Kees Wessels, Chef Zorg bij de Argumentenfabriek, duidt in Skipr-blog 4 recente publicaties over dure geneesmiddelen. Een feitelijk en to-the-point relaas, en daarmee onze leestip dubbel en dwars waard. Een feitelijke basis is onmisbaar in het verhitte debat rondom geneesmiddelenprijzen. De feiten laten zien dat de uitgaven per patiënt aan ziekenhuismedicijnen al jaren op rij dalen. En dat de uitgaven aan deze dure geneesmiddelen met ruim 2 miljard per jaar “slechts” 2,5 % van de totale zorguitgaven bedraagt. Kees Wessels: “Dat de zorgpremie stijgt door torenhoge geneesmiddelenprijzen lijkt mij moeilijk vol te houden.”
Dat
neemt niet weg dat we als maatschappij ons geld maar één keer uit kunnen geven,
en dus staat ook de betaalbaarheid van medicijnen onder druk. Daarom moeten we
werk maken van oplossingen waarbij gezondheidswinst, innovatie en
betaalbaarheid hand in hand gaan, zodat de zorg ook in toekomst betaalbaar en
toegankelijk blijft voor iedereen.
Voor wie op zoek is naar meer begrip over hoe de geneesmiddelenzorg in Nederland in elkaar steekt, is het boek: Zó werkt de geneesmiddelenzorg een must-read. In begrijpelijke taal en aan de hand van duidelijke illustraties maakt het boek onze complexe geneesmiddelenzorg inzichtelijk. HollandBIO werkte mee aan de uitgave.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-05-26 12:56:252020-05-26 12:56:26Leestip: dure geneesmiddelen, de feiten op een rij← #nieuws
De voortgang van het klinisch onderzoek in Nederland heeft een harde knauw heeft gekregen, zo blijkt uit de resultaten van een enquête van de Dutch Clinical Research Foundation (DCRF) naar de gevolgen van de corona-crisis op het klinisch onderzoek. 40% van de studies is tijdelijk stop gezet en 40% is in aangepaste vorm doorgegaan. Bijna 80% van de respondenten gaf aan dat het niet meer mogelijk was om nieuwe patiënten te includeren. Ook de financiële gevolgen zijn door ca. 33% van de respondenten als knelpunt geïdentificeerd.
Daarentegen, bleken medisch ethische toetsingscommissies (METC’s) ondanks de crisis in staat om hun werk te blijven uitvoeren en waren er nauwelijks meldingen van het niet in behandeling nemen van onderzoeksprotocollen of amendementen. De versnelde toestemmingsprocedures voor COVID-19 studies werd door verschillende partijen gezien als best-practice, waar we in de toekomst van kunnen leren.
SeraNovo has signed a second License Agreement with Carna Biosciences, Inc., a company engaged in the drug development of kinase inhibitors. Under the agreement, the companies will expand their existing collaboration and jointly develop an oral formulation of a new active ingredient with an enhanced bioavailability.
Utilising its proprietary Deep Eutectic
Solvent (DES) formulation platform, SeraNovo is formulating one of Carna’s
proprietary drugs to increase its oral bioavailability. The DES formulation
platform is based on GRAS excipients that are used for oral administration and
broadly used in the industry.
Niall Hodgins, Chief Executive Officer of SeraNovo commented: “After our initial licence announcement in October 2019, we are pleased to extend our relationship with Carna to include the formulation of another promising active ingredient. This expansion of our partnership with an industry leader is a great development towards wide spread adoption of our breakthrough formulation technology.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-05-19 15:41:092020-05-19 15:41:09SeraNovo Enters into a second License Agreement with Carna Biosciences← #nieuws
De afgelopen weken blikt de media regelmatig trots naar Leiden, waar Janssen Vaccines alle zeilen bijzet om zo snel mogelijk een coronavaccin te ontwikkelen. Janssen kan snel handelen, mede dankzij de eerdere aanschaf van Crucell’s AdVac en PER.C6 technologie. Technologie met Nederlandse wortels, zo lichten Dinko Valerio, Lex van der Eb en Ronald Brus in het Parool toe. Hoewel het geen geheim is dat de Nederlandse life sciences wetenschap van wereldklasse is, kunnen we de kennis daaruit volgens hen nog beter verzilveren.
