No Time to Wait: Can Early Access Schemes Work for Gene Therapies?
Author(s)
Moderator: Adrian Towse, MA, MPhil, Office of Health Economics, London, UK
Panelists: Francois Houyez, Patient Advocate, European Organisation for Rare Diseases (EURORDIS), Paris, France; Carole Longson, PhD, Carole Longson Consultant, Manchester, UK; Oswald Bentinck, MSc, Novartis Gene Therapies, Wollerau, Switzerland
Presentation Documents
ISSUE: Patients with high unmet needs often face rapid deterioration or even death while waiting for approval of new potentially transformative therapies. Hence most European countries have compassionate use mechanisms that enable pre-regulatory approval access to such medicines. Designed for repeat therapy, most require free provision of therapy for the duration of the scheme. Most gene therapies for rare diseases are single administration, giving the manufacturer no prospect of recouping any costs from those patients.
There is currently a stand-off between key stakeholders. Patients are no longer prepared to wait, demonstrated by a rise in medical crowdfunding campaigns. Payers, under pressure to allow access early, fear undermining HTA, pricing and reimbursement negotiations, particularly if high price benchmarks are set. Manufacturers worry about the sustainability of agreeing to provide one-time therapies for free. Several questions must be explored: How can early access mechanisms be designed to meet the needs of patients while protecting innovation? Where does the balance of responsibility between the manufacturer and the health care system lie? If manufacturers can be reimbursed during early access, how can we ensure the schemes are sustainable? How can stakeholders agree a price and a payment scheme? Is there a role for a European fund for early access?OVERVIEW: The moderator will briefly introduce the issue (4 mins) and the panel will then debate the concerns of paid early access schemes for one-time therapies like gene therapies and their perspective on the questions set out above. Panelists will each speak for 12 minutes, providing their perspectives on the issues. 20 minutes will be reserved for audience discussion.
The panel will be of interest to those working in health policy, the regulatory- HTA interface, industry and patient organisations, as well as payer and clinical representatives.Conference/Value in Health Info
Code
204
Topic
Health Policy & Regulatory