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CDC Issues Risk Advisory on Dressing Chickens For Halloween, Biotech Chickens May Help

What do chickens have to do with Halloween? Well, according the Centers for Disease Control and Prevention, the likelihood of contracting a harmful bacterium, such as salmonella, increases when dressing a chicken in a Halloween costume. This makes sense because any contact with a live animal, especially a bird, presents the chance for transmission of viruses from animal to human.

However, this claim caused quite the stir yesterday when news outlets incorrectly reported on the CDC's advisory, with many interpreting the news reports as a guidance to not dress chickens in costume at all. (Que the collective screams from Halloween-loving chicken owners across the U.S.)

The CDC later clarified the news reports, saying that they are not warning people against dressing up chickens in Halloween costumes, they just want you to take precautionary measures whenever handling fowl – whether to dress it in a miniature-sized sheriff costume or not.

But, this is a biotech blog, you say. Where does biotechnology come into play in all of this? Well, the CDC's warning about handling chickens stems from the fact that many birds do carry viruses and bacterium that can put human health at risk. Most notably, birds like chickens can carry the bird flu, otherwise known as H1N1.

Through genetic modification, however, researchers at The Roslin Institute of the University of Edinburgh and the University of Cambridge have created a chicken that does not transmit the virus to other chickens. Essentially, these GM chickens would be the dead end for a virus that can quickly spread in poultry flocks.

Moreover, through this modification, the risk of transmitting the bird flu to humans becomes greatly reduced.

Until these chickens are commercialized, however, chicken owners should definitely follow the advice of the CDC when handling their flocks. Luckily, dressing chickens up as Santa isn't a thing. Or is it?

Opportunities for Patient and Health Advocates

Last week, the seventh annual Patient and Health Advocacy Summit brought  hundreds of patient advocates, industry experts and policymakers together to engage on important issues facing the patient community.

The summit concluded with a session on patient-focused drug development (PFDD) with distinguished panelists representing diverse perspectives: Paul Hastings, President and CEO of Nkarta Therapeutics; Laurie Burke, MPH, Founder of LORA Group; Cartier Esham, PhD, EVP of Emerging Companies Section and SVP of Science and Regulatory at BIO; Annie Kennedy, SVP of Legislation and Public Policy at Parent Project Muscular Dystrophy; and Pujita Vaidya, MPH, Acting Director of Decision Support and Analysis Team at the Office of Program and Strategic Analysis of the Food and Drug Administration.

In the new PFDD era, patients are helping regulators and developers understand diseases more holistically, so we can expect the drug development process to increasingly focus on aspects that patients value in their treatment.  This new level of involvement provides exciting opportunities for patient and health advocates to have a voice throughout the regulatory development of new treatments.

In recent years, FDA has incorporated PFDD into its processes thanks to regulatory milestones such as the Prescription Drug User Fee Agreement V, the 21^st Century Cures Act and the evolution of criteria for FDA's clinical outcomes assessments (COA).  These developments have allowed FDA to incorporate patient perspectives into its regulatory approval process by integrating patient input, designing studies with the patient in mind, integrating patient-reported outcomes and better communicating information to patients and providers.

PFDD has led the FDA to progressively use patient data to better understand the disease or condition, conceptualize the clinical benefit and modify the outcomes measure.  Most prominently, this includes real-world evidence (RWE) and real-world data (RWD), both of which harness patient data that supports healthcare decision-making, namely coverage decisions, clinical practice use, and innovative clinical trial designs.

As Dr. Esham described, PFDD has brought about a monumental shift in drug development because, perhaps we didn't know what mattered most to patients before PFDD came to fruition.  When we listen to the patients, we learn things that can help inform the drug development process.  This feedback loop benefits from involving everyone in the ecosystem, from drug developers to payers to patients.  The next step is to build on these efforts by working together in the patient community to help define what it really means for the patient to have a positive experience.

The panel illustrated many opportunities where the patient community can have an impact on drug development.  Patient groups can meet directly with developers to provide information on their disease or condition.  At FDA, patients can now participate in COA development; inform understanding of a disease or condition; participate in development of best practice standards; and participate in generation of RWE and RWD to support new uses of approved drugs.  There are also open agency forums where the patient experience can be shared.  As Kennedy put it, "FDA has put us smack dab in the middle of [its] work."

