← #nieuws

SelectMDx-studie toont kosteneffectiviteit aan in Amerikaanse Gezondheidszorg

MDxHealth meldt de publicatie in The Journal of Urology van een studie die de kosteneffectiviteit van SelectMDx® voor prostaatkanker valideert. SelectMDx® is een niet-invasieve ‘vloeibare biopsie’ test die helpt bij het identificeren van patiënten met een verhoogd risico op agressieve prostaatkanker.

 

De studie toonde aan dat het opnemen van SelectMDx in het Amerikaanse gezondheidszorgsysteem kan resulteren in een gemiddelde volledige gezondheidswinst van 0,045 levensjaren, met een kostenbesparing van $1,694 per patiënt. Extrapolatie van deze gegevens naar een voorzichtige schatting van 311.879 patiënten per jaar die een biopsie ondergingen, resulteerde in 14.035 levensjaren in volledige gezondheidswinst met een jaarlijkse kostenbesparing van meer dan $500 miljoen voor ieder cohort.

 

De studie, ontworpen voor het verkrijgen van inzicht in de kosteneffectiviteit van SelectMDx in een populatie van Amerikaanse mannen met een verhoogd PSA-gehalte, laat een model zien met de impact van het gebruik van de niet-invasieve diagnostische test om mogelijk onnodige prostaatbiopsie uit te sparen. Het primaire doel was om veranderingen in gezondheidsresultaten in kaart te brengen, gemeten in voor kwaliteit gecorrigeerde levensjaren. De secundaire doelstelling was het evalueren van de zorgkosten vanuit het perspectief van de Medicare verzekeraar.

 

Dr. Matthew Resnick van de afdeling urologische chirurgie en gezondheidsbeleid van het Vanderbilt University Medical Center, Nashville, TN zegt: “Deze studie toont aan dat routinematig gebruik van SelectMDx als leidraad voor biopsiebeslissingen de gezondheidsresultaten verbetert en de kosten voor gezondheidszorg in verband met prostaatkanker-risicobeoordeling in een populatie van Amerikaanse mannen verlaagt. Wij zijn van mening dat deze strategie de waarde van conventionele risicobeoordelingsstrategieën vergroot door verbeteringen in zowel kwaliteit als kosten, de teller en noemer van de gezondheidswaarde-vergelijking.”

 

“Deze overtuigende studieresultaten zijn een verdere validatie van de positieve impact van SelectMDx voor het verlagen van de kosten voor de gezondheidszorg door ondersteuning in de begeleiding van behandelbeslissingen voorafgaand aan prostaatbiopsie,” zegt Dr. Jan Groen, Chief Executive Officer van MDxHealth. “Deze gezondheids- en economische waarde van vroegtijdige toepassing van SelectMDx tijdens het behandelingsproces, zullen onze onderhandelingen met Amerikaanse ziektekostenverzekeraars belangrijk ondersteunen.”

 

Dr. Groen vervolgt: “Dit is een belangrijke mijlpaal voor onze SelectMDx-test en versterkt onze strategie om verder voet aan de grond te krijgen in de markt voor eerstelijnszorg. Wij zijn van mening dat met deze uitbreiding de markt voor SelectMDx in de VS en Europa kan verviervoudigen tot meer dan twee miljoen patiënten per jaar”.

 

 

← #nieuws

Innovative digital pathology network will transform microscopy

,

Philips and Oxford University Hospitals NHS Foundation Trust (OUH), plan to create a digital pathology network to help drive faster and more efficient diagnoses for patients. OUH will deploy the Philips IntelliSite Pathology Solution at the John Radcliffe Hospital in Oxford, which will serve as a central laboratory service for partner sites at Milton Keynes University Hospital NHS Foundation Trust and Great Western Hospital NHS Foundation Trust in Swindon.

