee, charged with summarizing advances in diagnosing and treating serious mental illness in a written report to congress. The committee — which includes members from SAMHSA, the Department of Defense, the Health Resources and Services Administration (HRSA), and other federal health agencies — will also be required to evaluate the impact of federal programs on important public health outcomes (i.e., rates of suicide, preventable emergency room visits, and homelessness).

Other key mental health-related provisions of the bill include the following:

  • Authorizing a grant program for assertive community treatment for adults with serious mental illness;
  • Authorizing a grant program to enable local governments to improve crisis intervention among emergency responders, clinicians, and law enforcement and funding to build registries of available inpatient psychiatric beds;
  • Establishing policies to create new residency programs to train future primary care doctors and psychiatrists about integrating mental and physical health;
  • Designating a nationwide hotline and online tool for improving access to mental health and substance use providers; and
  • Enhancing grant programs that offer behavioral health services for homeless and justice-involved individuals.

Also, the Cures Act, while not amending the Health Insurance Portability and Accountability Act (HIPAA), does outline the circumstances by which clinicians can share information with family members when a loved one is in crisis. “We hope this will ease the chilling environment around disclosure of information to family members,” by providing clinicians with greater clarity regarding when they can disclose, Sperling said.

With regard to mental health parity — equal access and coverage from insurers for mental health issues compared with physical concerns — most states have not been aggressively auditing insurers, an APA representative told MedPage Today.

However, the Cures Act calls for a standardized approach to auditing, and, on a federal level, requires the Department of Labor to periodically publish reports to showcase the volume of cases the agency has pursued.

Efforts to ensure more consistency and transparency could produce a “sentinel effect,” said the APA official. The more that insurers believe they risk being audited, the more likely they are to take internal compliance seriously.

Electronic Health Records

The Cures Act also strengthens efforts to improve and enforce health information interoperability.

Beginning in January 2018, vendors’ relative interoperability will be evaluated, and by 2019, those not in compliance will lose certification, explained Mandy Long, chair of the Electronic Health Records Association (EHRA) Clinician Experience Workgroup for the Healthcare Information and Management Systems Society (HIMSS), speaking in a phone interview with MedPage Today during which a public relations official was present.

“There are real teeth to [the language in the bill], and [the penalties] grow over the course of a couple of years,” said Long, who is also vice president of product management at Modernizing Medicine, an EHR and practice management software company in Boca Raton, Fla.

She noted that vendors who engage in data blocking can be fined up to $1 million per violation.

Long, whose daughter has Turner syndrome, also spoke as the parent of an ill child about the challenge of keeping specialists in different hospitals current with her daughter’s health status and tests. “We call it the ‘patient’s Bible’ — that binder that patients create for themselves, or their parents create [of various medical records] — we lug it from visit to visit or, God forbid, if we end up in an emergency situation; it’s awful and frequently out of date.”

Long said she believes the bill’s requirements to promote a scalable integration structure will have benefits for patients as well as industry.

The College of Healthcare Information Management Executives (CHIME) offers more specifics on the health IT provisions of the bill related to information blocking, interoperability standards, and hardship exemptions for decertified EHRs.

Superbugs, Vaccinations, and Equity in Research

Other elements of the bill include provisions to fight superbug infections, such as allowing the FDA to require manufacturers or reusable medical devices to share their cleaning instructions and verify that such methods work. The Cures Act also creates new requirements for the National Institutes of Health to encourage scientists studying similar topics to collaborate, with the goal of increasing the volume of data on underrepresented populations (i.e., women, children, and minorities).

Finally, the bill aims to raise maternal vaccination rates through efforts to prevent vaccine shortages and by incentivizing drugmakers to develop new vaccines for pregnant women. In addition, the act encourages pediatric drug development by allowing products given a “rare pediatric disease designation” from the FDA before 2020 the chance to be considered for a voucher until 2022.

Unpacking the 21st Century Cures Act

When the 21st Century Cures Act was signed by President Obama earlier this week, many praised the bill as a tribute to bipartisanship. The act passed with overwhelming support in the Senate, and was dedicated to Joe Biden’s son, Beau, who died last year from brain cancer.

Although the act earmarks funds for the Beau Biden Cancer Moonshot and BRAIN initiatives, the act is more than 1,000 pages long and includes provisions for a myriad of healthcare issues.

“The idea of the bill was to get new drugs to patients more quickly and new provisions have been added onto that bill partially because it’s the end of the year, other things didn’t get passed that needed to,” says Shannon Firth, Washington correspondent for MedPage Today. “But essentially what this bill does is it earmarks funding for congress to focus on things like brain research and cancer research, and also it helps revise some of the practices of the FDA.”

Much of the reporting on the Cures Act has been focused on the ways it impacts the FDA. “One of the problems that both the NIH and the FDA has is staffing,” explains Firth. Both agencies have issues keeping researchers from moving on to better paying jobs. Firth continues, “So this bill puts money toward the FDA in terms of keeping those talented researchers there.”

But the act will also have a significant impact on how the agency reviews and screens new drugs. “What the bill does is, it helps promote something called ‘patient focused drug development.’ And while some patient groups may say it’s a good thing, there are on the other side, consumer groups who are very concerned about some of the provisions in the bill. They’re concerned that it would weaken the FDA’s review process,” she explains.

While the act was passed with rare bipartisan support, it had some major detractors. Senator Elizabeth Warren (D-MA) and Senator Bernie Sanders (D-VT) were both vocal critics of the Cures Act, in part because they felt it gave a lot to pharmaceutical companies, without getting much in return.

“One of the things they were very focused on was controlling drug prices. Senator Sanders put out there an amendment, he wanted to allow Medicare to negotiate with drug companies to help lower the prices of drugs,” says Firth.

“He wanted to allow the importation of drugs from other countries and a lot of other countries, they spend a lot less on drugs than we do.”

She continues, “Those were two ways he thought that this bill could’ve been improved by allowing drug companies, who are getting a lot out of this bill, to actually also put something back in.”

The Cures Act also designates money to helping stem the opioid epidemic by making more beds available in treatment centers and encouraging medically proven methods to treat opioid addiction.

Some of these provisions fall under the umbrella of behavioral health, and will be impacted by mental health parity provisions bolstered by the Cures Act. The parity act will make it more difficult for insurers to deny coverage for drug addiction rehabilitation treatments, a significant barrier for many addicts.

The act also addresses mental health more directly. “What the bill does for mental health is it strengthens coordination, it elevates mental health to a position within the Health and Human Services, and it strengthens mental health parity,” Firth explains.

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