Understanding the impact of rare diseases on the quality of life of patients and their relatives

The North American Neuro-Ophthalmology Society’s (NANOS) 48th Annual Meeting took place on February 12-17, 2022 in Austin, Texas

In collaboration with Cambridge University and Gensight Biologics, groupH published a poster at the conference based on the findings of a qualitative study to explore the impact of Leber Hereditary Optic Neuropathy (LHON) on patients’ and their relatives’ lives. LHON is a rare condition which leads to blindness and disability in teens and young adults.

Please click on the image to view the poster


Understand US Payer contracting trends and use our Gross-to-Net Easy Tool to calculate future US net prices

groupH has developed a simple US gross-to-net discount model including a proof-of-concept validation. This simple tool estimates applicable gross-to-net contracting discounts in the context of early-stage commercial assessments in the US market. The model has been developed based on in-depth primary market research with senior payers and secondary analysis. This paper was first presented during the EphMRA annual conference in June 2021.

Please click on the image below to access the paper




Estimating Global Patient Needs and Market Potential for Priority Health Technologies Addressing Antimicrobial Resistance

A groupH team has worked with the AMR Global R&D Hub over the last 12 months to produce data and analysis for a report on epidemiology and revenue projections for two therapeutics and two diagnostics in the Gram-negative space.

Click on the image below to read the full report, delivering commercial insights into novel treatments and diagnostics to combat antimicrobial resistance


UPDATE: our take on CMS’s latest memo on CAR-T reimbursement

Earlier in the year, we reviewed the reimbursement challenges that CAR-T therapies are experiencing in the US, assessed some of the proposed solutions, and outlined Payers’ expectations from next-generation CAR-T therapies. Click here to see the original analysis.

CMS published a memo providing an update to CAR-T reimbursement in early August that included a number of changes: (1) increased NTAP payments, (2) loosened coverage by dropping the coverage with evidence development (CED) requirement, and (3) reimbursement of off-label use.

While news headlines generally implied that CAR-T therapies will be fully covered and reimbursed, groupH analysis shows that this is far from the case.  We found that the increased NTAP payment is still inadequate, and furthermore, is offset by a reduced outlier payment.

Please click here to download a few slides outlining the key points from the CMS memo, and our take on how this impacts the reimbursement of CAR-T therapies.

Email morris.paterson@groupH.com to arrange an in-person presentation with your team.


CAR-T Reimbursement in the US – Challenges and an Assessment of Potential Solutions

CAR-T cell therapy has recently arisen as an exciting and innovative therapeutic modality. A wave of new biotechs have been established, several hundreds of clinical trials are underway, and multi-billion-dollar acquisitions have occurred. Despite rapid scientific and clinical advancements, Medicare reimbursement during these initial launch years has been underwhelming, particularly in the in-patient setting where CAR-T therapies are most frequently used.

To characterize the reimbursement challenges to CAR-T therapy today, we spoke with Payers who are covering these therapies, and Pharmacists and Finance VPs at medical institutes providing CAR-T therapies.

We found that inadequate reimbursement coverage for Medicare beneficiaries has led to hospitals with significant financial resources needing to subsidize treatments, while other institutions are not able to provide treatment. We believe that the recent CMS announcement (Feb 15th, 2019) that ties coverage to evidence development may prove to be too burdensome for some providers and could provide a legal route for providers to opt-out of providing CAR-Ts to Medicare patients.

In our white paper presentation, we explore some of the reimbursement solutions that have been proposed by the American Society of Bone Marrow & Transplantation (ASBMT) and the American Society of Hematology (ASH) and assess how these proposals meet the needs of Patients, Providers, Payers, and Manufacturers and their ease of implementation. We also outline a ‘Drop-Ship’ model that could eliminate a provider’s financial burden, and draws parallels to the Spark Therapeutics reimbursement model that has been implemented for the gene therapy Luxturna. We also propose a method to incorporate a 1-year outcomes assessment to address the needs of Payers.

We believe that these reimbursement issues are relevant to any company with a cellular or personalized therapy in development.

A short infographic summary of the paper can be found below. To download a copy of the entire presentation please click on the icon below, or email morris.paterson@groupH.com to arrange an in-person presentation with your team.



Approaches to manage ‘affordability’ of high budget impact medicines in key EU countries

The launch of hepatitis C (HCV) drugs such as sofosbuvir or ledipasvir has fostered the question of affordability of novel high budget impact therapies even in countries with high domestic product. European countries have developed a variety of mechanisms to improve affordability of such therapies, including ‘affordability thresholds’, price volume agreements or caps on individual product sales, and special budgets for innovative drugs.

While some of these mechanisms may help limit budget impact, there is still significant progress to be made in the definition and implementation of approaches to ensure affordability, especially in health systems where the growth potential in drug spending and/or in the patient contribution to health insurance are limited.

In this article, Nicolas Touchot, head of groupH’s Market Access practice, reviews how seven countries in Western Europe are approaching the question of affordability of novel therapies and are developing approaches to continue to reward new sciences while limiting budget impact. He also discusses the question of affordability of cost-effective but hugely expensive therapies and the implications for payers and for the pharmaceutical industry.

Click on the following link to read the full article:

Approaches to manage ‘affordability’ of high budget impact medicines in key EU countries

Will indication value-based pricing become a reality in Europe?

groupH’s Market Access practice, headed by Nicolas Touchot, has recently been part of a collaboration to research the feasibility and attractiveness of indication value-based pricing (IBP) in key EU countries.

Many targeted medicines are developed for different diseases and indications with common underlying biological mechanisms. The product benefit and therefore the ‘value’ is almost certain to vary by indication and by treatment.

At present there is no system in place to allow payers to fully capture this difference in value found across indications. This could lead ultimately to products never being developed in an indication for which they could represent a major therapeutic advance, simply because this would lead to a lower price than that achieved or achievable in other indications.

IBP has been proposed in the United States as a tool to address this issue, and is in the early phases of implementation there. However, in Europe, the picture is more patchy and implementation on a broader basis appears to be lagging behind the US.

Our latest research assessed how the reimbursement and pricing environment could allow for IBP in seven European countries, evaluating both incentives and hurdles.

Our findings suggest that broad implementation – including community-based practices – will be difficult in most countries covered, because of legal, data collection, and billing hurdles.

However, implementation at a smaller scale and scope e.g. for specialty products in hospitals could be feasible because of much lower hurdles.

Other mechanisms, such as use of managed entry agreements and volume-weighted average pricing defined through HTA outcome across indications, are likely to continue to be used to account for the difference in value across indications for most broadly prescribed treatments.

We believe, however, that indication based pricing is here to come because it is attractive in principle to all stakeholders. For community-prescribed pharmaceutical products it currently still encounters logistical and IT challenges, but for hospital-based specialty products there is a good chance. This will be the area where we see the first pilots.

Please click here to view the article, published this month in the Journal of Market Access & Health Policy.

For more information, please contact Nicolas Touchot.