Patient: Page 2


  • Capitol building, DC
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    Incentivizing rare disease R&D is getting tougher, but revamped policies could turn the tide

    Orphan drug development has long been incentivized by government policy. But as the financial math shifts, so do the risks of bringing rare disease drugs to market.

    By Feb. 25, 2025
  • A sign reading Food and Drug Administration is seen above a door to a government building.
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    Sarah Silbiger via Getty Images
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    In FDA job cuts, experts see threat of far-reaching impact

    "Any place that gets cut, it's going to have an impact, because there's not any spare personnel at FDA,” said former agency commissioner Robert Califf, of the layoffs.

    By Jonathan Gardner , Delilah Alvarado • Feb. 21, 2025
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    Trendline

    Oncology R&D

    Cancer research remains a prime focus for the industry and often leads to pharma’s most impactful breakthroughs.   

    By PharmaVoice staff
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    The kids are alright: How pharma can make inroads with Gen Z

    As drug development priorities shift, pharma companies need to understand younger patients to improve their reputation.

    By Feb. 12, 2025
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    Are orexins the next golden child of neuroscience?

    An emerging class of orexin-based drugs are on the cusp of disrupting care for sleep disorders — and potentially much more.

    By Feb. 10, 2025
  • A person holds two blue drug tablets in their left hand.
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    Permission granted by Vertex Pharmaceuticals
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    Deep Dive

    A new, non-opioid pain drug is here. Getting it to patients could be agony.

    After decades of research, Vertex Pharmaceuticals now has an approved pain medication. Can one of the world’s most powerful biotechs contend with a healthcare system that’s long favored opioids?

    By Jacob Bell • Feb. 3, 2025
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    Year in Preview

    Pharma’s forecast for 2025: Sowing seeds of a rebound

    Despite regulatory uncertainty, pharma is bouncing back from a market slump and is being fueled by innovation. 

    By Jan. 31, 2025
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    Stock via Getty Images
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    Gene therapies have been uneven for DMD — but these companies hope to turn the tide

    Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the belief the answer could come down to delivery.

    By Jan. 24, 2025
  • A special needs kid in a wheelchair smiling and being hugged by an adult
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    iStock.com/jarenwicklund

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    Sponsored by Accessia Health

    Beyond copays: Redefining assistance for those with rare diseases and chronic conditions

     Accessia Health helps ease the burden of managing a rare disease or chronic condition.

    Jan. 21, 2025
  • Elon Musk Capitol Hill
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    Anna Moneymaker via Getty Images
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    Musk’s rare disease torpedo, hospitals’ pharma fix and other policies leaders are tracking

    There’s more to pharma's regulatory story in 2025 than just the IRA.

    By Jan. 17, 2025
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    Opinion // Year in Preview

    PharmaVoice’s Crystal Ball: Industry shifts in R&D, policy and clinical trials

    Coming changes that could impact pharma from drug development to launch.

    By Jan. 10, 2025
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    iStock via Getty Images
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    Subcutaneous options offer convenience for patients — and a valuable window for drugmakers

    New formulations for under-the-skin application of drugs give pharmas another opening in a crowded market.

    By Kelly Bilodeau • Jan. 7, 2025
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    10 of our most read articles of 2024

    The high drama of an election year, industry layoffs, patent cliff strategies and other key trends that shaped pharma in 2024.

    By Dec. 20, 2024
  • A pregnant person is checked with a stethoscope in a doctor's office.
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    PeopleImages via Getty Images
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    Maternal mortality trends remain dire — and researchers face an uphill battle to solve them

    Rising maternal mortality, particularly among vulnerable populations, stems in part from a lack of research in pregnant women.

    By Dec. 17, 2024
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    Courtesy of Roche
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    3 closely watched drugs that failed in the clinic

    How recent setbacks and scattered successes could impact various sectors of the industry.

    By Kelly Bilodeau • Dec. 16, 2024
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    Will the ‘anti-woke’ movement derail DEI efforts in pharma?

    Amid the DEI backlash in corporate America, pharma leaders are quietly adjusting tactics.

    By Dec. 13, 2024
  • A person in a white coat points to an ultrasound image as another looks on.
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    Pony Wang via Getty Images
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    Deep Dive

    Endometriosis drug research, long underfunded, confronts familiar problems in women’s health

    Despite the disease’s prevalence, endometriosis remains misunderstood, and research into drugs that might treat it draws scant funding — problems that have deep roots.

    By Delilah Alvarado • Dec. 11, 2024
  • Scott Weintraub, senior vice president, U.S. business, Alexion
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    Permission granted by Alexion/AstraZeneca
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    How Alexion markets a rare disease drug by ‘starting with the end in mind’

    The biopharma, now an arm of AstraZeneca, needs to build the plane while flying it to be first in rare diseases with no other option.

    By Dec. 3, 2024
  • women mental health
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    Stock via Getty Images
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    Can the girl power of Arrivo’s depression drug help reverse its fortunes?

    After failing a mid-stage trial, the biotech shifted course and is now targeting females with the goal of proving gender can play a role in depression outcomes.

    By Kelly Bilodeau • Dec. 3, 2024
  • Pregnant women's health research
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    Getty Images via Getty Images
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    Specialized CROs aim to boost participation among women and other underrepresented groups

    Lindus Health’s women-focused CRO is the latest in a handful of research efforts to target specialized — and underrepresented — populations.

    By Alexandra Pecci • Nov. 27, 2024
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    Permission granted by Pfizer
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    Pfizer and Lilly’s telehealth prescribing platforms draw Senate scrutiny for ‘potential fraud’

    As Big Pharma embraces telehealth to sell medicines directly to patients, lawmakers wonder whether the programs skirt anti-kickback rules.

    By Kelly Bilodeau • Nov. 12, 2024
  • Jonathan Rigby, CEO, Sernova
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    Permission granted by Sernova
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    Profile

    CEO with type 1 diabetes works to deliver ‘world’s first’ functional cure

    Sernova’s technology helped type 1 diabetes patients achieve insulin independence in early- and mid-stage trials.

    By Alexandra Pecci • Nov. 5, 2024
  • A healthcare worker in blue scrubs talks with an elderly woman sitting next to a walker outdoors.
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    123RF/ Jozef Polc

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    Sponsored by CareMetx

    No more delays: Accelerating therapy starts by embedding hub services in provider workflows

    Integrating patient access programs with the systems healthcare providers use daily increases speed to therapy.

    By Shabbir Ahmed, Chief Commercial Officer at CareMetx • Nov. 4, 2024
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    Stock via Getty Images
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    A sickle cell cure exists. But patients need more than just gene therapy.

    Almost a year after two historic approvals, Pfizer is pulling a sickle cell disease treatment from the market, and the outlook for patients feels shakier.

    By Alexandra Pecci • Nov. 1, 2024
  • PV100 category rare disease warriors
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    Photo illustration: Industry Dive; Getty Images

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    PharmaVoice 100

    2024 PharmaVoice 100s: Rare Disease Warriors

    Pharma pros driving drug development and access for patients with high unmet needs. 

    By Oct. 29, 2024
  • Dengue vax
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    Buda Mendes via Getty Images
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    Dengue is spreading and Sanofi is pulling the U.S. market’s only vaccine. What’s next?

    As Sanofi prepares to halt production on its dengue vaccine in 2026, locally acquired infections in the U.S. are raising the alarm.

    By Alexandra Pecci • Oct. 23, 2024