Top 10 biotech bay companies by revenue
Biosimilars make progress in the Canadian market, but stakeholders see more work ahead
How development of gene therapies impact decisions on their place in therapy
FDA approves first therapy to treat patients with rare blood disorder
2019's fiercest women in life sciences
Emerging markets are ‘blue oceans’ for biosimilars
A new targeted gene therapy shows the promise and pitfalls of medical advances
4 ways to adapt to new oncology value-based models
Drug prices: shortages remain problem
As price pressure mounts, pharma companies tap analytics to cut costs
Non-binding guidance: real-world evidence in drug development and FDA submissions
Biosimilar drug market emerging trends, size, share and growth analysis by 2025
Here come the oncology biosimilars in the US: how low will prices go?
Will long-term post-marketing studies become the norm for gene therapy companies?
Why Japan’s new cost-effectiveness assessment scheme is “only a starting point”
Disinvestment activities and candidates in the Health Technology Assessment community: an online survey
Expect to see more conversations about the need for HTA-based investment decision-making.
Dual CAR-T cell therapy shows potential for treating multiple myeloma
Will the uptake of CAR-T therapies be constrained by clinical trials and patient preferences for waiting for next-generation CAR-T therapy?
The US Military Health System: promoting readiness and providing health care
Insight into the challenges in single-payer systems. People often forget that the US has a large, single-payer, patient-centered system comparable to the UK NHS.
Cystic fibrosis drugs rejected for use by NHS in Scotland
Patient groups renew the call for urgent access.
A comparison of EMA and FDA decisions for new drug marketing applications 2014–2016: concordance, discordance, and why
Differences in clinical data, and different conclusions even from the same data, drive FDA and EMA discordance in approvals.
A framework for aiding the translation of scientific evidence into policy: the experience of a hospital-based technology assessment unit
Post-approval market access insight: examining the local level decision-making that affects uptake and access of new therapies even after national-level coverage and reimbursement is approved.
Validation of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire core 30 summary score in patients with hematologic malignancies
Good to see the validity of the QLQ-C30 questionnaire expanded.
The fragility of phase 3 trials supporting FDA-approved anticancer medicines: a retrospective analysis
Great starting point for anyone working on drugs with a plateau effect in survival, and who is new to the fragility index but affected by questions of statistical power and the tail of the Kaplan-Meier curve.
Use of real-world data sources for Canadian drug pricing and reimbursement decisions: stakeholder views and lessons for other countries
The challenges with expanding the use of real-world evidence in pricing and reimbursement assessments: excellent outline from Don Husereau and colleagues.
Price increases of protected-class drugs in Medicare part D, relative to inflation, 2012-2017
The ongoing cost-pressure challenges facing Medicare Part D in the US: An interesting but short survey.
Overcoming current barriers to the use of CAR T-Cell therapy in the community setting
ATMPs present new levels of access and funding challenges for solid tumor CAR-T and TCR therapies, and for treatment in community hospitals.
Pharma & patient organisation collaborations could improve patient care
Price transparency rule could pose challenges
In the US, publishing prices isn’t necessarily in the best interest of the provider or the manufacturer. How many EU payers would reject total transparency if they thought they routinely got the best deal?
Pursuit of tumor-infiltrating lymphocyte immunotherapy speeds up
Encouraging to see new modalities, but can manufacturers bring the TIL vein-to-vein time closer to that of CAR-Ts and TCRs?
CMS finalizes long-sought rules for Medicare CAR-T coverage
Positive response from providers also, but still a long way to go on covering total costs.
Do no financial harm: how to improve cost transparency in cancer care
Financial toxicity is a poorly understood, vital element of the carer and patient experience. Dedicated research is needed into the diversity in costs and who bears them.
State-of-the-art for CAR T-cell therapy for chronic lymphocytic leukemia in 2019
Like solid tumors, cell and gene therapies for chronic, rather than acute, hematological conditions will present a significant jump in eligible patient population numbers and total costs to healthcare systems.
Artificial intelligence could globally revolutionize health care — unless it destroys it
A well-balanced cautionary article.
Paper proposes methodology to "Protect Health Systems" that use biosimilars
Interesting to see explorations of how the data underlying biosimilars vary.
A watchdog group sets aside emotions to assess drugs’ value. Patients say their lives are more than a number
Some interesting perspectives on a health outcomes version of thinking about growth versus attainment.
ARM Q2 2019 Regenerative Medicine Sector Report
Important reading for anyone working on Advanced Therapy Medicinal Products (ATMPs).
Willingness to pay for quality-adjusted life years (QALY): perspectives and contexts in Japan
Car-T: the first agreement for new therapies, based on actual results, is ready. ‘A new negotiation model that puts Italy at the forefront’
Notable that, with payment “for the first bag”, this deal rewards the manufacturer upfront; payment “for the first bag”, however, makes Novartis responsible for manufacturing failures but leaves uncertainty as to who is at risk if manufacturing delays mean that the patient cannot be reinfused.
Travis Broome discusses participating in multiple payment reform models
Good interview with Travis Broome. When it comes to alternative payment models and milestone-based payment models, far less has been explored about providers’ needs and heterogeneity relative to payers. The disruption of threatening buy-and-bill paradigms in the US should not be underestimated.
Lani Alison on how innovation is being used to improve quality metrics in oncology
Is time running out for value frameworks?
Could value frameworks be overtaken by formal HTA or HTA-like processes and value-based contracting? This #ValueInHealth paper makes a good case for caution.