As the two-day summit came to a close, Dr. Julie Gerberding, EVP for Communications, Global Policy and Population Health & Chief Patient Officer at Merck & Co., provided closing remarks to the crowd of patient and health advocates.  Her message was one of empowerment and togetherness.  She implored those present to mobilize their most potent asset: a collection of voices that acts as a powerful source of energy.

Gerberding tasked the community to come together with an articulate message so that advocates can capitalize on opportunities to make the patient experience more prominent throughout the drug development process.  Hopefully the summit empowered attendees with the tools and knowledge to do just that.

 

Trump administration squares off with drugmakers over plan

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BIO Patient & Health Advocacy Summit Concludes Today

Each year BIO brings the patient stakeholder community together for the BIO Patient and Health Advocacy Summit to network; to learn; to engage; to partner; and to discover.

Held this year-its seventh-at the Park Hyatt hotel in Washington, DC, the Summit concluded today after running a robust program focused squarely on patients and their immeasurable contributions to the drug development process.

The two days of educational panels addressed best practices for engaging with industry, patient access to life-saving medicines, the opioid crisis, the FDA's "Patient-Focused Drug Development" guidance and the Trump Administration's "American Patients First Blueprint"-a proposed plan for addressing the cost of prescription drugs. Two of the sessions were live streamed on Facebook.

The intimate setting for the event was perfect for the lunchtime roundtable discussions hosted by subject matter experts from drug development companies and BIO staff. Attendees could choose from among 12 different subject areas which included such diverse topics as unconscious bias in clinical research, genome editing, Medicare Part B access and newborn screening-to name a few.

BIO's One-on-one Partnering™ offered attendees individual meetings with potential collaborators, patient groups, industry reps and other stakeholders. This more targeted networking opportunity helped participants connect with the right people, enhancing their chances for building alliances that address unmet medical needs.

We talked with several attendees about their experience at the summit.

 

European Scientists Unite to Safeguard Gene-Editing for Crops

Leading researchers representing more than 85 European plant and life sciences research centres and institutes have endorsed a position paper that urgently calls upon European policy makers to safeguard gene-editing technologies in plant science and agriculture.

The scientists are deeply concerned about a recent European Court of Justice ruling concerning modern genome editing techniques that could lead to a de facto ban of innovative crop breeding.

The position paper argues the European farmers may be deprived of a new generation of more climate resilient and nutritious crop varieties that are urgently needed to respond to current ecological and societal challenges.

The joint document follows an outpouring of concerned statements of European research institutes over the recent months following the Court of Justice of the European Union (ECJ) ruling under which genome edited organisms must comply with strict EU Genetically Modified Organisms (GMO) legislation.

The position paper is evidence of a solid consensus among the academic life science research community in Europe on the negative consequences of the ruling.

"The support for this initiative from plant scientists all over Europe has been overwhelming," said Dirk Inzé, scientific director at VIB-University of Gent Centre for Plant Systems Biology and one of the initiators of the position paper.

"It clearly illustrates the current dichotomy in Europe: as European leaders in the field of plant sciences we are committed to bringing innovative and sustainable solutions to agriculture, but we are hindered by an outdated regulatory framework that is not in line with recent scientific evidence. With this mission statement we hope to promote evidence-informed policymaking in the EU, which is of crucial importance to us all."

– Dirk Inzé, scientific director at VIB-University of Gent Centre for Plant Systems Biology

The implications of a very restrictive regulation of innovative plant breeding methods are far-reaching, say the signatories, arguing that European agricultural innovation based on precision breeding will come to a halt because of the high threshold that this EU legislation presents.

To safeguard innovation in agriculture in Europe, the signatories of the position paper ask for a new regulatory framework that evaluates new crop varieties based on science.

Readers can view the position paper and add your name to the list of signatories here. ​

Patient Advocates Sounding the Alarm Over Fail First

The Centers for Medicare & Medicaid Services (CMS) has reversed a long-standing policy allowing Medicare Advantage plans to impose "step therapy" requirements for physician-administered drugs – also known as Medicare Part B.

