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Is an Effective Alzheimer’s Treatment Around the Corner?

  • Posted In FDA

More than 6.2 million Americans currently have Alzheimer’s disease, a number that is projected to double by 2050.  Despite a growing need to treat Alzheimer’s, some four decades of research have yet to yield any effective therapeutics.  This could change as soon as June 7, 2021, which is the date the Food and Drug Administration (FDA) may decide to approve a new Alzheimer’s treatment from Biogen.  Why has it been so hard to develop an effective Alzheimer’s treatment, and does Biogen’s drug mark a breakthrough?

Failed Research Efforts So Far

Since the National Institute on Aging first began studying ways to treat Alzheimer’s disease in earnest in 1978, progress on effective therapeutics has been extremely limited.  A major reason for the lack of progress is the newness of the field of neurobiology, especially as it applies to Alzheimer’s, dementia, and other complex brain disorders.  Most of the knowledge on the human brain has only been acquired within the last 40 years, while a better understanding of genetic and biologic pathways has only occurred within the last 30 years.  Additionally, brain lesions and biomarkers associated with Alzheimer’s could not be visualized until 2004.  As a result, meaningful research and development of  Alzheimer’s treatments could not accelerate until a quarter-century worth of understanding could be attained.

Since the 1990s, research pertaining to  Alzheimer’s treatments has focused on how the disease develops and progresses.  Alzheimer’s develops when toxic plaques clump together between neurons in the brain, resulting in inflammation and cell death in the brain.  Many potential drug treatments have failed while under development and at a higher rate than other drugs —  99.6% for Alzheimer’s drugs in development have failed, compared to an 80% failure rate for cancer drugs.  Among those failed potential therapies were a a pair of therapeutics from Eli Lilly and Roche, which saw their clinical trials end in February 2020.  Researchers point to the complexity of Alzheimer’s as one of the reasons why so many promising treatments have gone to the wayside.  The last Alzheimer’s therapeutic to receive FDA approval was Forest Labs’ Namenda in 2003, which along with the handful of other Alzheimer’s drugs, only alleviates symptoms temporarily.

Enter Stage-Right: Aducanumab

Like other recent drug development programs, Biogen’s aducanumab is an injectable, monoclonal antibody treatment that targets plaque build-ups between neurons.  If approved, aducanumab would be the first disease-modifying drug for Alzheimer’s, meaning it is effective in slowing cognitive decline.  This could open the door to Alzheimer’s becoming a chronic disease that people live with for many years, similar to diabetes and cancer.

According to data discussed during an FDA advisory committee meeting in November 2020, aducanumab proved effective in a small phase 2 clinical trial and one of two large phase 3 clinical trials.

Will FDA Give Aducanumab the Nod?

Mr. Barker, the answer is “not sure.”  While the experimental drug was shown effective in the small phase 2 and large phase 3 trials, a second phase 3 trial failed to yield positive results.  As a result, some members of the panel have expressed a desire to see more data from an additional phase 3 trial, although such an undertaking would require another three to five years of research.  Additionally, there is some concern about the actual benefits of aducanumab, which showed only modest changes on two objective measures used in the clinical trials: the Mini-Mental State Examination and the Alzheimer’s Disease Assessment Scale.

Also, there are concerns about side effects which may not only impact patients but also  the health care system as a whole.  30% of clinical trial participants who took the drug were found to have a reversible swelling of the brain, while over 10% had small brain bleeds.  These side effects were detected using amyloid positron emission tomography (PET) scans, and given the millions of Alzheimer’s patients, the large number of PET scans necessary to monitor for side effects would test the limits of neurologists, radiologists, and other clinicians.

What’s the Cost?

Aducanumab will be sure to have a high price tag if approved.  One year’s worth of aducanumab treatment could be priced as high as $50,000 per year, and if only a third of the 6 million Americans with Alzheimer’s were to take the drug, sales could reach $36 billion a year.  Since most people with Alzheimer’s begin noticing symptoms in their mid-60s, Medicare is likely to pick up the tab for most aducanumab treatments, not to mention the PET scans and other health care services required to monitor for side effects.

What Could Happen?

FDA is not in an enviable position on aducanumab.  The agency may determine that it cannot sign off on the safety and efficacy of the drug, or at least request more data before making a decision, despite the clear and significant need such a drug for those currently affected by Alzheimer’s and their loved ones.  As such,the projected growth in the number of Alzheimer’s patients and a lack of existing therapeutics mean any decision on June is sure to impact millions of people.

