Invokana® (canagliflozin) Significantly Reduced Major Cardiovascular Events and Kidney Failure in Patients with Type 2 Diabetes and Chronic Kidney Disease in New CREDENCE Analysis

CAMBRIDGE, UK: 11.06.19 – Mundipharma welcomes the results of a new subgroup analysis from the landmark Phase III CREDENCE study which shows Invokana® (canagliflozin) significantly reduced the risk of major cardiovascular (CV) events and kidney failure in patients with type 2 diabetes mellitus (T2DM) and chronic kidney disease (CKD) in patients with and without known cardiovascular (CV) disease.1 These results were presented today at the American Diabetes Association’s 79th Scientific Sessions in San Francisco, USA.

“Cardiovascular disease and kidney disease are two serious complications of type 2 diabetes that may shorten life expectancy by several years. This latest analysis of the CREDENCE study demonstrates that for patients with type 2 diabetes and chronic kidney disease, canagliflozin reduced the risk of a cardiovascular event, whether or not patients had already experienced one. Thus, early treatment may help to prevent clinical manifestations of cardiovascular disease – an important message for healthcare professionals managing these patients.” said David Wheeler, Professor of Kidney Medicine at University College London, UK and Honorary Consultant Nephrologist at the Royal Free London NHS Foundation Trust.

The Phase III CREDENCE study evaluated CV and renal outcomes in patients with T2DM and CKD taking either canagliflozin or placebo, in addition to standard of care. The primary results were recently added to the American Diabetes Association’s Standards of Medical Care in Diabetes and published in The New England Journal of Medicine in April this year.

In the new subgroup analysis of the clinical trial results, researchers specifically examined CV and renal outcomes in a primary prevention group, which included participants with CV risk factors but no history of CV disease (n=2,181; 49.6%) and a secondary prevention group, including patients defined as having a history of coronary, cerebrovascular or peripheral vascular disease (n=2,220; 50.4%).1

Building on the initial CREDENCE results presented at the World Congress of Nephrology in Melbourne in April 2019, this subgroup analysis showed that the CV results observed in the overall study population were consistent across the primary and secondary prevention groups, including all clinical subgroups and across groups defined by renal function. For CV death, heart attack and stroke, there was no evidence of heterogeneity between the primary and secondary prevention groups (p=0.25). Specifically, canagliflozin reduced the risk of the composite of CV death, heart attack and stroke by 32% in the primary prevention group (HR: 0.68; 95% CI: 0.49 to 0.94) and 15% in the secondary prevention group (HR: 0.85; 95% CI: 0.69 to 1.06).1

Furthermore, the renal results observed in the overall study population were consistent across the primary and secondary prevention groups. Specifically, canagliflozin reduced the risk of ESKD by 31% (HR: 0.69; 95% CI: 0.51 to 0.95; P-interaction: 0.89) and 33% (HR: 0.67; 95% CI: 0.47 to 0.96; P-interaction: 0.89) in the primary and secondary prevention groups, respectively.1

The full results showed that the CREDENCE study met its primary endpoint by demonstrating that canagliflozin reduced the risk of composite doubling of serum creatinine, end-stage kidney disease (ESKD) and renal or CV death by 30% [HR: 0.70; 95% CI: 0.59 to 0.82; p=0.00001].6 Furthermore, the CV results from CREDENCE found canagliflozin significantly reduced major CV events in the overall study population, including reducing the risk of CV death, heart attack or stroke by 20% (HR: 0.80; 95% CI: 0.67 to 0.95; p=0.01) and risk of CV death or hospitalization for heart failure by 31% (HR: 0.69; 95% CI: 0.57 to 0.83; p<0.001) and hospitalization for heart failure alone by 39% (HR: 0.61; 95% CI: 0.47 to 0.80; p<0.001).6

In addition, CREDENCE found the incidence rates of adverse events and serious adverse events were numerically lower for patients treated with canagliflozin as compared to placebo.6 For the subgroup analysis, safety outcomes were similar in both primary and secondary prevention groups. Of note, there was no difference in fracture risk or incidence of amputations in the primary and secondary prevention groups.1

“We are delighted the results from this subgroup analysis show canagliflozin can offer clinicians and their T2DM patients with chronic kidney disease protection from both CV and kidney disease, which are both high risk for this patient population” said Dr Vinicius Gomes de Lima, European Medical Affairs Lead. “As the first type 2 diabetes medicine to show this benefit to patients with or without known CV disease, there is a potential to positively improve the outcomes for patients living with type 2 diabetes.”