CAR T-Cell therapy: a microcosm for the challenges ahead in Medicare
A US perspective on the affordability (or inaffordabilty) of CAR-T treatment.
CMS proposed rule requires hospitals to make public their payer-specific negotiated charges
One to watch, not only because of CMS’ struggles with similar attempts, but because of the US presidential candidates’ policies that may emerge.
The rise of patient engagement
Number of manufacturers and generic drug pricing from 2005 to 2017
An interesting and important policy challenge: like biosimilars, the number of manufactured generics is increasing, but so are drug prices.
NICE reviews number crunching methods as advanced technologies hit market
For anyone working in HTA, the upcoming NICE review of how it develops guidance for drugs is one to watch.
Funding academic innovations in advanced therapies
What potential funders look for in a potential partner.
Implications of “no deal” Brexit for orphan and paediatric medicines
Data collection infrastructure for patient outcomes in the UK – opportunities and challenges for cell and gene therapies launching
Can the existing data collection infrastructures for patient outcomes enable outcomes-based reimbursement in the UK?
Swiss to take on big pharma with cheaper cancer treatment
A policy phenomenon to watch: will the use of homebrews emerge country by country on the basis of thoughtful policy?
CHMP issues positive opinion for 13 medicines
Potential payer perspectives on larotrectinib are identified in this on-demand webinar >> https://www.prmaconsulting.com/webinars/Future-proofing-for-the-2020s1.html
Some cancer patients are choosing experimental treatments rather than approved CAR-T therapies
With ongoing and increasing numbers of clinical trials, can patient/clinician enthusiasm for newer generation ATMPs dampen uptake of existing ones?
Selectively targeting cancerous T-Cells using CAR-T therapy
EUnetHTA: Practical considerations when critically assessing economic evaluations
An opportunity to comment on the EUnetHTA draft methodological guideline for assessing economic evaluations. Deadline is 20 September 2019.
Spanish authorities back plans to promote patent-free medicines
It will be interesting to see how this model might affect disappointment over the true impact of biosimilars on drug prices.
NICE numbers at 20
NICE: where is it doing well, and where it can do better on health technology assessments?
Panel discusses creative financing proposals
The so-called “Netflix model” is one of the developments in the US that payers elsewhere might consider and be able to implement.
Applying Machine Learning to Healthcare Outcomes Research
Helpful introduction to machine learning for #HEOR published in Value in Health.
A New Method to Determine the Optimal Willingness to Pay in Cost-Effectiveness Analysis
Take part in our consultation and let us know how we can best engage with patients and the public
MHRA looking to further patient centricity.
Pharma, late to digital game, rushes to catch up
How modernization is effecting the FDA
In a small study, a cancer vaccine assist beats immunotherapy drugs alone
Keen to stay out front in cell therapy, Gilead's Kite builds its own viral vector manufacturing operations
FDA Unveils Pilot Program to Expand Access to Investigational Oncology Drugs
NICE recommends funding for psoriasis and type 1 diabetes drugs
Cell therapy weekly: Taiwan expands availability of cell therapy
“Cell and gene therapy adoption is limited by the number of centers that have been approved to deliver it.”
A Review of Immune-Mediated Adverse Events in Melanoma
Like with CAR-Ts, manufacturers need to be able to articulate clearly to clinicians and payers how AEs with immunotherapies will be managed. This is a handy reference for thinking about how to approach this.
Improving affordability of new essential cancer medicines
Pairing targeted drugs could overcome breast, lung cancer treatment resistance
“It's been clear for some time that combinations can deliver superior outcomes – we now need innovative access schemes to make them affordable.”
ISPOR launches expanded HEOR Presentations Database
The @ISPORorg Presentations Database should be a go-to resource. Being able to find otherwise non-citable content is a great enhancement. Plenaries, workshops, and issues panels often contain incredibly insightful presentations
New drugs: where did we go wrong and what can we do better?
“Science is advancing, as is the regulatory framework. Phase 3 RCTs will likely always be important, but not always the answer, particularly if they delay access to drugs that offer a meaningful benefit to some patients.”
Personalised reimbursement: a risk-sharing model for biomarker-driven treatment of rare subgroups of cancer patients
“Diseases are becoming more precisely defined; in small populations and rare diseases, RCTs are unlikely to be the most efficient way to get innovative products to patients. @OUPAcademic describes one model being tried in the Netherlands in cancer.”
New Committee Brings Diverse Patient, Family, and Caregiver Voices to CADTH
“Good to see. Greater patient and carer perspectives in decision-making can never be enough.”
Using AI to predict the outcome of clinical trials
“Can AI help reduce the number of clinical trial failures? While promising, clinical trials are the lynchpin for knowing whether we can trust and pay for novel medicines. Without going through robust testing, should AI be entrusted with clinical development decisions?”
Pharma continues to get poor grades when it comes to sharing clinical trial data
“Will “poor grades” alone add “further pressure on the pharmaceutical industry to improve disclosure practices”? Incentives, policing, and regulation, along with making a positive argument that sways manufacturers, are still needed.”
Social Media Offers Unexpected Benefits for Oncologists, Patients
“We have been exploring social media analysis for this reason: such platforms allow for a wide, multi-participant conversation between many different perspectives about some of the most important issues in cancer and cancer treatment today.”
The fragility of phase 3 trials supporting FDA-approved anticancer medicines: a retrospective analysis
Lessons From Mercosur Multi-Country Pricing Negotiations
“An interesting phenomenon; the comparative politics of #HTA and other assessment processes across the EU would make it very challenging for a monopsonistic bloc to form here, although smaller blocs like FINOSE will be paying attention.”