Pathologists play a critical role in disease detection, particularly with cancer diagnosis. Traditionally, pathologists analyze tissue samples on glass slides under a microscope. When seeking a consultation with a sub-specialist, these glass slides must be transported to the second site, which can result in lost or damaged slides or delays in diagnosis. By creating a digital network, OUH will leverage the Philips IntelliSite Pathology Solution, including its Ultra Fast Scanner and Image Management System, to allow clinicians across the three regions and within the Thames Valley Cancer Network to collaborate remotely on patient cases. This enhanced collaboration will help reduce delays in slide transport times, encourage more efficient workflows across the sites, and enable quicker access to specialist pathology opinions. Digital pathology will also support NHS Improvement’s proposed Pathology networks, bringing together clinical expertise with the goal of enhancing patient outcomes.

Digital pathology will transform microscopy

“As an NHS Global Digital Exemplar committed to improving patient care by embracing the latest digital technologies and cross-site collaborations, this partnership aims to modernize patient care and offer innovative world-leading services,” said Professor Clare Verrill, Honorary Consultant in Cellular Pathology, Oxford University Hospitals. “Initially starting with some specialist areas, we hope to soon make our pathology services fully digital, meaning our laboratory teams can maximize efficiency and  focus on analysing samples rather than spending time manually transporting slides between hospitals.”

“With 70% of NHS diagnoses requiring a pathology sample, and with sample analysis becoming increasingly sophisticated while demand increases, Philips is committed to collaborating to modernize UK pathology services,” said Marlon Thompson, General Manager of Philips Digital Pathology Solutions. “Philips wants to help providers meet growing demand by moving pathology from the era of microscopes and fragile stacks of sample slides to one of clinical efficacy with sample images uploaded quickly and analysed robustly within the Philips IntelliSite Pathology Solution. I believe this agreement creates the first digital pathology network with scanning capabilities at multiple sites within the NHS in England, which is a great achievement and a very positive prospect for patients and clinicians alike.”

Philips IntelliSite Pathology Solution is an automated digital pathology image creation, viewing and management system comprised of an Ultra Fast Scanner and Image Management System. This solution contains advanced software tools to manage the scanning, storage, presentation, reviewing, and sharing of images. Philips IntelliSite Pathology Solution is available in various countries globally for primary diagnostic use. In addition to its CE-IVD clearance in Europe, it was the first – and currently only – digital pathology solution allowed to be marketed for primary diagnostic use in the U.S. since April 2017 and in Japan since December 2017.

Clinical cases will commence once network installation has completed, expected in Q3 2018.

Source: Phillips

← #nieuws

Biogen en overheid zijn het eens: SMA patiënten krijgen Spinraza

,

Biogen en de Nederlandse overheid hebben een overeenkomst bereikt over de vergoeding van Spinraza. HollandBIO is daar heel blij mee. Spinraza is het eerste en enige goedgekeurde medicijn voor de behandeling van spinale musculaire atrofie (SMA), een zeer ernstige, progressieve spierziekte. HollandBIO is er trots op een sector te mogen vertegenwoordigen die deze innovaties voortbrengt.

Door de overeenkomst komt het middel Spinraza voor circa 80 jonge kinderen met SMA vanaf 1 augustus in het basispakket. Dit is geweldig nieuws voor patiënten en hun ouders. Dorota Mazurkiewicz, Managing Director van Biogen Nederland licht de overeenkomst toe:

“Allereerst is dit prachtig nieuws voor de betreffende SMA-patiënten en hun families. SMA is een verschrikkelijke ziekte waar geen medicijn voor bestond, maar nu kunnen we patiënten nieuwe hoop bieden. We gaan nu met betrokken partijen in gesprek om de mogelijkheden voor voorwaardelijke vergoeding te verkennen. We hopen daarmee brede toegang voor alle SMA-patiënten te kunnen realiseren, ongeacht leeftijd of ziektetype, zoals ook het geval is in veel andere landen.”

We zijn er dus nog niet. Het middel betekent een kentering in het leven van patiënten. HollandBIO hoopt daarom dat alle patiënten met SMA zo snel mogelijk toegang krijgen tot Spinraza.