In Europe, canagliflozin is indicated for the treatment of adults with insufficiently controlled T2DM as an adjunct to diet and exercise. The initiation dose is 100mg once daily in adults with an eGFR of ≥ 60 mL/min/1.73 m2 and can be increased to 300mg once daily orally if tighter glycaemic control is needed. Canagliflozin should not be initiated if eGFR is < 60 mL/min/1.73 m2.  In patients tolerating canagliflozin whose eGFR falls persistently below 60 mL/min/1.73 m2 the dose should be adjusted to or maintained at 100mg once daily. Canagliflozin should be stopped if eGFR falls persistently below 45 mL/min/1.73 m27

Currently, 58 million people in Europe currently live with T2DM, which is set to rise to 67 million by 2045.3 Approximately 40% of these patients will develop diabetic kidney disease (DKD),2 which is associated with a high risk of CV disease (heart attack, heart failure and stroke) and also amplifies the risk of other diabetes complications including; a reduced quality of life, infections, fatigue, depression, adverse drug reactions and premature death.4,5

-END-

 Notes to the editors:

About the CREDENCE Clinical Trial6

The CREDENCE (Canagliflozin and Renal Events in Diabetes with Established Nephropathy Clinical Evaluation) study was the first dedicated and full recruited renal outcome trial evaluating renal and cardiovascular outcomes in people with type 2 diabetes mellitus (T2DM) and chronic kidney disease (CKD) with a sodium glucose co-transporter 2 (SGLT2) inhibitor. It was a Phase III randomised, double-blind, event-driven, placebo-controlled, parallel-group, 2 arm multi-centre study of the effects of canagliflozin on renal and cardiovascular outcomes in subjects with T2DM and CKD.  In particular, it compared the efficacy and safety of canagliflozin versus placebo at preventing clinically important kidney and cardiovascular outcomes in patients with T2DM and CKD when used in addition to standard of care, including a maximum tolerated daily dose of an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB).

About Canagliflozin7

Canagliflozin is an oral, once-daily medication which belongs to a class of medications called sodium glucose co-transporter 2 (SGLT2) inhibitors. SGLT2 inhibitors work by inhibiting SGLT2, which promotes the loss of glucose via the urine, lowering blood glucose levels in adults with T2DM. Canagliflozin was approved in the European Union by the European Commission in November 2013. It is indicated for the treatment of adults with insufficiently controlled T2DM as an adjunct to diet and exercise, as monotherapy when metformin is considered inappropriate due to intolerance or contraindications and in addition to other medicinal products for the treatment of diabetes. Approval was based on a comprehensive global Phase III clinical trial programme.

About the Mundipharma network

Mundipharma is a global network of privately-owned independent associated companies whose purpose is to move medicine forward.  With a high performing and learning organization that strives for innovation and commercial excellence through partnerships, we successfully transformed and diversified our European portfolio of medicines to create value for patients, payers and wider healthcare systems across important therapeutic areas such as Diabetes, Respiratory, Oncology, Pain and Biosimilars.

For further information please contact:

 Patrice Grand

European Director of Corporate Communications, Mundipharma Ltd

Email: Patrice.Grand@Mundipharma.com

Abbie Bell

Senior Account Manager, Havas SO

E-mail: Mundipharma@HavasSO.com

Tel: +44 (0) 20 3196 9919

References

1 American Diabetes Association’s 79th Scientific Sessions. Sponsored symposium ‘CREDENCE and CARMELINA—Results from Two Major Clinical Trials in Kidney and Cardiovascular Disease in Diabetes’. Presented at 7:30 a.m. – 9:30 a.m. (PT) on 11th June, 2019

2 Alicic R., et al. Diabetic Kidney Disease: Challenges, Progress, and Possibilities. Clin J Am Soc Nephrol. 2017; 12(12):2032-2045

3 IDF Diabetes Atlas Eighth Edition 2017. Available at: http://diabetesatlas.org/resources/2017-atlas.html. Last accessed June 2019.