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The Week in Review: June 1-4

Biden Calls Out Manchin, Sinema for Holding Back Democrats’ Agenda

During a June 2 event marking the 100th anniversary of the Tulsa race massacre, President Joe Biden said “two members of the Senate who voted more with my Republican friends” is the reason why the Senate has been unable to advance a bipartisan voting rights bill.  The President was referencing Sens. Joe Manchin (D-WV) and Kyrsten Sinema (D-AZ), whose strong support of the filibuster means Democrats are unable to advance key proposals with a simple majority.  Notably, Manchin and Sinema’s record indicates they vote with other Democrats most of the time.

Biden’s All-Out Push to Get 70% of Adults Vaccinated by July 4

President Joe Biden on June 2 announced a “national month of action” to help his Administration meet its goal of having 70% of American adults at least partially vaccinated for COVID-19 by July 4.  Some of the key actions Biden outlined to spur more vaccinations include partnering with child-care providers to offer free services to all parents getting vaccinated or recovering from the shots and requesting pharmacies to extend their hours for the month of June.  The Administration also announced an initiative to provide vaccinations at Black-owned barbershops.  Currently, 63% of US adults have received at least one COVID-19 vaccine shot.

Moderna Files Application for Full FDA Approval of COVID-19 Vaccine

On June 1, Moderna began filing for a Biologics License Application (BLA) with the Food and Drug Administration (FDA) that would allow full approval of its COVID-19 vaccine.  Full approval would allow Moderna to directly market its vaccine as well as make it easier for companies and government agencies to mandate vaccinations for employees.  Full approval is also seen as an important step in addressing vaccine hesitancy.  Pfizer became the first drug maker to seek full FDA approval for its vaccine on May 7.  However, FDA has yet to comment on the timeline for reviewing the BLA for either vaccine.

Administration Announces Plans for Sending 25 Million Vaccines Abroad

On June 4, the Biden Administration formally laid out a plan to send out an initial wave of 25 million COVID-19 doses to help countries that have been hit hard by the virus.  According to the plan, 19 million doses will be distributed via worldwide vaccination initiative COVAX to Central and South America, Africa, and Asia.  Additionally, the US will separately supply 6 million doses to over a dozen countries including Canada, Mexico, and Ukraine.  The first wave of doses is part of a broader Administration effort first announced on May 17 to contribute 80 million COVID-19 vaccine doses as part of a global vaccination effort.

ICYMI: DC Museum Features George Washington’s Whiskey Writing

Every Friday from now until the end of July, the Stephen Decatur House Museum in downtown DC will have on display a letter penned by George Washington in 1799.  The letter, which is on loan from the Distillery Spirits Council, focuses on Washington’s pre- and post-presidential career as a whiskey distiller.  In the letter, Washington asks his nephew to assist with purchasing with purchasing enough grain to produce 200 gallons of whiskey.

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All About OMB

The Office of Management and Budget (OMB) may not get as much attention as other Cabinet-level agencies like the Departments of Defense and the Treasury, but that doesn’t mean OMB is any less important.  In fact, OMB plays a critical role in managing the operations of the federal government and the budget process.  More so, OMB is responsible for reviewing regulations that shape Americans’ daily lives.  So, how does OMB do this?

The History of OMB

OMB’s origins date back to 1921, when the Budget and Accounting Act of 2021 established the Bureau of the Budget at the Department of the Treasury.  The bureau was eventually moved to the Executive office of the President in 1939, where it was reorganized into the Office of Management and Budget in 1970.

OMB Today

Comprised of nearly 500 career staff, The mission of OMB is to “assist the President in meeting policy, budget, management, and regulatory objectives and to fulfill the agency’s statutory responsibilities.”  OMB mainly carries out this mission through 5 main functions:

  1. Preparing a budget proposal to Congress;
  2. Supervising executive branch agencies;
  3. Reviewing all rulemaking and regulatory actions;
  4. Reviewing agency testimony, legislative proposals, and other communications with Congress; and
  5. Clearing Presidential Executive Orders.

