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).


  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