4 CDC. National Chronic Kidney Disease Fact Sheet, 2017. Available at: https://www.cdc.gov/kidneydisease/pdf/kidney_factsheet.pdf Last accessed June 2019

5 Thomas M., Cooper M., and Zimmer P. Changing epidemiology of type 2 diabetes mellitus and associated chronic kidney disease. Nature Review Nephrology. 2016; (12): 73-81

6 Perkovic, V. et al. Canagliflozin and Renal Outcomes in Type 2 Diabetes and Nephropathy. The New England Journal of Medicine. 2019; DOI: 10.1056/NEJMoa1811744

7 Canagliflozin SmPC. Available at: https://www.ema.europa.eu/en/documents/product-information/invokana-epar-product-information_en.pdf Last accessed June 2019

Invokana® (canagliflozin) Significantly Reduces the Risk of Renal Failure in Patients with Type 2 Diabetes and Chronic Kidney Disease in the Landmark Phase 3 CREDENCE Study

CAMBRIDGE, UK: 15.04.19 – The Mundipharma network of independent associated companies welcomes the CREDENCE study data which successfully demonstrated that Invokana® (canagliflozin) reduces the risk of renal and cardiovascular (CV) events and has an acceptable safety profile consistent with previous studies when added to standard of care in subjects with type 2 diabetes mellitus (T2DM).1 The study met its primary endpoint showing that canagliflozin reduced the risk of composite doubling of serum creatinine, end-stage kidney disease (ESKD) and renal or CV death by 30% [HR: 0.70; 95% CI: 0.59 to 0.82; p=0.00001].1 These findings were consistent across the individual components of the primary composite endpoint, as well as across all 15 subgroups tested.1

In addition, canagliflozin reduced the risk of the secondary renal endpoint composite of doubling of serum creatinine, ESKD, and renal death by 34% [HR:0.66; 95% CI: 0.53 to 0.81; p<0.001].1
The study also showed that canagliflozin reduced the risk of major adverse cardiac events (MACE) (composite of non-fatal myocardial infarction, non-fatal stroke and CV death) by 20% [HR: 0.80; 95% CI: 0.67 to 0.95; p=0.01], the risk of CV death and hospitalization for heart failure by 31% [HR: 0.69; 95% CI: 0.57 to 0.83; p<0.001], and the risk of hospitalization for heart failure alone by 39% [HR: 0.61; 95% CI:

0.47 to 0.80; p<0.001].1 In regard to safety data, the incidence rates of adverse events and serious adverse events were numerically lower for patients treated with canagliflozin as compared to placebo. There were no observed differences in the incidence of lower limb amputations (HR: 1.11; 95% CI: 0.79 to 1.56) or adjudicated fractures (HR: 0.98; 95% CI: 0.70 to 1.37). 1

As the first dedicated clinical trial to investigate a SGLT2 inhibitor for renal protection in patients with T2DM with CKD, the data from CREDENCE1 provides the first significant update in nearly 20 years regarding slowing the progression of CKD for this group of patients. The trial, which was stopped early in July 2018 due to a signal of overwhelming efficacy in the prevention of the primary endpoint, was conducted in more than 4,400 adults with T2DM at 690 sites in 34 countries across North America, Latin America, Europe, South Africa and Asia Pacific.1

In Europe, canagliflozin is indicated for the treatment of adults with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise. The initiation dose is 100mg once daily in adults with an eGFR of ≥ 60 mL/min/1.73m2 and can be increased to 300mg once daily orally if tighter glycaemic control is needed. Canagliflozin should not be initiated if eGFR is < 60 mL/min/1.73 m2. In patients tolerating canagliflozin whose eGFR falls persistently below 60 mL/min/1.73 m2 the dose should be adjusted to or maintained at 100mg once daily. Canagliflozin should be stopped if eGFR falls persistently below 45 mL/min/1.73 m2.4

“Canagliflozin is the first medical breakthrough in nearly 20 years proven to slow the progression of chronic kidney disease in patients with diabetes at high risk of developing kidney failure” said Professor Vlado Perkovic, Study Author and Executive Director of The George Institute, Australia, Professor of Medicine at UNSW Sydney. “These impressive results from the CREDENCE study have significant clinical implications for preventing kidney failure and improving health for millions of people living with chronic kidney disease and type 2 diabetes.”

