Global Pricing Insights – Last Week In Review – June 13 – 17, 2022

NAVLIN Brief

This past week, the U.S. House of Representatives passed H.R. 7667, the Food and Drug Amendments of 2022, which reauthorizes the Food and Drug Administration’s (FDA) user fee agreements for drugs and medical devices. A main component of the Act, H.R. 7667 would empower the FDA to remove from the market drugs that were granted accelerated approval if they don’t deliver on their promised clinical benefit.

The Senate Health, Education, Labor, and Pensions (HELP) Committee has passed the S. 4348, the FDA Safety and Landmark Advancements (FDASLA) Act for safe prescription drug importation from Canada. FDASLA Act would remove barriers that have prevented personal importation in the past and pave the way for FDA to establish a program for importation of prescription drugs for personal use; build on work FDA has begun to expand commercial importation of less costly prescription drugs from Canada by states, Indian Tribes, and other groups; and give FDA the tools it needs to quickly address any importation program that poses a risk to public health and safety. The Act would also reauthorize FDA’s prescription drug, generic drug, biosimilar, and medical device user fee programs, modernize and accelerate approval regulations, in particular real-world evidence (RWE) use and parameters for post-approval studies, and establish a rare disease pilot program, with the aim to advance drugs

Shionogi has officially signed an agreement with NHS England to begin an innovative subscription payment model reimbursement for Fetcroja (cefiderocol) in England. Under the terms of the agreement, Shionogi will be paid a fixed sum for antimicrobials based on a health technology assessment of their value to the NHS, rather than the volumes used. In April, the National Institute for Health and Care Excellence (NICE) published draft guidance estimating the value of Fetcroja and Zavicefta (ceftazidime–avibactam) on the country’s NHS, as part of the new initiative with NHS England, NHS Improvement and the Department of Health and Social Care (DHSC) to incentivize antimicrobial resistance (AMR) research. NICE is the first health technology assessment organization anywhere in the world to attempt to estimate the full value of an antimicrobial in this way.

THE DETAILS

COVID-19

The Italian Medicines Agency’s (AIFA) Technical Scientific Commission (CTS) has recommended extending the indication of AstraZeneca’s COVID-19 drug, Evusheld (tixagevimab / cilgavimab), to include people who are at high-risk of severe COVID-19 disease. According to AIFA, a clinician will decide on treatment with the drug based on individual patient assessments. The CTS will also continue to evaluate the efficacy and duration of the protective effect of the monoclonal antibody combination also with respect to the evolution of the epidemiological picture and circulating variants.

European Union (EU) Member States have increased pressure on Pfizer and other COVID-19 vaccine makers to renegotiate contracts. As reported, several EU Member States sent a formal request to the European Commission (EC) to review the terms of the contracts concluded with the manufacturers of COVID-19 vaccines to make them more flexible and allow Member States to receive vaccines according to their needs. Poland, the leading country in this attempt to revise contracts, has more than 30 million COVID vaccines in stock and would need to buy another 70 million under existing agreements, a Polish diplomat told Reuters.

The European Medicines Agency has begun a rolling review of Pfizer-BioNTech’s adapted Comirnaty vaccine, which aims to provide better protection against a specific variant or variants of SARS-CoV-2, the virus that causes COVID-19. Pfizer and BioNTech said in a joint statement that they are evaluating several variant-adapted vaccines, adding that “the composition of the variant-adapted vaccine will be discussed with global regulatory bodies to determine a potential vaccine booster approach for the Fall 2022 season.” Meanwhile, in a separate statement, the EMA said that the review would initially focus on chemistry, manufacturing and controls (CMC) which relate to the manufacturing of the vaccine.

The European Health Emergency Preparedness and Response Authority (HERA) has ordered 110,000 doses of Bavarian Nordic’s MVA-BN smallpox/monkeypox vaccine, so that European Union (EU) Member States, Norway and Iceland can distribute the vaccine in response to the current monkeypox outbreak. According to the company, deliveries of vaccines to HERA will commence immediately and will be completed “during the next months.”

POLICY

This past week, the U.S. House of Representatives passed H.R. 7667, the Food and Drug Amendments of 2022, which reauthorizes the Food and Drug Administration’s (FDA) user fee agreements for drugs and medical devices. A main component of the Act, H.R. 7667 would empower the FDA to remove from the market drugs that were granted accelerated approval if they don’t deliver on their promised clinical benefit. The Senate also has a similar bill in the works to revise and extend the user-fee programs for prescription drugs, medical devices, generic drugs, and biosimilar products – for the most part, the bills are quite similar, although the House introduced the provision regarding clinical trial diversity.

The Senate Health, Education, Labor, and Pensions (HELP) Committee has passed the S. 4348, the FDA Safety and Landmark Advancements (FDASLA) Act for safe prescription drug importation from Canada. FDASLA Act would remove barriers that have prevented personal importation in the past and pave the way for FDA to establish a program for importation of prescription drugs for personal use; build on work FDA has begun to expand commercial importation of less costly prescription drugs from Canada by states, Indian Tribes, and other groups; and give FDA the tools it needs to quickly address any importation program that poses a risk to public health and safety. The Act would also reauthorize FDA’s prescription drug, generic drug, biosimilar, and medical device user fee programs, modernize and accelerate approval regulations, in particular real-world evidence (RWE) use and parameters for post-approval studies, and establish a rare disease pilot program, with the aim to advance drugs.

