Laura Helbling
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Director of Research at Prevision Policy; Senior Writer for The RPM Report Covering pharma and biotech regulation and policy.
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Articles by Laura Helbling
Medicare Makes It Harder For Antibiotics To Win Enhanced Payment
Unconvinced that a special pathway is ‘the appropriate vehicle’ to incentivize antimicrobial development, the US CMS is ending a special pathway that made it easier for novel antibiotics to qualify for New Technology Add-On Payments. Payments had been below estimates. Antibiotic developers will likely be seeing a little less of this guy after the IPPS final rule.
Medicare Denies Being ‘Excessively Stringent’ In Reviews Of Add-On Payments
US CMS is defending its approach to determining eligibility for enhanced Medicare payments in the hospital setting. What sponsors sees as overly selective reviews is really just a result of steady advances in available therapy, the agency maintains. CMS argues it is not being stingy with NTAP approvals.
New US FDA Oncology Chief Highlights ‘Resilience’ In Changing Times
Angelo de Claro, the US FDA’s new head of oncology, praised the “institutional resilience” of the agency in the Oncology Center of Excellence’s annual report and said the office continues to “make a meaningful impact” during a time of change. Angelo de Claro said OCE continues to meaningfully impact patients despite the difficult times at the FDA.
ICER Panel Endorses COVID-19 Vaccines, But Not Enough Evidence To Distinguish Between Them
The group said Pfizer/BioNTech’s Comirnaty, Moderna’s Spikevax and mNexspike, and Sanofi’s Nuvaxovid, were effective and valuable, but questions remained about the value of the shots in 12- to 24-year-olds. ICER could play a larger role in vaccine policy given the ongoing issues with ACIP. (Shutterstock) Key Takeaways ICER’s New England Comparative Effectiveness Public Advisory Council said COVID-19 vaccines had intermediate long-term value for the money compared to no vaccination.
US FDA ‘Biomarker Incubator’ Pilot Will Explore Kidney Toxicity
CDER wants sponsors to share data on drug-induced kidney toxicity as part of a pilot program for a new “incubator” project to help qualify novel biomarkers, which could help accelerate their qualification. FDA officials want to aggregate biomarker data to help drug development. (Shutterstock) Key Takeaways The FDA wants sponsors to share data to advance the use of biomarkers of drug-induced kidney injury.
Innovation Incentives Among Senate Democrats’ Menu Of Drug Pricing Options
An outline of potential pricing policies released by Senate Democrats is intended to set an agenda should they flip the chamber in the midterm elections, but some could generate bipartisan support, including ideas to incentivize “high risk science.” Senate Democrats included incentives for “high-ri Senate Democrats want feedback on ways to incentivize high-risk science and lower drug prices.
US FDA Allergenics Staff Sees Single-Trial ‘Default’ As Helpful
The US FDA’s anti-allergy therapy review divisions are eager to support less burdensome development pathways for potential food allergy treatments, agency officials indicated during a recent “expert panel.” The FDA food allergy expert panel was the first with career FDA staff participating. (Shutterstock) Key Takeaways FDA career staff identified the FDA’s new single trial efficacy standard as potentially helpful for food allergy treatment development.
Insurance CEOs In The Hot Seat: House Hearings Emphasize Rx Access, Not Prices
The grilling of several leading US insurance executives by two House committees suggested one benefit from the Most Favored Nation pricing deals is that for one day, someone else was on the hot seat. Cigna CEO David Cordani testifies during a House Energy and Commerce Committee hearing on lowering health care costs.
CMS’ Literal Read Of Non-Opioid Pain Incentive Excludes Recently Approved Product
The FDA’s policies on analgesic labeling appear to be limiting the potential for novel non-opioid pain medicines to qualify for separate payments in Medicare after CMS said the indication must be for post-operative use. Vertex’s Journavx will not receive separate CMS payments under the NO PAIN Act. (Shutterstock) Key Takeaways The FDA’s analgesic labeling policies may be preventing some non-opioid medications from receiving federal reimbursement.
Generic Drug Onshoring: Bipartisan Support For Federal Procurement Reforms
A Senate hearing on the medical product supply chain suggests there is bipartisan support for ‘buy American’ policies for federal Rx purchases, and for country-of-origin labeling to help those policies expand to the private sector. Even with prominent bipartisan support, onshoring legislation does yet have much momentum.
Non-Opioid Analgesics Guidance On Chronic Pain: One Trial Plus Confirmatory Evidence May Be Enough
Alternative therapies designed to avoid, eliminate, or reduce the use of opioids may be eligible for Breakthrough Therapy Designation or Priority Review, the US FDA draft guidance says. The guidance breaks down the indication discussion into three groupings: condition-specific indications, group-specific indications, and general chronic pain claims.