Current Leadership

The organization is led by three-Senate confirmed positions: Director, the Deputy Director, the Deputy Director for Management.  The current Acting Director of OMB is Shalanda Young, who was confirmed as Deputy Director in March 2021.  The Biden Administration’s latest nominee for OMB Director is Kiran Ahuja, who previously served as Chief of Staff of the Office of Personnel Management and Executive Director of the White House Initiative on Asian Americans and Pacific Islanders during the Obama Administration.   While Ahuja, was favorably reported by the Senate Homeland Security and Governmental Affairs Committee, the Senate has yet to schedule a floor vote to consider her nomination.

Extension of the President with Congress

OMB play 3 key roles for the President as the Administration interacts with Congress:  the budget, statements on legislation, and the Congressional Review Act. 

Budget.  While the Presidential budget submission to Congress typically happens around February each year, OMB is active long before the annual tome hits the streets.  OMB works on average six months prior by sending budget instructions to agencies and later reviewing and analyzing agencies’ formal budget proposals.  Additionally and throughout the year, OMB provides guidance on a range of budgetary topics, including government shutdowns, continuing resolutions, and agencies use of discretion with mandatory spending.

SAPs.   OMB prepares Statements of Administration Policy (SAPs) on any pending legislative proposals that are not related to appropriations that communicate the President’s position on the affected legislation.  SAPs typically serve as a strong indicator of whether the President will veto or sign a bill into law.  OMB also reviews and clears congressional testimony, congressional correspondence, and draft bills from the agencies to ensure the documents are aligned with the Administration’s agenda.

Congressional Review Act.  The Congressional Review Act (CRA) is an oversight tool Congress can use to overturn a rule by a federal agency or department.  OMB is tasked with determining whether a rule is considered a “major rule” under the CRA.  In the case of major rules, Congress can pass a joint resolution of disapproval, which would veto the rule if signed into law by the President or approved by two-thirds of both chambers of Congress.

Role in Rulemaking

OMB plays a pivotal role in rulemaking, primarily through the Office of Information and Regulatory Affairs (OIRA).  Like its parent agency, OIRA is currently without a permanent leader; former National Labor Relations Board member Sharon Block has been serving as OIRA’s Acting Administrator since April 2021.  Under the current framework, OIRA is charged with reviewing all regulatory actions and determining whether the regulations are “significant” or “economically significant,” meaning they have an annual effect on the economy of $100 million or more.  For rules deemed significant and economically significant, however, agencies must submit several documents, including the draft text of the rule, an explanation of how the rulemaking will address a particular need, the potential costs and benefits of the rule, and in the case of rules considered economically significant, a regulatory impact analysis.

After receiving all necessary documents from agencies on significant rules, OIRA has 90 calendar days to review, with the option of extending the review period for one time by up to 30 days.  OIRA then proceeds to review the agency’s documents and return the rule to the agency in one of three ways:

  1. Consistent without change.  This means OIRA did not alter the proposed rule.
  2. Consistent with change.  This means OIRA generally agrees with the intent of the rule but made some substantive changes. This is the most common type of action OIRA takes.
  3. Returned.  This means OIRA has serious concerns with the agency’s proposed rule and does not approve the publication of a Notice of Proposed Rulemaking (NPRM). Returned rules are always accompanied by a return letter which is posted on OIRA’s online docket.   An example of a returned letter is a 2002 letter to the Office of Personnel Management rejecting proposed changes to the Federal Employee Health Benefits Program due to concerns over cost measurement and accounting principles.

Once OIRA has completed its review process, the proposed rule is published in the Federal Register in the form of a NPRM. The rule is opened to a public comment period generally lasting 60 days.  When the public comment period concludes, the agency makes any changes to the rule and resubmits it to OIRA for another round of review. Again, OIRA has 90 days to conducts its review process. When OIRA completes its final review, the agency may set a date for promulgation of the rule and publish the final rule in the Federal Register.  Members of the public can track rules under the review of OIRA at www.reginfo.gov.

Does OMB Matter?

Absolutely.  Even if OMB may not be mentioned as frequently as other agencies, its power cannot be understated.  OMB oversees a number of multi-trillion-dollar budgets that impact every aspect of American life, and OMB’s role in supervising federal agencies means it’s more or less in charge of 4.3 million federal employees.  Based on the goals of the Administration, OMB is an important partner with Congresson how much will be spent on health, defense, housing, and other key areas.

Furthermore, OMB’s role in coordinating and reviewing regulatory action makes it a key player in the day-to-day actions in government.  Whether it pertains to Medicare reimbursement, FDA user fees, price transparency, or otherwise, all rulemaking and regulatory action is subject to OMB’s review.  Despite a lack of attention, OMB’s impact is far-reaching.