← #nieuws

Galapagos announces design for PINTA Phase 2 trial with GLPG1205 in IPF

Galapagos announces the PINTA Phase 2 trial design with its GPR84 inhibitor GLPG1205 in patients with idiopathic pulmonary fibrosis (IPF). PINTA is a randomized, double-blind, placebo-controlled trial investigating a 100 mg once-daily oral dose of GLPG1205. The drug candidate or placebo will be administered for 26 weeks in up to 60 IPF patients. Patients may remain on their local standard of care as background therapy. Primary objective of the trial is to assess the change from baseline in Forced Vital Capacity (FVC in mL) over 26 weeks compared to placebo. Secondary measures include safety, tolerability, pharmacokinetics and pharmacodynamics, time to major events, changes in functional exercise capacity, and quality of life. IPF diagnosis will be confirmed by central reading. Recruitment for PINTA is planned in 10 countries in Europe, North Africa, and the Middle East. First dosing of an IPF patient is expected in the second half of 2018.

GLPG1205 is a GPR84 inhibitor discovered by Galapagos and fully proprietary to Galapagos. GLPG1205 showed a reduction in signs and symptoms in IPF animal models and has shown favorable tolerability in healthy volunteers and ulcerative colitis patients in previous trials. Galapagos currently has three drug candidates with distinct mechanisms of action in its fully proprietary portfolio aimed at building an IPF franchise: GLPG1690 in the ISABELA Phase 3 program, GLPG1205 in PINTA Phase 2, and GLPG3499, currently in pre-clinical development.

“GLPG1205 has shown signs of good activity in relevant animal models, and GPR84 has already been validated as a mechanism in combination with nintedanib[1] in IPF,” added Dr. Piet Wigerinck, Chief Scientific Officer of Galapagos. “We have a well-designed trial with PINTA for ‘1205 that we anticipate will give us new insights into the potential value of GPR84 inhibition as a mechanism to treat this highly fatal disease.”

 

Source: Galapagos

← #nieuws

First cohort enrolled in trial with innovative gene therapy for arthritis

Arthrogen, a clinical stage gene therapy company together with the Centre for Human Drug Research (CHDR), announced that the first three arthritis patients have been enrolled in a phase Ib gene therapy trial for treatment of arthritis with ART-I02.

ART-I02 is an adeno-associated virus (AAV5) vector encoding the human IFN-β gene under control of an inflammation-responsive promoter. ART-I02 is designed to produce the anti-inflammatory protein IFNβ in the synovial cells in the joint. The aim is to achieve a sustained clinical remission with a single treatment. This current clinical phase Ib trial, evaluates the safety, tolerability, pharmacokinetics, immunogenicity and anti-inflammatory activity of ART-I02 treatment in patients with rheumatoid arthritis (RA) or osteoarthritis (OA). In total 12 patients with RA or OA in their hand joints will be included. Parallel to this study, a similar phase Ib trial has started in Canada treating 15 patients with RA in their wrist.

“Patients with rheumatoid arthritis and osteoarthritis face chronic pain and immobility, even despite the availability of new, mostly systemic treatments.”, explains principal investigator Prof. dr. Koos Burggraaf of CHDR and he continuous: “In this trial we evaluate the safety of ART-I02 and we assess effect measures, hoping that this first step may prove to be an important milestone marking a new era in the treatment of arthritis, a chronic debilitating disease.”.

Robert Jan Lamers, Chief Executive Officer of Arthrogen comments ‘We would like to thank our investors, collaborators and participating patients in helping us achieve this pivotal milestone. Based on anticipated enrollment and follow-up timelines, the first set of data is expected to be available in the fourth quarter of 2018’.

Source: Arthrogen

← #nieuws

Eisai and Biogen announce positive topline results

Eisai and Biogen announced positive topline results from the Phase II study with BAN2401, an anti-amyloid beta protofibril antibody, in 856 patients with early Alzheimer’s disease. The study achieved statistical significance on key predefined endpoints evaluating efficacy at 18 months on slowing progression in Alzheimer’s Disease Composite Score (ADCOMS) and on reduction of amyloid accumulated in the brain as measured using amyloid-PET (positron emission tomography).