Pakistan Adopts Global PIC/S GMP Guidelines In ‘Landmark Regulatory Reform’
The Drug Regulatory Authority of Pakistan has adopted a new benchmark for good manufacturing practices, ensuring that the quality and safety of medicines produced in Pakistan meet international standards.
US FDA Updates COVID-19 Vaccine Formulations, Revokes EUAs, ACIP Use Decision Next
All COVID-19 vaccines are labeled for individuals 65 years and older or those with risk factors, and Moderna’s Spikevax is the only vaccine indicated for children as young as six months now that Pfizer’s emergency use authorization has been rescinded. Revocation of the EUAs means COVID-19 vaccines now will be marketed under a single pathway.
Changes In Pediatric COVID-19 Vaccine Recommendations Causing Coverage Confusion
The CDC’s current shared clinical decision-making recommendation for vaccinating healthy children age six months and older should trigger full coverage under the Affordable Care Act, but is not always happening at pharmacies and doctors’ offices, an Epstein Becker attorney said. Changes in COVID-19 vaccination recommendations are causing confusion about reimbursement.
US Strategic Reserve Bulking Up: New Order Focuses On API Stockpile
The White House is directing the Health and Human Services Department to replenish stockpiles of active pharmaceutical ingredients for “especially critical” medicines. President Trump's executive order also requires ASPR to develop plans for a second strategic active pharmaceutical ingredients reserve.
Paratek Pharmaceuticals, Inc.
http://paratekpharma.com/ Industry Pharmaceuticals Vaccines Specialty Pharmaceuticals Biotechnology Other Names/Subsidiaries Novace Transcept Pharmaceuticals Scrip Paratek To Acquire Optinose, Expanding Portfolio Beyond Antibiotics 20 Mar 2025 • By Less than two years after being taken private by Gurnet Capital and Novo Holdings, Paratek Pharmaceuticals has agreed to a merger with Optinose that will expand its product portfolio beyond the anti Scrip Scrip Asks…What Does 2024 Hold For Biopharma?
BIOSECURE Act Returning, Privacy, Gain-of-Function Bills Coming, Senate Sponsor Says
BIOSECURE Act Returning, Privacy, Gain-of-Function Bills Coming, Senate Sponsor Says Legislation prohibiting work with certain Chinese biopharma firms will be reintroduced soon, Democratic Sen. Gary Peters said. And it will not be the only bill tied to international biotech competition and security concerns that will be considered. The updated BIOSECURE Act will include adjustments based on issues that arose when the legislation was moving in 2024. (Shutterstock)
US FDA Rare Disease Case Studies Provide Development Models For Sponsors
US FDA Rare Disease Case Studies Provide Development Models For Sponsors Sanofi’s Xenpozyme and Sentynyl’s Nulibry are the first two case studies the FDA is using to continue educating rare disease sponsors on best practices. The rare disease case studies continue FDA efforts to educate sponsors on the best practices for rare disease product development. (Shutterstock)
Vertex Non-Opioid Pain Drug Draws Bipartisan Support In House Hearing
Vertex Non-Opioid Pain Drug Draws Bipartisan Support In House Hearing Congress continues to offer broad support for encouraging alternatives to opioid pain medicine, including proposals to speed coverage of new agents like Vertex’ Journavx. Capitol Hill is pushing for better insurance coverage of non-opioid pain medications. (Shutterstock)
Dose Optimization And Rare Diseases: Doing More With Less
Dose Optimization And Rare Diseases: Doing More With Less US FDA officials said dose optimization for rare diseases should not be sacrificed in the name of speeding access to medicines, but is the oncology approach spreading to rare diseases? The FDA said dose optimization may be more important for rare diseases because the small patient populations limit the number of possible clinical trials. (Shutterstock)
Prior Authorization Reform: 2025 Could Be A Year Of Changes
Analysis Executive Summary Prior authorization reforms could emerge as an important under-the-radar area of drug policy next year. An upcoming Medicare proposed rule, the extraordinary uptake of weight loss therapies, and the launch of the overhauled Part D benefit design will all drive attention to the issue. Source: Shutterstock Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
US PBM Reform: ‘Bust Them Up’ A Theme For 2025?
