Europe’s leading nations should commit to a rapid overhaul of health technology assessment (HTA) and bold initiatives such as new pan-European real world evidence (RWE) frameworks, in order to nurture the nascent cell and gene therapy revolution in the region.
That is the conclusion of a new report from The Alliance for Regenerative Medicine (ARM), the international advocacy organisation representing cell and gene therapy companies and the broader field of advanced therapy medicinal products (ATMPs).
ARM has more than 70 members across 15 countries in Europe, and is working closely with European stakeholders, looking to create a leading commercial and regulatory environment for ATMPs in the region.
The last 12 months have seen the European approval and launch of groundbreaking therapies in the field, most notably the first two CAR-T therapies, Novartis’ Kymriah and Gilead/Kite’s Yescarta, plus Spark/Roche’s gene therapy Luxturna, and most recently, the approval of Bluebird’s gene therapy Zynteglo.
Investment has been flooding into the cell and gene therapy pipeline in that same period, despite the fact that the commercial model for these therapies is far from established.
Janet Lambert, chief executive of ARM, commented:
Advanced therapy medicinal products have extraordinary potential to alleviate human suffering. Clinical studies indicate that many cell and gene therapies will provide a long-lasting, even curative effect for many patients with debilitating or fatal disorders. These treatments represent a growing share of the biopharma development pipeline, and an exciting wave of transformative therapies will be coming to market soon. As a society, it is imperative that we promote innovation in this area and tear down barriers to patient access.
Leaders from the HTA and reimbursement authority side who participated in the report included experts from leading EU nations, including Frank-Ulrich Fricke, health economist and member of the arbitration board on drug prices in Germany, Ulf Persson, health economist in Sweden, Pilar Pinilla-Dominguez, NICE’s ATMP expert in the UK and Mira Pavlovic, HAS/EMA adviser, early dialogue expert in France.
The experts recommended faster uptake of new payment models like conditional reimbursement, pay-for-performance, and annuity-based payments – however no healthcare system is keen to take on tens of millions in extra costs, which means access is still likely to be rationed.
Existing models include conditional reimbursement, such as England’s Cancer Drugs Fund and Scotland’s fund for ultra-orphan drugs.
Pay-for-performance deals for traditional drugs are also becoming established in Europe and the US, linking price or rebates to pre-defined outcomes.
Annuity models, whereby payments are spread over several years, are also being pioneered. Novartis’ $2.12m SMA gene therapy Zolgensma – now the world’s most expensive drug – has been launched in the US with such a scheme.
Meanwhile Bluebird is proposing a similar five-year instalment plan for its €1.57m beta thalassemia therapy Zynteglo.
The report recommends a number of key measures, including:
- A quick adoption of new payment models such as conditional reimbursement, pay-for-performance, and annuity-based payments
- ATMP-dedicated funds, allowing health systems to invest in ATMPs that offer the potential for long-term benefits
- Better-adapted health technology assessment (HTA) methods, including greater use of real-world evidence (RWE) and development of infrastructure required to collect and use high-quality RWE
- Expansion of opportunities for early dialogue between ATMP developers and payers, supported by increased EU funding
- Development of pan-European initiatives to ensure timely and effective access to cross-border healthcare for patients
Despite the potentially curative nature of these therapies, and the efforts to spread cost, there is resistance to the high prices in Europe and the US.
Analysts at Bernstein last week put out an update on early US reimbursement decisions on Zolgensma, finding that initial coverage decisions on the gene therapy from 11 payers to be “surprisingly restricted.”
Slow uptake, coupled with manufacturing cost and capacity problems could hobble the field’s development. Numerous pharma and biotech firms are gambling on these issues being resolved in the next few years in order for them to recoup their own upfront investment.
The next therapy area which could be transformed by cell therapy is multiple myeloma, where BCMA-targeting CAR-T therapies are in the pipeline. However next-generation antibodies with comparable efficacy but with lower costs and complexity in manufacturing could undercut these candidates.