Did You Know?

Did you know that you can meet with OMB?  OIRA has an open door policy, which means the office takes meetings with outside stakeholders on regulations under review.  Meeting participants and materials are publicly disclosed and provide an interesting window into additional input the agency receives beyond formal written comments on the rule.  This month’s hot topic appears to be the physician fee schedule, among other health care related ruled.

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The Promise of Non-Profit Drug Companies

With trust in pharmaceutical companies waning and drug prices climbing, it’s clear providers and patients are frustrated with drug manufacturers.  Out of this frustration, providers and philanthropists have banded together to form non-profit drug companies, which aim to deliver low-cost generics by eschewing the profit-driven focus of other drug makers and embracing a mission to make drugs accessible and affordable.  But in an industry dominated by for-profit players, do non-profit drug companies stand a chance at making a difference?

The Major Non-Profit Players

Over the past few years, a handful of non-profit companies have emerged.

  • Civica Rx, the most prominent non-profit drug maker, formed in September 2018 when several major health systems teamed up to manufacture their own generics.  The company grew out of a need to bring competition to the pharmaceutical industry, with the hopes of reigning in high prices for generics.  Civica first began manufacturing two antibiotics in March 2019 and signed agreement to produce injectable medications later that year.  In January 2021, Civica announced plans to build a 120,000 square foot facility just south of Richmond, VA to produce sterile injectable drugs for hospitals.  Civica has also expanded into drug development – in  January 2020, it signed a deal with Thermo Fisher Scientific to develop and manufacture its own drugs.  The company also notably partnered with 18 Blue Cross and Blue Shield companies in January 2020 to form a new subsidy to reduce the cost of generics.
  • Phlow Pharmaceuticals formed in January 2020 to produce active pharmaceutical ingredients (APIs) used in drug manufacturing.  Beginning in May 2020, the federal government awarded Phlow a total of $812 million to manufacture medicines at risk of shortage, including medicines for the COVID-19 response.  Notably, Phlow partnered with Civica on the new facility near Richmond, VA.
  • Harm Reduction Therapeutics formed in 2017 with the goal of preventing opioid overdose deaths by bringing a low-cost, over-the-counter naloxone spray to market.  In June 2020, the company received $6.5 million in financial support from Purdue Pharma, which produces opioids for prescription use.
  • Medicines360 formed in 2015 to develop and provide low-cost contraceptives to women.  In October 2019, the Food and Drug Administration (FDA) approved a hormonal intrauterine device (IUD) Medicines360 developed with Allergan that prevents pregnancy for six years, the longest approved duration for IUD use in the US.

Advantages of Non-Profit Drug Makers

When it comes to delivering lower-cost drugs, non-profit pharmaceutical companies have several advantages.

  • Non-profit drug companies focus on generic drugs, meaning they are out of the patent protection period and in the public domain.
  • Additionally, thanks to their non-profit model, these companies don’t need to raise prices to honor fiduciary obligations to shareholders.  Excess funds can also be invested back into the organization to enable it to continue its non-profit mission.
  • Civica specifically operates on a membership model for hospitals that allows the company to make long-term commitments to members at a fair price.  This arrangement also allows Civica to offer the same price per unit to each hospital member.

Challenges for Non-Profit Drug Makers

Success for non-profit drug companies is not guaranteed, and they face major obstacles to fulfilling their mission.

  • Despite financial assistance from philanthropy, grants, and other means, non-profit companies lack access to capital through multiple investment streams that for-profit companies can more readily access.  This leaves non-profits at a serious disadvantage when it comes to research and development, which involves raising large amounts of capital. (Provigil)
  • When it comes to certain rules and regulations, the non-profit status of companies like Civica doesn’t entitle them to many breaks or special treatment.  For instance, the FDA imposes user feels on all drug manufacturers, regardless of their status or mission.
  • Some reimbursement and payment policies may present obstacles  for non-profits to offer lower prices, particularly Medicaid’s “best price” rule.  To remain sustainable, non-profit drug makers may offer higher prices for some purchasers while keeping prices low for uninsured or low-income patients.  By offering low prices to the aforementioned groups, non-profits would be required to offer the same low price across Medicaid.

Will Non-Profit Drug Companies Make a Difference?