More accurately described as "fail first," this dangerous policy will force some patients to fail first on a medicine preferred by their insurance companies before they can receive the therapy prescribed by their doctors. Allowing insurers to stand between patients and the right treatments they need – as determined by their health care provider – threatens the health and well-being of America's seniors. That's why this abrupt regulatory decision has patient advocates sounding the alarm:

"Step therapy protocols that require patients to try and fail certain treatments before allowing access to other, potentially more appropriate treatments can both harm patients and undercut the physician-patient decision-making process." - American Medical Association, the largest association of physicians and medical students in the United States

"We are not persuaded that this plan provides [cancer patients] or the health care system meaningful financial relief, while at the same time it adversely affects beneficiaries' treatment choices and imposes on them the responsibility for pursuing exceptions to receive proper treatment." - American Society of Clinical Oncology, CancerCare, and 14 other organizations under the Cancer Leadership Council umbrella

"When these policies interfere with the patient-physician relationship, they can result in delayed treatment, increased disease activity, loss of function, and potentially irreversible disease progression. For providers, step therapy exacerbates administrative burdens as they help patients navigate complicated and often opaque coverage determination processes." - 40 groups including the American Academy of Neurology, the National Psoriasis Foundation, and the US Pain Foundation

"Fail first policies limit the options available for patients and their physicians to treat conditions, forcing individuals to try the treatment that is preferred by the insurance company (often an older, cheaper medicine) rather than the medicine prescribed by the physician. … Policies that sacrifice the health of patients in the hope of cutting program costs undermine the promise Medicare represents for so many individuals." - Part B Access for Seniors and Physicians Coalition, who garnered 240 groups to sign its fail first letter

"Ensuring patients can get the right medicine at the right time is instrumental in containing long-term healthcare costs," former Sen. Tom Coburn recently wrote in The Hill. And while this statement is true, implementing shortsighted polices like step therapy would do just the opposite. In its current form, CMS' proposal lacks basic patient protections and should be pulled back before it harms America's most vulnerable population.

Making SMA Newborn Screening a Reality: Collaborating with the Patient Advocacy Community

Each year, roughly 12,500 newborns in the U.S., or one out of every 300 infants, are diagnosed with one of the 29 conditions that every newborn is screened for shortly after delivery.  Ranging from cystic fibrosis to Sickle cell anemia, the conditions often require immediate and intensive medical intervention.  This list of conditions includes spinal muscular atrophy (SMA), which until earlier this year, was excluded from the Recommended Universal Screening Panel (RUSP), the expert-approved list of conditions that the Secretary of Health and Human Services (HHS) recommends that states test for at the time of birth.

SMA is a degenerative neurological condition that destroys nerve cells in the brain stem and spinal cord, affecting a person's ability to move, eat, and even breath. The majority of infants diagnosed with the most severe form of SMA rarely live past the age of two.  The disease's life-threatening effects reinforce why early detection and diagnosis can make all the difference for those that carry the genetic mutation that causes SMA.

That's why, in recent years, Biogen came together with partners in the SMA community, including Cure SMA and other rare disease advocates, to develop a compelling case for adding SMA to the RUSP. Together with the SMA community, we sought to highlight the positive impact that SMA's inclusion would have for patients, their families and communities and the healthcare system as a whole.

The first step in the process was developing the scientific and public health justification for the inclusion of SMA. By establishing a multi-faceted justification – illustrating the benefits and potential lives saved by routine testing – and collaborating with our partners, we were able to succeed when past efforts had fallen short. In February 2018 the Advisory Committee on Heritable Disorders in Newborns and Children recommended to the HHS Secretary that SMA be added to the RUSP.  The HHS Secretary affirmed the advisory committee's recommendation in July of this year.

But the journey to achieve universal screening did not end with the Committee's endorsement, or the HHS Secretary's subsequent approval.  The SMA Newborn Screening Coalition, led by Cure SMA and originally created to support inclusion of SMA on the RUSP, has now turned its focus to state newborn screening programs, encouraging each state to adopt the RUSP recommendation and screen all newborns for SMA.  Momentum is building for the adoption of the SMA screening recommendation.  To date, 4 states have adopted and implemented the recommendation, with pilot programs and significant legislative action occurring in more than 15 other states.