Approximately 58 million people in Europe currently live with T2DM, which is set to rise to 67 million by 2045.2 If left untreated, patients are at greater risk of
developing serious complications, such as CV disease and diabetic kidney disease (DKD).3 DKD is the leading cause for progression to ESKD, accounting for 50% of cases in the developed world.5 It is associated with a high risk of CV disease (heart attack, heart failure and stroke) and also amplifies the risk of other diabetes complications including; a reduced quality of life, infections, fatigue, depression, adverse drug reactions and premature death.6,7

“With nearly 24 million type 2 diabetes patients in Europe likely to develop diabetic kidney disease, we are delighted with the results from the CREDENCE study which demonstrated superiority of canagliflozin, when added to the standard of care,” said Dr Vinicius Gomes de Lima, European Medical Affairs Lead. “Type 2 diabetes is a growing epidemic in Europe and effective treatments are needed to help reduce the burden of the disease in patients. In particular, treatments are called for to improve renal outcomes which is of real importance in this disease.”

-END-

Notes to the editors:

About the CREDENCE Clinical Trial1
The CREDENCE (Canagliflozin and Renal Events in Diabetes with Established Nephropathy Clinical Evaluation) study was the first dedicated and full recruited renal outcome trial evaluating renal and cardiovascular outcomes in people with type 2 diabetes mellitus (T2DM) and chronic kidney disease (CKD) with a sodium glucose co-transporter 2 (SGLT2) inhibitor. It was a phase 3 randomised, double-blind, event-driven, placebo-controlled, parallel-group, 2 arm multi-centre study of the effects of canagliflozin on renal and cardiovascular outcomes in subjects with T2DM and CKD. In particular, it compared the efficacy and safety of canagliflozin versus placebo at preventing clinically important kidney and cardiovascular outcomes in patients with T2DM and CKD when used in addition to standard of care, including a maximum tolerated daily dose of an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB).

About Canagliflozin
Canagliflozin is an oral, once-daily medication which belongs to a class of medications called sodium glucose co-transporter 2 (SGLT2) inhibitors. SGLT2 inhibitors work by inhibiting SGLT2, which promotes the loss of glucose via the urine, lowering blood glucose levels in adults with T2DM. Canagliflozin was approved in the European Union by the European Commission in November 2013. It is indicated for the treatment of adults with insufficiently controlled T2DM as an adjunct to diet and exercise, as monotherapy when metformin is considered inappropriate due to intolerance or contraindications and in addition to other medicinal products for the treatment of diabetes. Approval was based on a comprehensive global Phase III clinical trial programme.4

About the Mundipharma network
Mundipharma is a global network of privately-owned independent associated companies whose purpose is to move medicine forward. With a high performing and learning organization that strives for innovation and commercial excellence through partnerships, we successfully transformed and diversified our European portfolio of medicines to create value for patients, payers and wider healthcare systems across important therapeutic

For further information please contact:

Tiffany Fretwell
Communications Lead, Mundipharma International Ltd
Email: Tiffany.Fretwell@Mundipharma.com
Tel: +44 (0) 7773 199 422

Abbie Bell
Senior Account Manager, Havas SO
E-mail: Mundipharma@HavasSO.com
Tel: +44 (0) 737 680 2980

References

1 Perkovic, V. et al. Canagliflozin and Renal Outcomes in Type 2 Diabetes and Nephropathy. N Engl J Med. 2019 DOI: 10.1056/NEJMoa1811744

3 IDF Diabetes Atlas Eighth Edition 2017. Available at: http://diabetesatlas.org/resources/2017-atlas.html. Last accessed March 2019.

3 Update to International Diabetes Federation, 2016, Complications of Diabetes [Online] Available at: https://www.idf.org/aboutdiabetes/what-is-diabetes/complications.html Last accessed March 2019.