Shionogi has officially signed an agreement with NHS England to begin an innovative subscription payment model reimbursement for Fetcroja (cefiderocol) in England. Under the terms of the agreement, Shionogi will be paid a fixed sum for antimicrobials based on a health technology assessment of their value to the NHS, rather than the volumes used. In April, the National Institute for Health and Care Excellence (NICE) published draft guidance estimating the value of Fetcroja and Zavicefta (ceftazidime–avibactam) on the country’s NHS, as part of the new initiative with NHS England, NHS Improvement and the Department of Health and Social Care (DHSC) to incentivize antimicrobial resistance (AMR) research. NICE is the first health technology assessment organization anywhere in the world to attempt to estimate the full value of an antimicrobial in this way; the resulting appraisal documents were then used to inform commercial discussions between NHS England, NHS Improvement and the companies to agree payment levels in the subscription-style contracts.

The National Healthcare Security Agency (NHSA) has released its plan for adjusting the National Reimbursement Drug List (NRDL) for 2022 and is now soliciting feedback until the end of the month. Last year’s NRDL (2021 version) included a total of 2,869 drug varieties – 1,486 western medicines and 1,374 traditional Chinese medicines (TCMs) – with 74 new listings covering 21 clinical groups, and 67 new drugs (or indications for already-listed products) were included through negotiations that resulted in an average price reduction of 61.71%. In addition to outlining eligibility criteria for 2022 NRDL listings, the NHSA posted its agenda: preparation stage during May-June 2022; declaration stage during July-August 2022; expert review stage in August 2022; negotiation stage during September-October 2022; and final results in November 2022.

HTA

The National Institute for Health and Care Excellence (NICE) has recommended AbbVie’s Venclyxto (venetoclax) for treating chronic lymphocytic leukaemia (CLL) in adults. The recommendation is backed by additional evidence collected as part of the Cancer Drugs Fund managed access agreement for Venclyxto for CLL.

The National Institute for Health and Care Excellence (NICE) has published guidance stating that it is unable to make a recommendation on Astellas’s Padcev (enfortumab vedotin). Padcev is a cancer medicine for treating adults with urothelial cancer (a cancer of the bladder and urinary tract) after platinum-containing chemotherapy and a PD-1 or PD-L1 inhibitor. NICE said that the company confirmed there is unlikely to be enough evidence that the technology is a cost-effective use of NHS resources in this population.

PRICING & REIMBURSEMENT

Argentinian health officials have met with representatives from Novartis to discuss setting up a risk-sharing agreement (RSA) for the company’s spinal muscular atrophy (SMA) gene therapy, Zolgensma (onasemnogene abeparvovec). At the meeting with Sonia Tarragona, Chief of Staff of the Ministry of Health, and Natalia Grinblat, Undersecretary of Medicines and Strategic Information, Novartis representatives presented a letter of intent for an RSA for Zolgensma. The health ministry previously entered into an agreement with Biogen stipulating reimbursement of its SMA drug, Spinraza (nusinersen) for those with certain types of SMA, with a cost reduction for the drug. 

The transparency committee (TC) of the French National Authority of Health (HAS) is in favor of reimbursing an indication extension of Kalydeco (ivacaftor) plus Kaftrio (ivacaftor/tezacaftor/elexacaftor) to include patients with cystic fibrosis aged six years and older, with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 

The High Health Authority (HAS) of France has published a favorable opinion for reimbursing Ryeqo (relugolix/ norethisterone/ estradiol) for the treatment of moderate to severe symptoms of uterine fibroids in adult women of childbearing age. In a release, HAS states that Ryeqo was superior to placebo in reducing fibroid-related bleeding, but in the absence of a study versus a clinically relevant comparator, it was not possible to give the therapy a rating in relation to the other medicinal treatments for the same indication. As such, HAS concluded that the medical service rendered (SMR) by Ryeqo is “Important” while the actual therapeutic benefit is “Absent V.”

Following the initiation of a $191 million rise in its medicines budget over the next couple of years, New Zealand’s Pharmaceutical Management Agency (Pharmac) announced the first wave of specific funding changes, planned for implementation from the start of July, this year. To start off, Pharmac said it will designate funds to back blood and breast cancer therapies as well as more funds for eight multiple sclerosis treatments. The agency is also easing access to HIV pre-exposure prophylaxis (PrEP) and post-exposure prophylaxis (PEP) through revisions to funding criteria.

DRUG APPROVAL

China’s National Medical Products Administration (NMPA) has approved Everest Medicines’ Trodelvy (sacituzumab govitecan or SG) for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation and rare pediatric disease designation to Ionis Pharmaceuticals’ ION582, an investigational antisense medicine for the treatment of Angelman syndrome, a rare neurogenetic disorder caused by the loss of function of the maternally inherited Ubiquitin Protein Ligase E3A (UBE3A) gene. ION582 targets UBE3A.

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved Boehringer Ingelheim’s Jardiance (empagliflozin) as a treatment for adults with symptomatic chronic heart failure. The approval in the UK means that Jardiance becomes the first approved treatment for all forms of symptomatic chronic heart failure, including those with heart failure with preserved ejection fraction (HFpEF), due to its effect on reducing the likelihood of people with heart failure to be admitted to hospital or die from cardiovascular causes.

The U.S. Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ Amvuttra (vutrisiran), an RNAi therapeutic administered via subcutaneous injection once every three months (quarterly) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has accepted for review the marketing authorization application (MAA) for aumolertinib, a third-generation epidermal growth factor receptor (EGFR) -tyrosine kinase inhibitor (TKI), in development for the first-line treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with activating EGFR mutations and for the treatment of adult patients with locally advanced or metastatic EGFR T790M mutation-positive NSCLC.

The U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for Jazz Pharmaceuticals and Redx’s JZP815, a pan-RAF inhibitor for the treatment of solid tumors and hematologic malignancies that contain mutations in the MAPK pathway, enabling Jazz to proceed with initiating a clinical trial for JZP815.

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