Study 201 (ClinicalTrials.gov identifier NCT01767311) is a placebo-controlled, double-blind, parallel-group, randomized study in 856 patients with mild cognitive impairment (MCI) due to Alzheimer’s disease (AD) or mild Alzheimer’s dementia (collectively known as early Alzheimer’s disease) with confirmed amyloid pathology in the brain. Efficacy was evaluated at 18 months by predefined conventional statistics on ADCOMS, which combines items from the Alzheimer’s Disease Assessment Scale-cognitive subscale (ADAS-Cog), the Clinical Dementia Rating Sum of Boxes (CDR-SB) scale and the Mini-Mental State Examination (MMSE) to enable sensitive detection of changes in early AD symptoms. Patients were randomized to five dose regimens, 2.5 mg/kg biweekly, 5 mg/kg monthly, 5 mg/kg biweekly, 10 mg/kg monthly and 10 mg/kg biweekly, or placebo.

Topline results of the final analysis of the study demonstrated a statistically significant slowing of disease progression on the key clinical endpoint (ADCOMS) after 18 months of treatment in patients receiving the highest treatment dose (10 mg/kg biweekly) as compared to placebo. Results of amyloid PET analyses at 18 months, including reduction in amyloid PET standardized uptake value ratio (SUVR) and amyloid PET image visual read of subjects converting from positive to negative for amyloid in the brain, were also statistically significant at this dose. Dose-dependent changes from baseline were observed across the PET results and the clinical endpoints. Further, the highest treatment dose of BAN2401 began to show statistically significant clinical benefit as measured by ADCOMS as early as 6 months including at 12 months.

BAN2401 demonstrated an acceptable tolerability profile through 18 months of study drug administration. The most common treatment emergent adverse events were infusion-related reactions and Amyloid Related Imaging Abnormalities (ARIA). Infusion related reactions were mostly mild to moderate in severity. Incidence of ARIA-E (edema) was not more than 10% in any of the treatment arms, and less than 15% in patients with APOE4 at the highest dose per the study protocol safety and reporting procedures.

Detailed results of the study will be presented at future academic conferences.

“The 18-month results of the BAN2401 trial are impressive and provide important support for the amyloid hypothesis,” said Jeff Cummings, M.D., founding director, Cleveland Clinic Lou Ruvo Center for Brain Health. “I look forward to seeing the full data set shared with the broader Alzheimer’s community as we advance against this devastating disease.”

“This is the first late-stage anti-amyloid antibody study to successfully achieve statistically significant results at 18 months, further validating the amyloid hypothesis,” said Lynn Kramer, M.D., Chief Clinical Officer and Chief Medical Officer, Neurology Business Group, Eisai. “We will discuss these very encouraging results with regulatory authorities to determine the best path forward. We continue to work towards the goal of delivering BAN2401 to patients and healthcare professionals as early as possible.”

“The prospect of being able to offer meaningful disease-modifying therapies to individuals suffering from this terrible disease is both exciting and humbling,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “These BAN2401 18-month data offer important insights in the investigation of potential treatment options for patients with Alzheimer’s disease and underscores that neurodegenerative diseases may not be as intractable as they once seemed.”

As reported in December 2017, the study did not achieve its primary outcome measure which was designed to enable a potentially more rapid entry into Phase III development based on Bayesian analysis at 12 months of treatment. Upon the final analysis at 18 months using predefined conventional statistical method, the study did demonstrate a statistically significant slowing of disease progression on the key clinical endpoint (ADCOMS) after 12 months of treatment in patients receiving the highest treatment dose (10 mg/kg biweekly) as compared to placebo.

This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that any investigational uses of such product will successfully complete clinical development or gain health authority approval.

 

Source: Biogen

← #nieuws

Data show Imnovid-based triple therapy PFS benefit in blood cancer

Celgene has presented Phase III data showing a significant progression-free survival (PFS) benefit in patients with multiple myeloma taking a triple combination therapy of Imnovid, Velcade and low-dose dexamethasone.

According to results of the Phase III OPTIMISMM trial, adding Imnovid (pomalidomide) to Velcade (bortezomib) and low-dose dexamethasone significantly extended PFS to 11.2 months compared to 7.10 months for those taking the latter two alone, equating to a reduction in the risk of disease progression or death by 39 percent.