Though our work in newborn screening focuses on the rare disease SMA, our efforts also impact others in the rare disease community. Collaborating with our partners, we have sought to create a framework for other conditions seeking inclusion on the RUSP.  We advocated for the passage of the Newborn Screening Saves Lives Act, which reauthorizes grants for use in revising and extending services related to newborn screening, streamlines the newborn screening process and helps train healthcare individuals. Passed in 2014, the Act ensures that infants are screened for devasting conditions and that their parents and healthcare providers can obtain the resources they need to make effective healthcare decisions. The Act also allows the federal government to undertake pilot studies to expand newborn screenings for heritable disorders nationwide.

Together with our partners in patient advocacy and the medical community, we have worked hand in hand to advocate for early diagnosis of disorders for infants, with the hope of making early access to potentially life-saving medical care and therapies a reality. These partnerships were vital to our recent success in adding SMA to the RUSP and will continue to be important as we identify, diagnose and aim to create innovative treatments for other rare diseases.

Alfred W. Sandrock, Jr., M.D., Ph.D., is Executive Vice President and Chief Medical Officer at Biogen, and has served on the Executive Committee since 2015. He was named Chief Medical Officer in 2012 and since joining the company in 1998, has held several senior executive positions, including Senior Vice President of Development Sciences, Senior Vice President of Neurology Research and Development, and Vice President of Clinical Development, Neurology. Dr. Sandrock received his B.A. in human biology from Stanford University, an M.D. from Harvard Medical School, and a Ph.D. in neurobiology from Harvard University. He completed an internship in medicine, a residency and chief residency in neurology, and a clinical fellowship in Neuromuscular Disease and Clinical Neurophysiology (electromyography) at Massachusetts General Hospital.

Watchdog Report Reaffirms Concerns With Fail First

A new report by the Office of Inspector General at the U.S. Department of Health and Human Services (HHS) reaffirms concerns with a regulatory decision that undermines the health and well-being of America's seniors.

In an abrupt reversal of long-standing policy, the Centers for Medicare & Medicaid Services (CMS) announced that Medicare Advantage health plans can impose "step therapy" requirements on patients who need drugs covered under Medicare Part B.

The new directive - which affects more than one-third of all Medicare beneficiaries - will force some patients to fail first on a medicine preferred by their insurance companies before they can receive the therapy prescribed by their doctors.

More than 230 patient and provider groups have warned the new policy "could put patients' health at risk" and impose greater administrative burdens on physicians.

But what does this have to do with the HHS Inspector General? Well, in a new report, the independent watchdog reveals that patients are often wrongly denied care by health plans participating in the Medicare Advantage program. According to the report:

• The "central concern" is that health plans may put profits before patients. The report notes "potential incentives" that encourage health plans to "inappropriately deny access to services and payment in an attempt to increase their own profit."
• Health plans routinely deny coverage, forcing patients to file appeals or forego the medical care they need. In fact, 75 percent of all coverage or payment denials issued by health plans were overturned at the first stage of the appeal process, which strongly suggests the insurance industry is taking a "deny first" approach to caring for America's seniors.
• Seniors face a "confusing and overwhelming" appeals process, which may explain why only 1 percent of coverage denials are actually appealed.
• The often inaccurate denials of coverage by health plans are particularly worrisome for seniors who rely on Medicare Advantage and "may be especially burdensome for beneficiaries with urgent health conditions."

These startling facts do not bode well for seniors, particularly as the Trump administration provides these same health plans new tools to restrict seniors' access to prescription medicines.

And now UnitedHealth-the largest insurance carrier in the Medicare Advantage program-is rushing to impose the flawed fail first approach on millions of seniors starting next year. As the Society for Women's Health Research cautioned in a recent letter, the current approach is flawed because it "lacks basic beneficiary protections and agency oversight that should be fundamental to any utilization management policy."

The Trump administration should reverse course and withdraw this harmful policy. At the very least, it should not allow insurance companies to rush forward until basic safeguards are in place that ensure seniors aren't wrongly denied the medicines they need.