4 Canagliflozin SmPC. Available at: https://www.ema.europa.eu/en/documents/product-information/invokana-epar-product-information_en.pdf Last accessed April 2019

5 Tuttle KR., et al. Diabetic kidney disease: a report from an ADA Consensus Conference. Diabetes Care. 2014; 37(10):2864-83.

6 CDC. National Chronic Kidney Disease Fact Sheet, 2017. Available at: https://www.cdc.gov/kidneydisease/pdf/kidney_factsheet.pdf Last accessed July 2018

7 Thomas M., Cooper M., and Zimmer P. Changing epidemiology of type 2 diabetes mellitus and associated chronic kidney disease. Nature Review Nephrology. 2015; (12): 73-81.

European Commission approves Pelmeg▼® (pegfilgrastim) as a biosimilar treatment to reduce the duration of neutropenia and incidence of febrile neutropenia in adults treated with chemotherapy

Mundipharma announces Chief Financial Officer

European Commission expands labelling for INVOKANA® and VOKANAMET® to include positive data on cardiovascular and renal outcomes

Pharmaceutical market access in Europe, by Will Dunlop

The pharmaceutical regulatory process in Europe is not a one size fits all. 

Europe’s payers share a quest for value, but differ in their approaches. Local expertise and partnering is critical.

Gaining market access in pharma across Europe has always presented challenges to pharmaceutical firms. The region has a common regulator, the European Medicines Agency, and most of its citizens receive healthcare from government-funded, often country-wide, health systems. Yet behind this top-level homogeneity lies a series of very different national and regional markets. Each of these has its own set of health policies and practices, its own culture and language, and its own tough pricing and reimbursement hurdles.

Efforts are underway to harmonize European health technology assessment methods and requirements. But they can only go so far (read more on this). Health systems reflect national health policy, clinical practices, social priorities and ultimately the wishes of voting citizens of each nation. The upshot is that clinical and economic data that is accepted in one market may not cut it in another. The standard-of-care in Germany for a particular condition may differ from that in France. Economic models that convince UK payers might not impress those in the Netherlands. The financial health of particular countries or regions will also heavily influence their willingness and ability to pay for new medicines.

What European payers all do share, however, is an aggressive quest for value. Often, they apply similar criteria (1). Because most European purchasers are tax-payer funded, that quest is more urgent than in the US market, where a far greater proportion of citizens are covered by commercial (private or employer-sponsored) insurers. Europe houses some of the most globally influential Health Technology Assessment (HTA) agencies – for example, guidance from the UK’s National Institute of Care & Health Excellence (NICE) will reach international audiences with countries also actively adopting NICE methodology.

Biosimilars – Highlight market access challenges across Europe 

Biosimilars nicely illustrate both the similarities and differences among European payers. Biosimilars are lower-priced, close-copies of specialist drugs like Herceptin® or Remicade®.  The financial pressure faced by many European governments was in large part what drove Europe to embrace these medicines in the mid 2000s, almost a decade ahead of the US. The EMA approved its first biosimilar in 2006, and around 40 more since. The US Food and Drug Administration (FDA) has approved 11. (2)

Today, amid growing competition, price discounts on biosimilar drugs in Europe range from approximately 15 to over 50%. (3).With more in the pipeline as patents on originator drugs expire, biosimilars are becoming one of the most important treatment classes for patients and health systems.

Deep local knowledge is crucial when developing market access strategies 

Of course, biosimilar uptake (like that of any other drug class) isn’t homogenous across Europe. These are complex treatments for often serious diseases, and Europe’s national payers, and clinician-prescribers don’t all share the same attitude toward them. Appropriate education, high-quality data, and a deep understanding of local concerns, practices and regulations are key to ensuring that these important medicines are safely integrated into health systems. Equally important is understanding the local purchasing or tendering system. There is substantial variation across Europe in how biosimilars are procured, with almost exclusively national tendering in some countries, and regional or local tendering in others.

Such local knowledge is key to successful market access and uptake of all medicines in Europe, biosimilar or otherwise. Partnering with experts that have their feet on the ground and a finger on the pulse in each market offers international R&D-based organisations an effective way to ensure that European patients can access their medicines.

