Gene therapies, many of which offer short-term treatment regimens, directly address the underlying cause of disease. As a result, they could bring transformative benefits to patients by halting disease progression and in some circumstances they may offer the prospect of a cure.
Questions are being raised in Europe around whether adopting these technologies into routine clinical care threatens the financial sustainability of health systems. There are also concerns regarding the challenges that gene therapies bring: higher upfront prices, uncertainty about the long-term duration of clinical benefit and potential safety concerns.
Despite this, the promise of transformative value through long-term benefits and potentially curative treatments is motivating stakeholders to explore solutions to overcome these challenges and bring gene therapies to patients.
In a webinar held on 16 March 2021, prominent stakeholders, representing policy, patient and industry perspectives debated the key issues surrounding the adoption of gene therapies into health systems.
This summary report provides an overview of the different perspectives that were presented and the key themes that emerged from the webinar. Throughout we highlight quotes, encourage you to replay short clips from the event, and invite you to offer your perspective on the key topics.
Firth I and Hampson G, 2021. Gene therapies: are we ready? Event Summary. Office of Health Economics. Available from: https://ohe.turtl.co/story/gene-therapies-debate-report
Grace Hampson, Associate Director OHE ghampson@ohe.org
Former Minister of Health for Ireland
Managing Director, ESAH Pharma
Director of Public Affairs, EURODIS
Simone Boselli joined EURORDIS in April 2017. A member of the European and International Advocacy team, Simone contributes to European policy development and specifically represents EURORDIS in policy discussions on access to therapies. Simone has also previously worked for two leading consultancies in Brussels and specialised in health advocacy and government affairs.
Mary Harney is a former senior Irish politician. She served as Tánaiste from 1997 to 2006, Minister for Enterprise, Trade and Employment from 1997 to 2004, and as Minister for Health and Children from 2004 to 2011. Mary provides consultancy and advisory services to a range of corporate clients in the technology, healthcare, pharmaceutical and financial services sectors.
Annie Hubert provides a wealth of experience and services in healthcare biotech, with expertise in market access, regulatory and public policy relating to advanced therapies. Specialties: ATMPs, EU Government Affairs, Regulatory, Market Access, and Public Policy. Until December 2020, Annie was Senior Director, European Public Policy for the Alliance of Regenerative Medicine.
The debate brought together three perspectives: policy makers, industry and patients. Each group has different priorities but they expressed optimism about gene therapies and confirmed that some of the challenges are shared between different groups.
PATIENTS Patients are excited for gene therapies but the delay in access feels long. There is concern that gene therapies are not affordable at current prices. The hope is that in the future there will be no delay between regulatory approval and patient access across Europe. Innovation in processes around regulatory assessment, value appraisal and reimbursement decision making need to keep up with scientific innovation.
Watch the patient perspective here
POLICY There is a general lack of education among "the political class" about gene therapies with some people simply remarking "are those are the ones with the high price?". Short-term opportunity cost is a particular constraint for this group. Without data, we don't know what we are getting.
Watch the policy perspective here
INDUSTRY There is optimism now that some therapies are being approved and reimbursed. However there are practical barriers to widespread use of novel payment mechanisms including accounting rules and data infrastructure. A societal perspective for value assessment would help to capture the full value that gene therapies bring. Early dialogue is needed between all stakeholders to make sure that development decisions match the expectations of regulators, payers, health technology assessment (HTA) agencies, patients and clinicians.
Watch the industry perspective here
Patients, regulators and clinicians are ready for gene therapies. HTA agencies are gaining experience. Policy makers and payers are not ready. Some people may be anti-gene therapy because of a fear of this new technology.
Evidence generation in real world settings will be needed to overcome uncertainty around clinical outcomes. Registries, electronic health records all need to be adapted for this.
Speakers looked to collaboration across Europe to overcome some of the barriers to gene therapies. Allowing patients to travel for treatment, EU data standard setting, as well as collaboration to remove European legislative barriers to gene therapies.
Science has delivered transformative therapies, but there are practical barriers to delivering them to patients. Some of the barriers discussed by speakers were:
• Lack of strong data systems for monitoring outcomes • Accounting standards that make annuity payments difficult • HTA process that are not ready to assess the full value of gene therapies.
Early collaboration between all stakeholders is needed to make sure plans are in place to limit delays in access. Companies, regulators, HTA agencies, payers as well as patients and clinicians all need a seat at the table.
Watch the chair, Adrian Towse, summarise the key discussion points from the debate
