India’s NPPA Cuts Off-Patent Drug Ceiling Prices by Half
The National Pharmaceutical Pricing Authority (NPPA), India’s drug pricing agency, has announced it will cut the ceiling price of off-patent drugs by half to address profiteering in the pharmaceutical industry. The list of drugs covers much of the country’s National List of Essential Medicines (NLEM). Many pharma companies have lambasted the plan, saying it will negatively impact their profits.
US CMS Announces New Strategy to Lower Medicaid Drug Prices
The US Centers for Medicare and Medicaid Services (CMS) has announced a new proposal to help lower prescription drug prices for Medicaid. The rule would establish a survey for drugmakers about drug manufacturing costs and would ensure that Medicaid payments for therapeutics, especially high-price cell and gene therapies, are reasonable via the Medicaid Drug Rebate Program (MDRP).
CMS Using Real-World Evidence in Medicare Drug Pricing Negotiations
The US Centers for Medicare and Medicaid Services (CMS) noted in a recent guidance that it will consider real-world
US House Members Call on FDA to Address Chemotherapy Shortages
A bipartisan group of US House representatives is calling on the Food and Drug Administration (FDA) to address ongoing shortages of key chemotherapy drugs. In a letter written by Debbie Dingell (D-MI) and Tim Wahlberg (R-MI), lawmakers highlight the current shortages of cisplatin and carboplatin and point out they were either caused or exacerbated by a manufacturing hold last year on Intas Pharmaceuticals over quality issues.
FDA’s CDER Looking Towards Quicker Accelerated Approval Withdrawal Approach
The US Food and Drug Omnibus Reform Act (FDORA) empowered the Center for Drug Evaluation and Research (CDER) with the ability to expedite the withdrawal of drugs that were granted accelerated approval if confirmatory trials are not successful. The new process, which has yet to be rolled out, will be covered in a new guidance that will be up for public comment.
Value in Health Regional Affairs: Developing Generic Drugs for Rare Diseases
Orphan drug development in the US and EU languished for years due to high development costs and low sales until it was bolstered by legislative action. While this has brought powerful therapeutics to patients, these drugs are still prohibitively expensive. In a new Value in Health: Regional Issues article, learn about the challenges of developing generic orphan drugs and the value they could bring to patients and drugmakers.
US FDA Approves Gene Therapy for “Butterfly Children”
The US Food and Drug Administration (FDA) has approved Krystal Biotech’s Vyjuvek, the first gene therapy for dystrophic epidermolyis bullosa (DEB), a rare disease that leaves affected patients, dubbed “butterfly children” with skin that damages extremely easily and heals poorly. The treatment, which would cost $24,250 per dose, is applied to wounded skin, delivering a collogen gene that is missing in patients.
Orphan Drug Status Plays into Drug Pricing
Orphan drugs, treatments for rare diseases with limited therapeutic options, are usually less profitable for pharma companies due to high development costs and small patient populations. However, orphan drug designation (ODD) offers monetary incentives to companies developing drugs for rare diseases. As a result, ODD plays a role in pharma drug pricing decision-making.
Only Some US Prescription Drug Prices are 10X Higher than Other Countries
A common talking point in US drug pricing discussions is that US residents pay 10 times as much for prescription medicines than people in other nations. While this is true for many drugs, the picture is more complex. Making such an analysis is complicated by the comparator nations, the drugs studied, and whether they are brand name.
Price of Multiple Sclerosis Disease Modifying Therapies Keeps Creeping Up
Disease modifying therapies (DMTs) for multiple sclerosis (MS) have a significant positive impact on patient outcomes but are often expensive. A recent report by GlobalData finds that prices for MS DMTs are on a steady increase, with drugs like Tysabri by biogen or Gilenya by Novartis increasing by 12%. This impacts patients, according to many experts.
Patients Regularly Switch Between Psoriasis Biologics
A recent real-world data study published in the Journal of Dermatological Treatment finds that patients with psoriasis change up their biologic therapeutics. Claims data from nearly 8,000 patients taken revealed that, on average, 14.4% of patients changed biologics within 12 months of the study’s two year period, while over a quarter swapped by 24 months.
Older US Adults Skipping Meds Because of Price
A recently published study in JAMA Network OPEN found that at least 20% of adults 65 and up skipped their medication, cut back their dose, or took drugs intended for other patients due to financial reasons in 2022. The figure is a jump from 2016, when one in seven older patients reported skipping medications for the same reason. Many of those who did maintain their drug regimen faced significant financial hardships.
Talking 340B with Sara Hearn of RxBenefits
The US 340B program requires drugmakers to provide certain medications at a significant discount to safety-net care centers that provide care for patients in economically disadvantaged areas through Medicaid and Medicare Part B. In a new BenefitsPRO interview with Sarah Hearn of RxBenefits, learn how the 340B program has impacted manufacturers and commercial payers.