Non-profits like Civica Rx and Phlow are new entrants to the drug market, and only time will tell if their efforts will pay off in the form of lower drug prices.  However, non-profits could follow a few strategies to increase their odds of success.  While Civica Rx and Medicines360 have become involved in drug development, non-profit drug manufacturers would be better off focusing on generics given their disadvantage in research and development.  Additionally, non-profit drug makers could push for policy and regulatory changes to give them a more favorable position in the market, such as reducing or waiving user fees for non-profits, increasing price transparency, or creating an exemption under the Medicaid “best price” rule.

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The Week in Review: May 24-28

Moderna Says Its COVID-19 Vaccine Is 100% Effective in Teens

On May 25, Moderna announced that its COVID-19 vaccine is 93% effective in children aged 12-17 after the first dose and 100% effective after the second.  Additionally, data from the company’s phase 2/3 clinical trial consisting of 3,700 adolescents identified no serious side effects.  Moderna plans to ask the Food and Drug Administration “in early June” to amend its emergency use authorization to allow individual as young as 12 to receive the vaccine.  Currently, only Pfizer’s COVID-19 vaccine is authorized for use in teens.  If approved, Moderna’s COVID-19 vaccine would greatly increase the supply of shots available to middle and high school students ahead of the next school year.

“Lab Leak” Theory on Virus’s Origin Gains Steam

Following growing concerns that COVID-19 may have originally escaped from a laboratory, President Biden directed the intelligence community to “redouble” its efforts to investigate the origins of the COVID-19 pandemic.  These concerns are fueled by reports that three employees at China’s Wuhan Institute of Virology were hospitalized with symptoms consistent with COVID-19 and “common seasonal illness” in November 2019.  However, hard evidence that COVID-19 originated in a lab has yet to be found, and NIAID Director Anthony Fauci told a Senate committee on May 26 that he still believes the “most likely scenario is that [COVID-19] was a natural occurrence.”  The intelligence community is expected to report its findings to the President in 90 days.

Senators Take Action on Public Option, Rural Health Care Workforce

On May 25, top Democrats on bicameral health committees issued a request for information (RFI) to gather feedback on a proposal to create a government-run public health insurance option.  Among the issues the RFI seeks feedback on are the criteria used for determining prices, the role of states, and how a public option would interact with Medicare and Medicaid.  While President Biden campaigned on creating a public option, the proposal faces an uphill battle from Republicans and health care stakeholders who are strongly opposed.   In other congressional news, four bipartisan senators reintroduced legislation on May 27 to extend a program that allows international doctors to remain in the US upon completing their residency, so as long as they practice in rural areas that are suffering from physician shortages.  Notably, the bill would expand the number of physicians who can participate in the program and would open participation to physicians’ spouses.

Former Virginia Sen. John Warner Dies at Age 94

On May 25, former Virginia Senator John Warner died of heart failure in his Alexandria home at 94 years old.  Warner, who represented Virginia in the Senate from 1979 to 2009, was a moderate Republican who occasionally broke ranks with his party on high-profile issues.  For instance, Warner opposed Robert Bork’s nomination to the Supreme Court and supported several gun control bills.  Warner, a veteran of both World War II and the Korean War, chaired the Rules and Armed Services committees for several years each in the Senate.  In his post-Senate career, Warner worked as a Senior Advisor for the Washington, DC law firm Hogan Lovells.

CMS Delays Medicaid “Best Price” Policy for 6 Months

On May 26, the Centers for Medicare and Medicaid Services (CMS) issued a proposed rule to delay the effective date of a rule requiring drug manufacturers to include discounts they offer to patients when calculating the “best price” for drugs under Medicaid’s drug rebate program.  According to CMS, the delay is intended to provide the agency, states, and drug makers time to address concerns over patient access and make changes necessary to implement the new requirements.  The rule, which was initially set to go into effect on January 1, 2022, received mixed feedback from stakeholders.

ICMYI: Congress Set to Review Intelligence Report on UFOs

US intelligence agencies are expected to deliver a report to Congress next month on UFOs, or “unidentified ariel phenomena” (UAPs) in the military’s parlance.  The report is a result of the FY 2021 omnibus appropriations bill that was signed into law in December 2021. Interest in UFOs has grown among both the general public and lawmakers after the Department of Defense declassified videos taken by the US Navy of strange objects flying near warships and aircraft.  Pentagon officials say they’re studying UAPs to determine whether the objects represent a threat to national security.  It remains to be seen to what extent the report will contain newly declassified information.

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