NAVLIN Brief
Saudi Arabia is slimming down its reference basket from 20 countries to 16 countries, effective at the end of July 2022. The kingdom’s new reference basket will be comprised of Australia, Austria, Belgium, Canada, France, Germany, Hungary, Italy, Japan, Netherlands, Poland, Portugal, South Korea, Spain, Sweden, and the United Kingdom. Saudi Arabia conducts international reference pricing (IRP) every five years, using the lowest price among its reference countries to establish a price in the kingdom, although any price changes in its basket will trigger a price review.
On Thursday, the National Institute for Health and Care Excellence (NICE) hosted a webinar to announce the launch of its real-world evidence framework, which aims to identify when real-world data can be used to reduce uncertainties and improve guidance, and clearly describe best practices for planning, conducting and reporting real-world evidence studies to improve the quality and transparency of evidence. Kicking off the launch, Dr. Samantha Roberts, CEO of NICE explained that the document can “help us with monitoring the uptake of new technologies to make sure patients are benefitting to the extent we’ve seen in clinical trials. The framework aims to improve the quality of real-world evidence informing NICE’s guidance but does not set the minimum acceptable standards for the quality of evidence.”
Speaking at the 2022 VWV PING Conference, Dr Samantha Roberts, Chief Executive at the National Institute for Health and Care Excellence (NICE) offered a view into her “emerging thinking” on NICE’s current capabilities and future goals, having now held the role for five months. Last year NICE launched its 5-year strategy with an aim to keep ahead of the challenges of a “rapidly changing” health and care landscape, allowing NICE to evolve from producing full guidelines to adopting a more modular, living style of recommendations, enabling rapid updates that incorporate the latest evidence to reach healthcare professionals faster than ever before. Speaking at the event, Dr Roberts confirmed that NICE was “not going to rewrite” the plan, despite the disruption of COVID-19, noting “I completely agree with the direction of travel laid out in the plan.”
THE DETAILS
COVID-19
Speaking at the 2022 VWV PING Conference, Dr Samantha Roberts, Chief Executive at the National Institute for Health and Care Excellence (NICE) offered a view into her “emerging thinking” on NICE’s current capabilities and future goals, having now held the role for five months. Last year NICE launched its five-year strategy with an aim to keep ahead of the challenges of a “rapidly changing” health and care landscape, allowing NICE to evolve from producing full guidelines to adopting a more modular, living style of recommendations, enabling rapid updates that incorporate the latest evidence to reach healthcare professionals faster than ever before. Speaking at the event, Dr Roberts confirmed that NICE was “not going to rewrite” the plan, despite the disruption of COVID-19, noting “I completely agree with the direction of travel laid out in the plan.” NICE has its job cut out for it at the moment, given the record pressures placed on NHS England: “I’ve worked in healthcare for 35 years. I’ve never seen anything like this,” Dr Roberts added. “We currently have an incredibly overstretched NHS. We all need to be doing things in a very slick way where the patient benefit is clear. We at NICE need to adapt to accommodate these changes – we don’t want to be an analog service in a digital world.”
The World Health Organization’s (WHO) technical advisory group (TAG-CO-VAC) has said that a modified COVID-19 vaccine composition may be warranted to broaden immune protection against divergent SARS-CoV-2 S protein antigens. According to TAG-CO-VAC, citing available data, a variant-adapted vaccine may benefit those who have already received the primary series of shots – however, TAG-CO-VAC acknowledges that there are uncertainties as its position is based on limited data from animal models, inference from Omicron infection in primed and unprimed individuals, and preliminary clinical data in humans vaccinated with an Omicron vaccine candidate. To address uncertainties, TAG-CO-VAC stated that it strongly encourages the generation of clinical data on immune responses in humans to a primary series and/or booster dose of Omicron-specific vaccines across different vaccine platforms.
The European Medicines Agency has begun a rolling review of Moderna’s adapted COVID-19 vaccine, Spikevax, which aims to provide better protection against specific variants of SARS-CoV-2.
The World Trade Organization (WTO) members have unveiled a new package of trade accords, endorsing a Trade-Related Aspects of Intellectual Property Rights (TRIPS) waiver for COVID-19 vaccines. Under an arrangement known as compulsory licensing, WTO members agreed to a vaccine patent waiver to boost supplies of COVID-19 shots to lower-income countries.
Infants and children from six months to five years of age will have access to COVID-19 vaccines in the U.S. this week, following the Director of the Centers for Disease Control and Prevention (CDC) endorsement for this age group. Receiving a COVID-19 vaccine protects younger children from severe disease and lasting complications not yet well-understood, according to the agency’s advisory panel.