FDA Adcomm Tentatively Recommends Sarepta’s Duchenne Muscular Dystrophy Gene Therapy
An advisory committee for the US Food and Drug Administration (FDA) has voted 8 to 6 to recommend accelerated approval for Sarepta Therapeutics Duchenne muscular dystrophy gene therapy. The narrow vote comes despite concerns from several regulators about the treatment’s benefit-risk profile and data supporting the application.
NICE Recommends Expanding Approval for AstraZeneca’s Heart Failure Med
The UK’s National Institute for Health and Care Excellence (NICE) has recommended that Astrazeneca’s heart failure med Forixga (dapagliflozin) should have its indication expanded to adults regardless of the degree of their left ventricular ejection fraction (LVEF). The drug was previously indicated with patients with reduced LVEF of 40% or lower.
Real-World Data Backs SPRINT® PNS System for Pain Relief
Real-world data (RWD) published in Pain Physician found that SPR Therapeutics’ percutaneous peripheral nerve stimulation (PNS) device significantly reduced pain in patients. The data, which came from over 6,100 patients, showed that more than 70% of patients saw a 50% or greater reduction in their pain symptoms.
FTC Requests Information from Group Purchasers in PBM Probe
During its ongoing probe into the pharmacy benefit manager (PBM) industry, the US Federal Trade Commission (FTC) requested information regarding two group purchasing organizations, which negotiate discounts for PBMs. The two companies included Zinc Health and Ascent Health Services, adding to the growing list of companies interrogated in the probe.
US Court of Appeals Considering Limiting Access to Mifepristone
Comments from Republican-appointed federal judges on the US Court of Appeals for the Fifth Circuit reveal their openness to restricting access to mifepristone, a key component for medication abortions. The judges and the plaintiffs, a group of conservative care providers, noted that the drug is more dangerous than decades of real-world evidence (RWE) indicate. In addition, the judges’ comments revealed they were largely unaware of the approval process of the Food and Drug Administration (FDA) and did not understand how telehealth worked.
US Runs Out of J&J’s COVID-19 Vaccine
The US Centers for Disease Control and Prevention (CDC) has announced that the US is officially out of J&J’s COVID-19 vaccine. The final batches of the vaccine expired earlier this month and now the agency is ordering all remaining vials to be disposed of. The vaccine’s administration has long been restricted to certain groups with limited access to the more widely used mRNA vaccines.
PTC’s Phenylketonuria Treatment Meets Phase III Endpoint
PTC Therapeutics announced positive results from a phase III clinical trial for its drug sepiapterin in the treatment of phenylketonuria (PKU), a rare disease causing excessive accumulation of the amino acid phenylalanine. The drug met the primary endpoint of reducing blood levels of phenylalanine in both adult and pediatric PKU patients in the trial.
Real-World Evidence Study Backs Adalimumab for Childhood Uveitis
Newly published research in Ophthalmology and Therapy finds that adalimumab is effective and safe in the treatment of pediatric non-anterior uveitis, an inflammatory eye disease. The study, using real-world evidence (RWE) from 21 pediatric patients, found that all patients benefited from the therapy and half were deemed fully responsive, with no additional inflammation in the affected eyes.
Max Foundation and BeiGene to provide Free Brukinsa to 29 LMICs
The Max Foundation, a Seattle-based organization dedicated to health equity, is collaborating with the biotech BioGene to provide Brukinsa to patients with chronic lymphocytic leukemia (CLL) in 29 lower- and middle-income countries (LMICs). The program will last for 3 years and will also provide education on disease management and diagnosis at participating treatment sites.
India’s NPPA Announces New Price Caps for Similar Drugs Sold by Same Drugmaker
India’s National Pharmaceutical Pricing Authority (NPPA) has rolled out a new policy capping prices on similar drugs sold by the same pharma company to the price of the least expensive version. The agency made the move based on the Drug Price Control Order, which it used previously to make medical devices like stents and knee implants more affordable to patients.
Drugmakers Are Putting Family-Funded Rare Disease Therapeutic Research on Hold
Families of patients have often led funding for rare disease therapeutic development through donation campaigns. However, many drugmakers have put these programs on hold, with 26 rare disease projects shelved in the past 16 months. This has caused an outcry from patient advocates. Pharma analysts argue financial reasons are to blame.
NICE Recommends Gilead’s Bulevertide for Hepatitis Delta Virus
The UK’s National Institute for Health and Care Excellence (NICE) has announced its approval of Gilead Sciences’ bulevirtide for the treatment of hepatitis delta virus (HDV) in adults with compensated liver disease. This recommendation comes after the US Food and Drug Administration (FDA) shot down Gilead’s application for issues with manufacturing and delivery.