Overall response rate, one of the study’s secondary endpoints, was also significantly higher in the triple therapy arm at 82.2 percent versus 50.0 percent, while time to treatment response was shorter (0.9 months versus 1.4 months), complete response was higher (15.7 percent versus 4.0 percent), and duration of response was longer (13.7 months versus 10.9 months).

OPTIMISMM is the only Phase III trial to report data with a triplet combination in patients who have all received prior Revlimid (lenalidomide) therapy. In the UK, over 16,000 patients have been treated with lenalidomide since 2009, and this represents a patient population for which there is a growing unmet medical need, Celgene said.

“Results from OPTIMISMM are promising and show the potential of pomalidomide in combination with other therapies, earlier in the myeloma treatment pathway,” noted Dr Neil Rabin, consultant haematologist at University College London Hospitals.

“There is a growing unmet need for those patients who have been treated previously with lenalidomide, and combination therapies are key to meet that need. These data are therefore very encouraging.”

 

Source: PharmaTimes

← #nieuws

Aon Life Sciences Clinical Trials Risk Map

The globalization of clinical trials continues to be on the rise, leading to a plethora of regulatory and operational challenges for the Life Sciences industry. Aon’s 6th edition Clinical Trials Risk Map highlights clinical trials insurance regulations and complexities for over 80 countries throughout the world leveraging Aon’s global network and clinical trials expertise. Aon’s global network is positioned to help life sciences companies efficiently and effectively secure international clinical trials coverage that complies with the regulatory requirements of individual countries, ultimately minimizing the risk of financial volatility.

 

For a free download of the map, click here: http://www.aon.com/getmedia/8752564c-44f6-42af-8976-685baec822b2/aon-life-sciences-clinical-trials-risk-map.aspx

← #nieuws

GROZ: Handen ineen voor een vitaal functionerende samenleving

Morgen start de Topsector Life Sciences & Health (Health~Holland) het nieuwe initiatief GROZ. GROZ staat voor de kanteling van onze gezondheidszorg en is een nationale samenwerking op het gebied van de maatschappelijke uitdaging Gezondheid en Zorg. Binnen GROZ slaan burgers, zorgprofessionals, ondernemers, wetenschappers, financiers en overheid de handen ineen, zowel op lokaal als nationaal niveau. Het blijft niet uitsluitend bij praten. Alle partijen werken samen aan concrete zorgvernieuwingen met als doel een vitaal functionerende samenleving met een gezonde economie.

 

De kracht van burgerinitiatieven

Regionale initiatieven door én voor burgers leiden tot innovatieve, lokaal gedragen oplossingen voor uitdagingen in de gezondheidszorg. Burgers weten zich daarbij steeds meer gesteund door hun gemeente, zorgaanbieders, lokale GGD en ondernemers. Krachtige voorbeelden zijn Vitaal Vechtdal, Austerlitz Zorgt en Stichting Gezondheidscentra Nijkerk. Hoe kunnen dit soort initiatieven ook elders in Nederland de gezondheidszorg kantelen?

 

Startsein voor GROZ

Meer en meer zal de behoefte van burgers centraal staan in het gezondheidszorgsysteem. Daarom start de eerste GROZ bijeenkomst op 4 juli met burgerinitiatieven. Zij komen bijeen om elkaar aan de hand van tien concrete succesvoorwaarden te informeren en inspireren. Vervolgens bepalen de partijen gezamenlijk wat er vanuit andere stakeholders nodig is om deze burgerinitiatieven succesvol te laten groeien.

 

Werken aan de maatschappelijke uitdaging Gezondheid en Zorg Vanuit de behoeften die de burgerinitiatieven op 4 juli ophalen, organiseert de Topsector LSH in de tweede helft van 2018 nog een drietal bijeenkomsten met gezondheidszorgprofessionals, ondernemers en investeerders en financiers. Hier volgen de thema´s mankracht, het veranderde aanbod binnen bedrijven en financieringsstructuren elkaar op. GROZ wil hiermee een domino-effect creëren waardoor partijen gezamenlijk in beweging komen voor de maatschappelijke uitdaging Gezondheid en Zorg.

 

De Topsector LSH en haar coalitiegenoten geven met GROZ het startsein voor een nationale transformatie van de gezondheidszorg, waar burgers, kwaliteit, toegankelijkheid en betaalbaarheid voorop staan.

