Gepast gebruik én een passende organisatie van de zorg – samen gepaste zorg – zijn hard nodig voor het behoud van betaalbare, kwalitatief hoogstaande en toegankelijke zorg in Nederland. De Nederlandse Zorgautoriteit (NZa) en het Zorginstituut (ZIN) presenteren daarom deze week een advies en actieplan in één. Goed voor een efficiëntieslag binnen het bestaande systeem, maar wat HollandBIO betreft draaien we nog teveel om de hete brij heen: passende zorg vraagt geen evolutie, maar revolutie!

Ook HollandBIO ziet al jaren verspilling in de zorg, doordat patiënten niet altijd (gelijk) de juiste behandeling ontvangen. Wat zijn we daarom blij met de aanbeveling van de NZa en ZIN dat de gezondheid en het functioneren van het individu het nieuwe uitgangspunt moet worden. Alleen botst dit pontificaal met onze huidige manier van evidence based denken, waarbij we alleen behandelingen toelaten die gemiddeld genomen in een gestandaardiseerde groep effect laten zien. Hoe gaan we deze patstelling oplossen?

Om verspilling tegen te gaan, gaat ZIN onder meer bewezen niet-effectieve zorg uit het basispakket halen. HollandBIO vraagt zich af hoe dit in de praktijk uitpakt. Want wat gaat ZIN doen als een middel in de ene patiënt niks doet, maar bij een ander wel aanslaat? Blijven we varen op de evidence based aanpak op groepsniveau, of komt er – in lijn met de bovengenoemde ambitie – ruimte voor een meer gepersonaliseerde afweging?

In plaats van de evolutie in het tegengaan van verspilling en het meer op het individu inzetten en vergoeden van behandelingen, heeft HollandBIO meer vertrouwen in de bio(tech)revolutie, zoals eerder beschreven door McKinsey. De aankomende jaren zullen we dankzij technieken als Whole Genome Sequencing steeds beter kunnen voorspellen waarom de ene persoon ziek wordt, en de ander niet. Waarom een bepaald geneesmiddel in de ene persoon wel werkt en in de ander niet. Data is de sleutel tot succes, en het hebben van brede registers op patiëntniveau een randvoorwaarde. Daarom zou Nederland partijen die bijdragen aan het inzamelen, vastleggen en analyseren van deze data met een rode loper moeten onthalen. Juist deze spannende innovaties laat het rapport buiten beschouwing – en dus staat HollandBIO te trappelen om dit perspectief alsnog ter tafel te brengen!

Lees hier het advies en actieplan van NZa en ZIN: https://www.nza.nl/actueel/nieuws/2020/11/30/zorginstituut-en-nza-schetsen-in-advies-randvoorwaarden-passende-zorg

The European Commission (EC) has approved MenQuadfi^® for active immunization of individuals from the age of 12 months and older against invasive meningococcal disease caused by Neisseria meningitidis serogroups A, C, W and Y.⁹

“Meningococcal meningitis can take one’s life in as little as one day and leave survivors with severe permanent disabilities.^10,11 In Europe, there were more than 3,000 cases of Invasive Meningococcal Disease in 2018, half of them caused by serogroups C, W and Y,”⁸ says Thomas Triomphe, Head of Sanofi Pasteur. “One case is one too many. It is our ambition to make this vaccine available worldwide to further expand protection to as many people as possible. The European Commission’s approval of MenQuadfi takes us one step closer to achieving this goal.”

The European Commission’s decision is based upon results from a robust and comprehensive international clinical program, including seven pivotal Phase 2 and 3 randomized, active-controlled, multi-center studies. The immunogenicity and safety of MenQuadfi were evaluated in over 6,300 healthy individuals aged 12 months and older, who received a single dose of MenQuadfi.^{1,2,3,4,5,6,7}

MenQuadfi was compared with other licensed combination vaccines across all age groups. It demonstrated a good safety profile and induced a high immune response against all four serogroups (A, C, W and Y) consistently across all studies.^{1,2,3,4,5,6,7}

“The introduction of a new vaccine against four of the major serogroups of meningococcal disease is very welcome news. The disease is unpredictable and remains the biggest cause of sepsis and septic shock in children across Europe today,¹²” says Professor Federico Martinón-Torres, Pediatrician and Clinical Researcher, Head of Pediatrics and Vaccine Research Unit at Hospital Clínico Universitario de Santiago in Spain. “Meningococcal disease is vaccine-preventable but, in spite of its threat, there is currently no common immunization schedule for it in Europe. The approval of MenQuadfi in Europe will contribute to our efforts to protect against, and help defeat, this truly devastating disease.”