Analysis Executive Summary Another House hearing again showcases the bipartisan desire to enact reforms to the US pharmacy benefit management sector. Legislators also sound ready to push for more significant actions than “transparency” measures would promise, but that means waiting until 2025. Source: Shutterstock Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
US Sickle Cell Gene Therapy Contracting Demo Attracting Interest From States
Analysis Executive Summary The US Medicaid agency says states representing more than 80% of sickle cell patients are interested in a centralized contracting program, but House Republicans want legislation, not administrative interventions, to shape value-based payment for curative therapies. Source: Shutterstock Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
US Medicare Agency Reconsidering Proposed Accelerated Approval Payment Demo
Analysis Executive Summary The US Medicare agency is not moving forward with a proposal to cut payments for drugs with accelerated approval status pending further discussions with the FDA about whether new regulatory authorities have addressed concerns about timely completion of confirmatory trials. Source: Shutterstock Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
US FDA Drug Center Tries New Approach To Old Consistency Problems
Analysis Executive Summary Drug developers should not expect the US FDA's new Quantitative Medicine Center of Excellence to dramatically impact the agency's long-standing problem of ensuring more consistent use of innovative regulatory approaches in application reviews. Source: Shutterstock Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
Drug Shortage Could Be Addressed By ‘Resilience Rating’ From Private Entity Under HHS Plan
Analysis Executive Summary US HHS is proposing a public-private partnership to assign ratings to manufacturers based on quality/resilience of supply, tied to a purchasing incentive program for providers. The non-agency approach may be key to overcoming industry and political objections. You may also be interested in... Existing Subscriber? Sign in to continue reading. New to Generics Bulletin? Request a free trial today!
Drug Shortage Could Be Addressed By ‘Resilience Rating’ From Private Entity Under HHS Plan
Analysis Executive Summary US HHS is proposing a public-private partnership to assign ratings to manufacturers based on quality/resilience of supply, tied to a purchasing incentive program for providers. The non-agency approach may be key to overcoming industry and political objections. You may also be interested in... Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
A Final Push For US Hepatitis C Eradication Plan In 2024
Analysis Executive Summary The Biden Administration is hoping that it can attach funding for a national hepatitis C ‘subscription’ program into the delayed fiscal 2024 appropriations bills. You may also be interested in... Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
A Final Push For US Hepatitis C Eradication Plan In 2024
Analysis Executive Summary The Biden Administration is hoping that it can attach funding for a national hepatitis C ‘subscription’ program into the delayed fiscal 2024 appropriations bills. You may also be interested in... Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
Menopausal Status Is Another Focus For US FDA Trial Inclusion Efforts
Analysis Executive Summary US FDA is emphasizing the need to assess the effects of menopause on drug safety and efficacy as a continuation of the agency’s efforts to encourage clinical trial sponsors to look more systematically for differences in clinical trial subpopulations. You may also be interested in... Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
Gene Therapy Cost Effectiveness: US ICER Supports Million-Dollar Prices
Analysis Executive Summary Institute for Clinical and Economic Review continues to assign high values to novel gene therapies, suggesting that Orchard’s pending ‘arsa-cel’ for MLD could be cost effective at a price as high as $4 million per treatment. You may also be interested in... Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
Gene Therapy Cost Effectiveness: US ICER Supports Million-Dollar Prices
Analysis Executive Summary Institute for Clinical and Economic Review continues to assign high values to novel gene therapies, suggesting that Orchard’s pending ‘arsa-cel’ for MLD could be cost effective at a price as high as $4 million per treatment. You may also be interested in... Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
Author Detail
Acccess Token : Refresh Token : Entitlement Claim : Pink Sheet is part of Pharma Intelligence UK Limited This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group. Existing Subscriber? Sign in to continue reading. New to Pink Sheet?
US FDA Outlines Psychedelic Drug Trial Principles, But It Won’t Be An Easy Trip
Analysis Executive Summary The US FDA is offering its first formal advice for potential approaches to develop psychedelic drugs for therapeutic indications. But it won’t be easy. You may also be interested in... Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
Antibiotic Payment Incentives: Advocates Still Looking For More
News Executive Summary After some success in enacting new regulatory and exclusivity incentives in the US over the past decade, antibiotic research advocates are urging action on the payment/reimbursement side. One start-up CEO argues that Medicare needs to rethink its approach to payment for hospital antibiotics. You may also be interested in... Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today!
Diversity Plans For US FDA: Oncology Experience Will Inform New Policies
US FDA will be transitioning from the 'diversity plans' outlined in recent guidance to the new, legislatively directed "diversity action plans" by the end of 2023. The oncology group is leading the way in gathering early perspectives on industry approaches.