With support from mRNA specialist eTheRNA, Belgian-based Univercells is partnering with Afrigen Biologics to develop a novel mRNA vaccine using intellectual property from collaboration partners, to help produce the first Africa-owned COVID-19 vaccine. Their collaboration aims to address major challenges to rolling out COVID-19 vaccines in Africa, in particular the lack of local cost-effectiveness production and the need for cold- and super-cold chains. The agreement will also pioneer a new model of manufacturing mRNA vaccines, as Quantoom Biosciences, a Univercells company, is developing an mRNA production technology that encompasses all steps of RNA production, with distributed and decentralized manufacturing in mind to allow for large-scale production.
POLICY
Saudi Arabia is slimming down its reference basket from 20 countries to 16 countries, effective at the end of July 2022. The kingdom’s new reference basket will be comprised of Australia, Austria, Belgium, Canada, France, Germany, Hungary, Italy, Japan, Netherlands, Poland, Portugal, South Korea, Spain, Sweden, and the United Kingdom. Saudi Arabia conducts international reference pricing (IRP) every five years, using the lowest price among its reference countries to establish a price in the kingdom, although any price changes in its basket will trigger a price review.
Pharma companies operating in Italy could face fines if they fail to comply with a new law No 62/22 on “transparency rules on relationships between manufacturing companies, healthcare professionals and organizations,” dubbed the “Sunshine Act.” Earlier this year, the bill was approved and is set to come into force on June 26 – the Sunshine Act is inspired by the legislation of the United States, where, in 2010, the Physician Payments Sunshine Act (PPSA) was approved in order to increase the transparency of financial relationships between healthcare-related professionals, healthcare organizations and pharmaceutical manufacturers. Law No 62/22 will replace the existing self-regulating code of ethics dictated by the associations, which directed member companies to disclose all transfers of value, directly or indirectly, between healthcare professionals, organizations, and certain third parties on their websites.
Generics sold from Mark Cuban’s Cost Plus Drug Company could have offered Medicare $4 billion in savings in 2020, according to a new study in the journal Annals of Internal Medicine. The report lays out a comparison between the prices of 89 generics offered by Cost Plus Drug in 2022 and the prices Medicare Part D plans paid in 2020. Fluctuations in drug costs between 2020 and 2022 were taken into account, with researchers finding that the $8.1 billion price tag offered to Medicare could have been $4.5 billion if it had sourced 77 generic drugs from Cost Plus.
According to the Dutch Patient Federation, the proposed GVS “modernization” is “so complicated that it is better to wait to introduce” the amendments. In a letter dated June 20, sent to the House of Representatives, the Federation “pleads” for postponement of the initiative, as it believes that “Only postponing the introduction can ensure that patients and their organizations have enough time to think about the reimbursement system (GVS).” The letter also urges the reorganization to properly involve and weigh the experiences of patients when assessing the use of medicines.
Senators Susan Collins (R-ME) and Jeanne Shaheen (D-NH) have unveiled the “Improving Needed Safeguards for Users of Lifesaving Insulin Now Act,” dubbed the “INSULIN Act.” The INSULIN Act would lower list prices to 2021 net Medicare rates for plans beginning on or after January 1, 2024 and prevent insurance plans and Pharmacy Benefit Managers (PBMs) from collecting rebates on insulins that limit list price to the 2021 net prices for Medicare Part D or equivalent levels.
HTA
The Federal Joint Committee (G-BA) has given Deciphera Pharmaceutical’s Qinlock (ripretinib) a rating of “major additional benefit” for the treatment of adults with gastrointestinal stromal tumor (GIST) who have already been treated with three or more medicines of the ‘kinase inhibitor’ class, including a drug called imatinib. The rating is backed by the results of the Phase 3 INVICTUS study, which compared Qinlock in combination with Best Supportive Care (BSC) versus placebo and BSC. According to G-BA, the price of Qinlock is EUR 24,903.40 after applying statutory discounts. Qinlock received a marketing authorization valid throughout the EU on November 18, 2021.
In a flurry of decisions posted to the Federal Joint Committee of Germany (G-BA)’s website, the group has determined that the added benefit of a number of drugs – Zeposia, Forxiga and Repatha – is “not proven.” First, the organization determined that the added benefit of Zeposia, when indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response or are intolerant to conventional therapy or a biologic, is “not proven.” Next, G-BA posted a decision regarding Repatha for the treatment of a number if indications and ages, and finally assessed the use of Forxiga for the treatment of insulin-naïve children and adolescents aged 10 to 17 years with diabetes mellitus type 2 who have not achieved adequate glycemic control with their previous drug therapy consisting of at least one blood-sugar-lowering drug in addition to diet and exercise, and patients who have not achieved adequate glycemic control with their previous insulin regimen in addition to diet and exercise.