In order to better address the global need for meningococcal disease prevention over the life course, Phase 3 studies are ongoing to investigate the vaccine in infants from 6 weeks of age.^{13,14,15,16,17,18}

Invasive meningococcal disease (IMD) epidemiology is highly unpredictable and varies widely across geographies and over time. In Europe, with the increase in incidence of IMD caused by hypervirulent serogroup W, several countries have introduced MenACWY conjugate vaccination into their routine vaccination schedules. However, considerable variation remains between European countries, leaving room for outbreaks in unprotected and vulnerable populations.¹⁹

In 2018, 3,233 individuals contracted invasive meningococcal disease in Europe, and approximately 1 in 10 did not survive. Of the total number of cases, 2,911 were reported to be serogroups B, C, W or Y, of which almost half (47%) were serogroup C, W or Y.⁸ Rates were highest in infants, followed by children under 5 years, with a second peak in those aged 15–24 years.⁸

Source: Sanofi (Press release)

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced that the European Commission (EC) has granted marketing authorization for OXLUMO™ (lumasiran), an RNAi therapeutic, for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups.

PH1 is an ultra-rare orphan disease characterized by excessive oxalate production, which can lead to life threatening end-stage renal disease (ESRD) and other systemic complications. Heterogeneity in disease manifestation often contributes to delays in diagnosis – particularly in adult PH1 patients, with a median time from symptoms onset to diagnosis of approximately six years. Untreated PH1 leads to progressive kidney damage; patients with advanced kidney disease require intensive dialysis to help filter waste products, including oxalate, from their blood until they are able and eligible to receive a dual or sequential liver/kidney transplant, an invasive procedure associated with a high risk of morbidity and mortality, and life-long immunosuppression.

“Prior to now there have been no approved treatment options for PH1 in Europe, so this is a potentially life-changing milestone for people diagnosed with this ultra-rare, debilitating disease – many of whom are infants and children – and their families. Lumasiran will address the urgent unmet need that exists for patients with PH1 and its approval today marks our continued commitment to rare disease communities,” said John Maraganore, Ph.D., Chief Executive Officer, Alnylam Pharmaceuticals. “Alnylam has taken lumasiran from identification of compound to regulatory approval in just six years and we will progress with the same sense of urgency as we work with national reimbursement bodies across Europe to bring lumasiran to patients.”

Lumasiran is an RNAi therapeutic targeting the hydroxyacid oxidase 1 (HAO1) mRNA that encodes glycolate oxidase (GO) – an enzyme upstream of the disease-causing defect in PH1. By degrading the HAO1mRNA and reducing the synthesis of GO, lumasiran stops the production of oxalate – the toxic metabolite that directly contributes to the clinical manifestations of PH1.

“PH1 affects patients of all ages. It is particularly devastating when infants are born with the condition and develop kidney failure within the first few months of life. PH1 patients develop kidney stones from the overproduction of oxalate, and in many we see a progressive decline in kidney function, which can ultimately lead to life-threatening end-stage kidney disease. Until recently the only treatment options available have been combined liver and kidney transplantation, with vitamin B6 slowing down kidney failure in a limited number of sensitive patients,” said Sally-Anne Hulton, M.D., Consultant Paediatric Nephrologist, Birmingham Women’s and Children’s Hospital NHS Trust, UK. “For the first time, lumasiran provides those of us treating PH1 children and adults with a new therapeutic option to tackle the root cause of this disease and prevent the production of oxalate. The data show meaningful and sustained reductions in urinary and plasma oxalate with an encouraging safety and tolerability profile, providing us with hope for improving care for these patients.”

“In line with our Patient Access Philosophy, Alnylam is committed to being as innovative commercially as we have been scientifically,” said Brendan Martin, Acting Head of CEMEA, Alnylam. “While the ability to enter into innovative agreements varies by country and local regulations, we intend to work with health authorities across Europe to achieve responsible and sustainable access arrangements for lumasiran that address the diverse patient population affected by PH1, which ranges from infants to adults, and we will adapt to the local context. Our goal is to ensure that all patients in need have access to lumasiran while minimizing budget uncertainty for health services.”

The approval in the European Union is based on efficacy and safety findings from both the ILLUMINATE-A and ILLUMINATE-B Phase 3 studies of lumasiran. In the ILLUMINATE-A study conducted in adults and children six years or older, lumasiran achieved the primary endpoint with a 53 percent mean reduction in urinary oxalate relative to placebo and showed a 65 percent mean reduction in urinary oxalate relative to baseline. Eighty-four percent of patients achieved normal¹ or near-normal² levels of urinary oxalate and more than half of patients (52 percent) reached normalization, compared to zero percent in the placebo group. Findings from the ILLUMINATE-A pivotal study were presented in June 2020 at the virtual European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress. In the ILLUMINATE-B Phase 3 study, the efficacy results and safety profile of lumasiran in infants and children under the age of six years were found to be similar to those observed in ILLUMINATE-A. Results from the ILLUMINATE-B pediatric study were presented on October 22 at the virtual American Society of Nephrology (ASN) Annual Congress.

Lumasiran was granted Priority Medicines (PRIME) designation by the EMA as well as Orphan Designation in the European Union. Lumasiran was also granted an Accelerated Assessment by the EMA, which is awarded to medicines deemed to be of major public health interest and therapeutic innovation and is designed to bring new treatments to patients more quickly. This approval in the European Union follows the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in October 2020. Alnylam has filed a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA). The FDA has granted a Priority Review for the NDA and has set an action date of December 3, 2020 under the Prescription Drug User Fee Act (PDUFA).

Source: Alnylam (Press release)