Ook HollandBIO is ervan overtuigd dat
Nederland met de juiste aanpak internationaal kan aansluiten bij de kopgroep op
vaccin- en geneesmiddelenontwikkeling. Wij zetten daarom vol in op een
vruchtbare voedingsbodem voor het Nederlandse bedrijfsleven. Zo komen we tot
een ijzersterk innovatieklimaat waarin tech transfer soepel verloopt, waar voldoende
financiering is voor de doorgroei van start- en scale-ups en waar de successen
van de sector met oranjegevoel gevierd mogen worden. Want wie kennis zaait, zal
bedrijvigheid oogsten.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-05-19 13:09:222023-09-27 16:31:07Het oranjegevoel binnen de biotech← #nieuws
Het Zorginstituut beantwoordt de veel gestelde vragen rondom het beleid voorwaardelijke toelating weesgeneesmiddelen, conditionals en exceptionals. De voorwaardelijke toelating biedt de mogelijkheid om geregistreerde, veelbelovende geneesmiddelen voor ernstige, vaak zeldzame ziekten, waarvan de effectiviteit (nog) onvoldoende is aangetoond eerder beschikbaar te maken. Lees meer
Mymetics, a pioneer and leader in the research and development of virosome-based vaccines against life threatening and life disabling diseases, has signed a Research Collaboration Agreement with Baylor College of Medicine and Texas Children’s Center for Vaccine Development for evaluating their rationally designed recombinant protein-based SARS-CoV and SARS-CoV-2 antigens and combining them with Mymetics’ virosomes for the development of a safe and effective Covid-19 vaccine.
Since the end of April, Mymetics has started a project for the rapid development of a Covid-19 virosome-based vaccine and is thereby partnering with leading academic institutions to explore and select the best SARS-CoV antigens, which could not only be efficacious in protecting against Covid-19 but also safe, affordable and accessible for use in the global population.
Baylor College of Medicine in Texas is a leading institution with a track record for developing low cost, safe and effective vaccines against emerging and neglected tropical diseases through collaboration with the Texas Children’s Center for Vaccine Development. The vaccine center already has been successful in developing potential vaccine against SARS-CoV and with this expertise has already initiated development of a SARS-CoV-2 recombinant protein-based vaccine.
“Our vaccine center has traditionally focused on advancing recombinant protein-based vaccines, but we recognize that for them to be effective, appropriate delivery platforms need to be considered, especially to ensure long-term immunity. Through the collaboration with Mymetics we will evaluate the coupling of our COVID-19 vaccine candidates to their flexible carrier to bring a new dimension towards accelerating vaccines for global access,” said Dr. Maria Elena Bottazzi, Professor, Pediatrics & Molecular Virology & Microbiology at Baylor College of Medicine and co-director of Texas Children’s Hospital Center for Vaccine Development.
“There is no question that if we want to get back to a life as before and a normal functioning economy, the world needs the rapid development of a safe and efficacious vaccine against Covid-19. The only serious way to get there fast is to have global collaborations to bring cross functional expertise together, establish result focused projects to explore and select the best vaccine candidates coupled with safe platform technologies and move them forward to clinical trials,” said Ronald Kempers, CEO of Mymetics Corporation. “Mymetics virosomes have proven to be a safe, efficacious and flexible vaccine carrier with other infectious diseases and we believe, with the right antigens and the right collaborations, we can have a serious attempt at developing a virosome-based Covid-19 vaccine. We are very much looking forward to this collaboration with the team at Baylor College of Medicine.”
This collaboration is supported through the combined resources of Mymetics, Baylor and Texas’s Children’s Center for Vaccine Development and covers the initial preclinical development. To speed-up the development of a potential Covid-19 vaccine, Mymetics and Baylor are seeking additional funding opportunities to start in parallel the GMP manufacturing and preparations for clinical trials. The aim is to have a virosome-based Covid-19 vaccine candidate evaluated in humans within 12 months.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2020-05-18 14:37:002020-05-19 14:43:13Mymetics and Baylor College of Medicine start Research Collaboration for Virosome-based Covid-19 Vaccines← #nieuws
Lava Therapeutics has entered into a research and license agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to discover and develop novel bispecific antibodies to gamma-delta T cells for the treatment of cancer. The collaboration was facilitated by Johnson & Johnson Innovation.
“We are excited to enter into this collaboration with Janssen, a global innovator and leader in the development of new medicines,” said Stephen Hurly, chief executive officer of Lava Therapeutics. “We strongly believe in the strength of our bispecific gamma-delta T cell engager platform and are committed to creating highly potent, target-specific therapeutics with increased durability and safety over current T cell-based approaches.”
Under the terms of the agreement, Lava Therapeutics will perform discovery and product development activities, and is eligible to receive an undisclosed financial package consisting of an upfront payment and potential development and commercial milestones, and future tiered royalties.
Intravacc, one of the leading
translational research and development vaccine institutes with an extensive
track record in developing viral and bacterial vaccines, today
announced the development of three COVID-19 vaccine concepts. These
concepts are all based on Intravacc’s platform technologies, that have been
proven safe in humans.
“This
global COVID19 pandemic requires an international effort to understand the
complexity of the disease and to develop innovative COVID-19 emergency and conventional
vaccines”, said Dr.
Jan Groen CEO of Intravacc. “We also need to establish partnerships with
pharma for large scale manufacturing and global distribution of these
vaccines”.