The National Institute for Health and Care Excellence (NICE) has released a new management guideline for multiple sclerosis in adults, rejecting the use of Fampyra (fampridine) as it is “not cost-effective at the current list price.” Despite acknowledging in the guidance that “Fampridine is a clinically effective treatment for some people,” NICE is sticking to its draft guidance from December 2021 in which it rejects the use of the treatment. Fampyra is already approved for NHS use in Scotland and Wales for eligible people with MS but cannot be used in England. Phillip Anderson, head of policy at the MS Society, called the decision a “crushing blow” to many in the MS community.
The Federal Joint Committee of Germany (G-BA) has said that the added benefit of Abecma (idecabtagene vicleucel) is “non-quantifiable” for the treatment of adults with multiple myeloma (MM) who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti‑CD38 antibody and whose disease has worsened since the last treatment.
PRICING & REIMBURSEMENT
On Thursday, the National Institute for Health and Care Excellence (NICE) hosted a webinar to announce the launch of its real-world evidence framework, which aims to identify when real-world data can be used to reduce uncertainties and improve guidance, and clearly describe best practices for planning, conducting and reporting real-world evidence studies to improve the quality and transparency of evidence. Kicking off the launch, Dr. Samantha Roberts, CEO of NICE explained that the document is a “first draft, and as you can imagine it’s a living document. There is a ubiquity of data unlike anything we have seen before and health systems are under increasing pressure. It is not unreasonable to believe that RWE will have a real role in solving these tricky problems… It can help us with monitoring the uptake of new technologies to make sure patients are benefitting to the extent we’ve seen in clinical trials. The framework aims to improve the quality of real-world evidence informing NICE’s guidance but does not set the minimum acceptable standards for the quality of evidence.
The Shanghai Medical Procurement All-in-One (SMPA) has announced the bidding and procurement rules for China’s seventh round of volume-based procurement (VBP), which includes 61 drug varieties across 147 specifications (downloadable PDF list of products available in article) and will take place on July 12, 2022. Allowing a maximum of 10 manufacturers to be shortlisted, the rules detail pricing stipulations and supply details for both main and standby supplies to China’s provinces. For the sixth round of VBP, which focused solely on insulin, China selected 42 insulin products from 11 companies; in total, 210 million doses of insulin were bought, with an average price cut of 48%, according to the National Healthcare Security Administration (NHSA).
According to local media site, Polityka Zdrowotna, the Polish Health Ministry is discussing potential changes to the reimbursement act so that the ministry itself would cover popular drugs with reimbursement, without waiting for a move from the manufacturer. As it stands, for the drug to be reimbursed, the company has to report to the ministry and pay for it. However, “We want certain basic preparations to be reimbursed to a greater extent than before,” explained the Deputy Minister of Health Maciej Miłkowski in an interview with DGP. He added, “This rule is also to apply to those drugs that have been present on our market for a long time.”
DRUG APPROVAL
The European Commission (EC) has approved Amryt’s Filsuvez – formerly oleogel-S10 – for the treatment of partial thickness wounds associated with dystrophic and junctional Epidermolysis Bullosa (EB) in patients from six months of age.
The European Commission (EC) has authorized a variation for Rhythm Pharmaceuticals’ Imcivree (setmelanotide) that allows for dosing in patients with loss-of-function biallelic pro-opiomelanocortin (POMC) or biallelic leptin receptor (LEPR) deficiency who have mild, moderate, or severe renal impairment.
The U.S. Food and Drug Administration (FDA) has approved an expanded indication for Merck’s Vaxneuvance (Pneumococcal 15-valent Conjugate Vaccine) to include children six weeks through 17 years of age.
The European Commission (EC) has approved Novartis’ Tabrecta (capmatinib) as a monotherapy for the treatment of adults with advanced non-small cell lung cancer (NSCLC) harboring alterations leading to mesenchymal-epithelial-transition factor gene (MET) exon 14 (METex14) skipping who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy.
The European Medicines Agency (EMA) has granted Orphan Designation to Midatech’s MTX110 for the treatment of recurrent glioblastoma (rGBM) in adult patients and diffuse intrinsic pontine glioma (DIPG) and medulloblastoma in pediatric patients.
The biopharmaceutical company, Hepion Pharmaceuticals, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its orally administered novel cyclophilin inhibitor, rencofilstat, for the treatment of hepatocellular carcinoma (HCC).
Malaysia’s National Pharmaceutical Regulatory Agency (NPRA) has approved Roche’s Evrysdi (risdiplam) as the country’s first drug used to treat spinal muscular atrophy (SMA) in adults and children two months and older.
Learn more about NAVLIN by EVERSANA and our Global Pricing Solutions.
Contact us with your questions and global pricing needs, and an expert will follow up shortly.