US FDA Outlines Psychedelic Drug Trial Principles, But It Won’t Be An Easy Trip
28 Jul 2023 Analysis Executive Summary The US FDA is offering its first formal advice for potential approaches to develop psychedelic drugs for therapeutic indications. But it won’t be easy. You may also be interested in... As Australia takes the lead in the prescribing of psilocybin and MDMA for mental health disorders, the European Medicines Agency has revealed some details of its regulatory agenda in this area in a letter to members of the European Parliament.
US FDA Outlines Psychedelic Drug Trial Principles, But It Won’t Be An Easy Trip
28 Jul 2023 Analysis Executive Summary The US FDA is offering its first formal advice for potential approaches to develop psychedelic drugs for therapeutic indications. But it won’t be easy. You may also be interested in... As Australia takes the lead in the prescribing of psilocybin and MDMA for mental health disorders, the European Medicines Agency has revealed some details of its regulatory agenda in this area in a letter to members of the European Parliament.
Medicare Proposes Earlier Deadline For Rx Add-On Payment Candidates
19 Apr 2023 News Executive Summary Starting in fiscal year 2025, products would need to meet an earlier, May deadline for FDA approval in order to be eligible for enhanced payments in the Hospital Inpatient Prospective Payment System (IPPS). The change would give CMS more to handle the surge in NTAP applications. You may also be interested in...
Drug Shortages Back In US Congressional Spotlight
18 Apr 2023 Analysis Executive Summary Drug shortages were a significant focus of legislative attention in the US before the COVID-19 pandemic, and Congress is not likely to drop the issue now that the public health emergency is winding down. You may also be interested in... President Biden’s 100-day plan relies on Defense Production Act authorities to increase resilience of critical domestic pharmaceutical supplies.
Providers Still See Drawbacks To Opioid Education Mandates
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Mandatory Prescriber Education For Opioids; Is This The Time?
Ask The Analyst Email Print Bookmark 08 Nov 2021 Analysis Executive Summary US FDA is once again asking whether its opioid Risk Evaluation & Mitigation Strategy program for opioids should make continuing education requirements mandatory for prescribers. This time there may be no choice but to move forward with the plan. You may also be interested in... Many supporters believe Robert Califf checks all the boxes, but if nominated he would also face many familiar confirmation challenges.
Providers Still See Drawbacks To Opioid Education Mandates
Ask The Analyst Email Print Bookmark 08 Nov 2021 Analysis Executive Summary Participants in a US FDA meeting on opioid REMS did not treat a move to mandatory education as a foregone conclusion. Instead, they delved frequently into alternate approaches to enhance the education itself. You may also be interested in... US FDA is once again asking whether its opioid Risk Evaluation & Mitigation Strategy program for opioids should make continuing education requirements mandatory for prescribers.
Ex-Commissioners See Mostly Positives In Public View Of US FDA After COVID
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US FDA Exploring Options To Improve Study Of Drugs In Elderly
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US FDA Exploring Options To Improve Study Of Drugs In Elderly
Ask The Analyst Email Print Bookmark 08 Apr 2021 News Executive Summary New incentives will most likely not be the immediate focus, but working group to explore ideas on enhancing understanding of drug effects in elderly populations will likely be formed by US FDA. You may also be interested in...
New Tech Payment For Old Antibiotics: US Medicare Effort Brings Lots Of Interest
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New Tech Payment For Old Antibiotic: US Medicare Effort Brings Lots Of Interest
UsernamePublicRestriction Your username does not meet the requirements. Sorry - this email domain is not allowed. Sorry - public email accounts are not allowed. Please provide a work email address. An account with that username already exists. Unfortunately we've not been able to process your registration. Please contact support.
Mandatory Efficacy Trials: US FDA Affirms Broad Scope Of New Post-Market Authority
ASK THE ANALYST Email Print Bookmark 06 Nov 2019 Analysis Executive Summary Updated guidance on its authority to mandate postmarketing studies affirms that a new power to impose requirements to look for diminished efficacy reaches beyond the opioid drug class. Existing Subscriber? Sign in to continue reading. Need a specific report? 1000+ reports available Buy Reports New to Pink Sheet?
Now It Really Is Personal: US FDA 'Rushing' To Develop Policy For 'Bespoke Therapies'
ASK THE ANALYST Email Print Bookmark 12 Jun 2019 Analysis Executive Summary Recent cases of individual designed treatments have FDA playing a bit of catch up. The concept has been on the horizon, but now “bespoke therapies” are here. Existing Subscriber? Sign in to continue reading. New to Pink Sheet? Start a free trial today! Register for our free email digests: It's a perfect fit, but the US FDA needs a way to see if it measures up.
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