To develop safe and
effective COVID-19 vaccines, Intravacc is focusing on emergency and
conventional vaccines. Intravacc’s emergency vaccine is designed to activate
cytotoxic T-cell responses. This emergency vaccine would reduce mortality
and morbidity and as a result reduces the pressure on the public health system.
For this vaccine Intravacc uses its outer membrane vesicle (OMV) technology.
For its conventional
COVID-19 vaccine Intravacc will use OMV’s as a carrier for spike proteins
of the virus to induce a protective antibody response. For the third concept
Intravacc’s approved Vero cell platform will be used to develop an intranasal
vector vaccine based on Newcastle disease virus, that can replicate in humans
but does not cause disease.
The expected development timelines for these COVID-19 vaccines to enter into phase 1 range from 9 to 21 months.
Gisteren verscheen de jaarlijksemonitor dure geneesmiddelen medisch-specialistische zorg 2020. Goed nieuws, want dankzij de introductie van nieuwe geneesmiddelen kunnen steeds meer patiënten rekenen op een behandeling. De keerzijde is dat hierdoor de uitgaven aan geneesmiddelen ook groeien. En helaas groeit onze zorgbudgetboom niet tot in de hemel. Daarom moet er nu gezamenlijk werk gemaakt wordt van oplossingen die bijdragen aan het beheersen van de stijgende uitgaven, zodat de medisch-specialistische zorg ook in de toekomst ook voor iedereen toegankelijk blijft. Lees het persbericht van de NZa hier.
MyoKardia has announced positive topline data from the company’s
Phase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of
patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM) (clinicaltrials.gov NCT03470545).
Mavacamten demonstrated a robust treatment effect: the primary and all
secondary endpoints of the EXPLORER trial were met with statistical
significance (p≤0.0006 for all endpoints). Mavacamten was well tolerated, and
meaningful improvements in symptoms, functional status and quality of life, as
well as reduction or elimination in obstruction of the left ventricle, were
observed among patients on treatment versus placebo.
“The extraordinary data from the EXPLORER pivotal trial confirm
mavacamten’s ability to relieve dynamic outflow obstruction, control symptoms
and improve quality of life in patients with hypertrophic
cardiomyopathy,” said Iacopo
Olivotto, M.D., Careggi
University Hospital and lead clinical investigator for the
EXPLORER-HCM clinical trial. “HCM is the most common inherited
cardiovascular disease, and patients face an uncertain journey that all too frequently
includes debilitating symptoms, as well as serious complications, such as heart
failure, stroke and cardiac arrest. Mavacamten is the first drug
developed to target the specific molecular defect of the disease.
EXPLORER represents a major achievement toward a precision-medicine
approach in cardiomyopathies and should provide great hope to a community
painfully aware of the lack of disease-specific treatment options.”
The 30-week treatment with mavacamten resulted in a highly
statistically significant outcome relative to placebo (p=0.0005) for the
primary endpoint in the EXPLORER-HCM trial, a composite functional analysis
designed to capture the treatment effect of mavacamten relative to placebo on
both symptoms and cardiac function.
All secondary endpoints also demonstrated statistically
significant and clinically meaningful improvements for mavacamten as compared
to placebo. Secondary endpoints in the EXPLORER-HCM trial evaluated
improvements in post-exercise left ventricular outflow tract (LVOT) peak
gradient (p<0.0001), New York
Heart Association (NYHA) functional classification (p<0.0001),
peak VO2 (p=0.0006),
the Kansas City Cardiomyopathy Clinical Summary Score (KCCQ-CSS) (p<0.0001),
and the HCM Symptom Questionnaire Shortness of Breath Domain Score
(p<0.0001).
Mavacamten was well tolerated and demonstrated safety results
comparable to placebo, with no new findings observed. Ninety-eight percent of
patients enrolled completed the study. Of the two percent who dropped
out, none were due to reduced ejection fraction or symptoms of heart failure.
Overall rates of adverse events, serious adverse events, and cardiac
adverse events, including atrial fibrillation, were comparable for patients
treated with mavacamten and placebo.
“The resoundingly positive data from EXPLORER bring us a
significant step closer to improving the lives of people with serious
cardiovascular conditions, starting with HCM, a debilitating disease estimated
to affect one in every 500 people,” said Tassos
Gianakakos, Chief Executive Officer of MyoKardia. “The activity
and tolerability profile observed for mavacamten in this pivotal study
underscores the profound impact and potential for therapeutics that target the
underlying biology of disease. We look forward to the submission of
MyoKardia’s first New Drug Application and, importantly, to serving the many
patients that stand to benefit from mavacamten.”
The EXPLORER-HCM clinical trial is part of MyoKardia’s pivotal program studying mavacamten as a treatment for symptomatic, obstructive hypertrophic cardiomyopathy. MyoKardia plans to submit a New Drug Application to the U.S. Food and Drug Administration (FDA) in the first quarter of 2021. Results from the Phase 3 EXPLORER-HCM clinical trial will be submitted to a future professional meeting in 2020.