For all its challenges, Europe is too important for any drug company to ignore. With an ageing population of over 700 million in Europe and five of the world’s top pharmaceuticals markets by value (4), Europe’s healthcare systems need, more than ever, therapies that can demonstrably improve health outcomes.

For more information on how to navigate Europe’s complex healthcare systems, contact: businessdevelopment@mundipharma.com

References:

1. Dunlop, W.C.N., Mullins, C.D., Pirk, O. et al. PharmacoEconomics (2016) 34: 1051. https://doi.org/10.1007/s40273-016-0427-

2.Cohen, J. (2018) What’s Holding Back Market Uptake of Biosimilars? Forbes, June 20, 2018. https://webcache.googleusercontent.com/search?q=cache:u_xncKPArUwJ:https://www.forbes.com/sites/joshuacohen/2018/06/20/whats-holding-back-market-uptake-of-biosimilars/+&cd=20&hl=en&ct=clnk&gl=lu&client=safar

3. Mullard, A. (2017) Bracing for the biosimilar wave. Nature Reviews Drug Discovery, March 2017. http://www.nature.com/articles/nrd.2017.36

4. https://www.worldatlas.com/articles/countries-with-the-biggest-global-pharmaceutical-markets-in-the-world.html

 

 

What the patient access experts are saying

My roundup from the recent PharmAccess Leaders Forum, NextLevel Pharma, London by Will Dunlop, Head of Market Access, Mundipharma International

I had the privilege of attending and speaking at the PharmAccess Leaders Forum, NextLevel Pharma, in London recently. Pricing, reimbursement and patient access experts from Health Technology Assessment (HTA) agencies, pharmaceutical firms, academia and consulting shared their views on patient access progress and challenges. Here is what I took away from the first day:

Pan-European HTA is an important goal, but cannot replace local expertise

Efforts continue to simplify and expedite European patients’ access to new medicines through a more coordinated HTA process. The European Network for Health Technology Assessments (EUnetHTA) program seeks to increase collaboration and information-exchange across national HTA agencies. Its latest initiative, Joint Action 3, assembles 81 regional, national and not-for-profit partners to develop common assessment methodologies, and to pilot and produce joint clinical assessments as well as full HTA reports.1

The EUR 20 million program is a worthy initiative and may well provide greater consistency across national HTA processes.1 But it cannot, in the foreseeable future, replace companies’ need for deep expertise at the national, pan-national and local level in order to successfully access key European markets.

That’s because Europe’s health systems, though they share some similarities, are also very different – in how they’re funded, how they’re organized, and in their priorities. These concerns were communicated at the event. Representatives from Germany’s reimbursement authority, the Federal Joint Committee (GBA), raised some specific concerns about further efforts to harmonise European HTA.2

 

HTA agencies want more engagement with pharma

Several of the major HTA agencies, including NICE in England and Wales, and GBA in Germany, are doing what they can to help manufacturers assemble the right evidence to ensure swift patient access. They’re promoting greater consultation with pharmaceutical companies, and are trying to make their decision-making processes more transparent.

This is a much-needed move. In Germany in 2017, only 2 out of 229 GBA assessments received a “major” additional benefit 3, which suggests there is more work to be done in aligning pharma’s and GBA’s expectations. GBA now has an online consultation platform4, while NICE continues to promote new forms of engagement through the NICE office for Market Access (OMA). 5

These initiatives can help inform a companies’ market access strategy through the therapy life cycle, and we have found our own interactions with HTA highly valuable. But such meetings supplement, rather than replace, the deep market knowledge and understanding of local health systems that pharmaceutical companies must have.

 

Pricing and outcomes-linked deals are increasing, despite challenges

Novel drug pricing and payment arrangements, and outcomes-linked reimbursement schemes continue to generate lively discussion and interest. The recent approval in the US of high-priced cell- and gene-therapies are making these discussions more urgent. Some of these specialised medicines are designed as one-time treatments with the potential to significantly improve outcomes for some patients over the long-term. They cost hundreds of thousands of dollars, but robust evidence of their long-term efficacy is still lacking.