 

← #nieuws

First two CAR-T cell medicines recommended for approval in the EU

The European Medicines Agency (EMA) has recommended the first two marketing authorisations for chimeric antigen receptors (CAR) T-cells medicines in the European Union (EU). Kymriah and Yescarta are advanced therapies for blood cancer. They belong to a new generation of personalised cancer immunotherapies that are based on collecting and modifying patients’ own immune cells to treat their cancer.

 

Kymriah and Yescarta are also the first medicines supported through EMA’s PRIority MEdicines (PRIME) scheme to receive positive opinions from the Committee for Medicinal Products for Human Use (CHMP). The voluntary scheme provides early and enhanced scientific and regulatory support to medicines that have the potential to address, to a significant extent, patients’ unmet medical needs. Kymriah was granted eligibility to PRIME on 23 June 2016, for the treatment of acute lymphoblastic leukaemia (ALL). Yescarta was granted eligibility to PRIME on 26 May 2016 for the treatment of diffuse large B-cell lymphoma (DLBCL).

 

“CAR-T cells transform the fight against serious and often fatal diseases in the EU,” said Dr Martina Schüssler-Lenz, chair of the Committee for Advanced Therapies (CAT). “Kymriah and Yescarta offer an innovative approach where patients’ cells are reprogrammed and reinjected to attack the cancer.”

 

Because Kymriah and Yescarta are advanced-therapy medicinal products (ATMPs), they were assessed by the CHMP and the CAT, the Agency’s expert committee for cell-, gene- or tissue-based medicines which is responsible for the evaluation of these products. The CAT adopts an advisory opinion, which is taken into account by the CHMP when giving its recommendation regarding the authorisation of the medicine concerned.

 

“Innovative treatments such as CAR-T cells have potential to change the outlook for patients with cancer, but they also come with new scientific and regulatory challenges,” commented Dr Tomas Salmonson, Chair of the CHMP. “From the beginning, we have worked to establish a robust system of data collection for the post-authorisation phase that would suit the specificities of these two medicines. We have used a wide range of tools – scientific advice, a specific workshop on patient registries for CAR-T cells, PRIME, to name just a few, to enable us to define the methods to tightly monitor the benefit-risk profile of these medicines and manage their risks once they are on the market, so that patients can benefit from these innovative treatments.”

 

The main safety concerns related to the administration of CAR-T cells are cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and neurologic toxicities. Both can be life-threatening and in some cases even fatal. Monitoring and mitigation strategies for these side effects are described in the product information and in the risk management plan that is an integral part of the authorisation. CRS can be treated successfully with RoActemra (tocilizumab). The CHMP also recommended adding the treatment of CAR-T cell induced CRS as an indication for this medicine.

 

Another important risk management measure for Kymriah and Yescarta is the utilisation of a patient registry to monitor the long-term safety and efficacy of these therapies, as a condition for the marketing authorisation. The application of registries to support the benefit-risk evaluation of CAR-T cell products and their post-authorisation follow-up was discussed in a specific workshop in early 2018, as part of EMA’s initiative on patient registries. Furthermore, EMA has qualified a registry for collection of post-authorisation safety and efficacy data; this qualification opinion is now open for public consultation.

 

Kymriah is indicated for the treatment of paediatric and young adult patients (up to 25 years of age) with B-cell ALL that is refractory or in second or later relapse, and in adult patients with relapsed or refractory DLBCL after two or more lines of systemic therapy.

 

Yescarta is indicated for the treatment of adult patients with relapsed or refractory DLBCL and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy.

 

The opinions adopted by the CHMP are an intermediary step on Kymriah’s and Yescarta path to patient access. The CHMP opinions will now be sent to the European Commission for the adoption of decisions on EU-wide marketing authorisations. Once the marketing authorisations have been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of these medicines in the context of the national health system of that country.

 

Detailed information about the support offered by the Agency during the development, including information on the topics for which scientific advice was obtained, and the assessments of these two medicines will be made public in their respective European Public Assessment Reports (EPARs).

Source: EMA