Akshay Kumar, Principle at Pope Woodhead, outlined the access barriers facing such medicines, many more of which are expected on the market in the next few years. There is no established reimbursement pathway to suit such drugs, nor any mechanism to spread the initial high upfront cost. Value frameworks within which to compare the treatments to existing therapies are also lacking.

The situation is already forcing more creative risk-sharing deals between manufacturers and payers, though. In the US, where we have seen a rise in the profile of such arrangements6, Spark Therapeutics has agreed to rebate certain US health insurers if its gene-therapy LUXTURNA™ (voretigene neparvovec-rzyl), for patients with a form of blindness called biallelic RPE65 mutation-associated retinal dystrophy, fails to deliver expected outcomes. These outcomes, assessed according to pre-agreed measures, will be monitored at 30 days, 90 days and at 30 months.7 The company is also discussing possible installation payments with the (government) Centers for Medicare and Medicaid Services (CMS).

Novartis also announced an outcomes-based deal with CMS around its cell-therapy Kymriah (tisagenlecleucel), indicated for certain kinds of severe pediatric blood cancers. CMS will only pay if patients have responded to the treatment at the end of the first month.8

Risk-sharing deals have a slightly longer history in Europe, with mixed results. Italy’s health care system has reportedly saved far less than expected from its performance-based schemes – just €121 million out of a total of €3,696 million in relevant drug costs.9 There are also concerns about the added cost and administration required for such schemes.

Despite the challenges, many payers remain open to innovative pricing and outcome schemes. In a survey of EU payers, led by Mundipharma International, 85% of respondents said innovative pricing agreements would be more attractive than a discount under specific circumstances, such as if they offered greater cost reduction or helped manage uncertainty.10

 

Pharma firms must organise themselves to achieve patient access

 Presentations by Ana Céspedes (Merck KgA, Darmstadt, Germany) and myself had similar themes about what the inside of a pharmaceutical company needs to look like to successfully achieve patient access. Ana proposed five ways to help fully integrate a product’s value story across its life cycle, thus ensuring patient access. These including upgrading R&D objectives from drug approval to drug reimbursement; aligning company functions around patient value and outcomes; allocating investment into continuous value demonstration as a competitive advantage; making pricing and contracting central strategic priorities and upgrading the company’s go-to-market model to include medical affairs-sales-value, access and policy.

I presented research on “When Science is Not Enough: A Framework Towards More Customer-Focused Drug Development.” This research, conducted with the University of Cambridge Judge Business School, discussed the economic, behavioural and organisational barriers to achieving a customer-focused business model, and provided possible solutions.11 One of those is the BEACON framework, a practical and accessible tool to help communicate patient and payer value throughout any organisation.12

If you missed the conference, wish to learn more about Mundipharma’s market access capabilities, or for any potential partnership discussions, please do not hesitate to get in touch (will.dunlop@mundipharma.com).

References

  1. https://ec.europa.eu/health/sites/health/files/technology_assessment/docs/com2018_51_en.pdf; https://www.eunethta.eu/ja3-archive/
  2. https://ec.europa.eu/health/technology_assessment/consultations/cooperation_hta_en
  3. http://cddf.org/files/2017/09/1720-Florian-Jantschak.pdf
  4. https://www.g-ba.de/institution/themenschwerpunkte/arzneimittel/nutzenbewertung35a#abschnitt-11
  5. https://www.nice.org.uk/about/what-we-do/office-for-market-access
  6. http://www.commonwealthfund.org/publications/issue-briefs/2017/sep/outcomes-based-contracts-high-drug-spending
  7. http://ir.sparktx.com/news-releases/news-release-details/spark-therapeutics-announces-first-their-kind-programs-improve
  8. https://www.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm-ctl019
  9. https://www.sciencedirect.com/science/article/pii/S1098301514047251
  10. Innovative pharmaceutical pricing agreements in five European markets: A survey of stakeholder attitudes and experience https://www.healthpolicyjrnl.com/article/S0168-8510(18)30051-4/fulltext
  11. “When Science is Not Enough: A Framework Towards More Customer-Focused Drug Development.” https://rdcu.be/NoW8
  12. “BEACON: A Summary Framework to Overcome Potential Reimbursement Hurdles” https://rdcu.be/NjST

 

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