Written by: Keith Tolley, Director, Tolley Health Economics Ltd, UK, Course Leader at the VSCR/SFU Diploma Programme in November 2018, and Max Lebmeier, Athena Market Access Solutions Ltd., UK
Much innovation in new health care technologies comes from small and medium biotech and pharma companies, with many recent advances in the areas of rare diseases such as muscular dystrophy, and gene therapies for retinal disorders.
Road to Reimbursement – A Challenging Process
However, all face the same ultimate challenge of getting to market, getting marketing authorization, and then reimbursement to generate sales and return on investment. But addressing only the requirements to obtain regulatory marketing authorization alone is not enough to also obtain reimbursement. To gain reimbursement, a biotech company also needs to consider evidence generation addressing the needs of payers and reimbursement authorities. Health Technology Assessment (HTA) organizations, as well as a pricing strategy which reflects the value of the product in light of payer and HTA requirements, are needed tools in this process. Negative reimbursement decisions, niching of allowed/reimbursed use of the product at best, curtails the potential for sales and return of investment and can prohibit it completely. Delays in reimbursement decision making, due to insufficient evidence generation within clinical development programs or alongside clinical trials and regulatory approval processes or pricing beyond what payers and HTA organizations consider value for money, can lead to substantial delays in a new innovative product gaining market access.
Achieving reimbursement and market access requires meeting the value needs of health care payers around the globe, who have Government funded Health Technology Assessment (HTA) agencies working on their behalf, appraising the clinical and health economic evidence to determine the clinical and cost-effectiveness of the new therapy versus current practice. The internationally recognized agencies include the National Institute for Health and Clinical Excellence (NICE) and Scottish Medicines Consortium (SMC) in the UK, Institute for Quality and Efficiency in Health Care (IQWiG), in Germany, the Institute for Clinical and Economic Review (ICER) in the USA and there are similar organizations across all key markets.
The focus of HTA is on measuring therapeutic value through patient relevant outcomes, such as health related quality of life (HRQoL) rather than surrogate measures and biomarkers that have traditionally been used to obtain regulatory approval.
Innovative Products Needs Investors
A big conference of biotech companies held in Amsterdam in Spring 2018 (Bio Europe)was a showpiece meeting including small and medium biotech/pharma companies with a whole range of innovative products in early development to meet unneeds across a multitude of rare and not so rare diseases, with many seeking to attract new investors and support from larger pharma to drive beyond phase 1 trials into phase 2 and 3 expansion, and to help achieve market access and successfully launch their new innovative therapies
However, most attention at this meeting was company presentations on the science and promise of their new therapies. Science and promise needs to equate to value to get the optimal price and reimbursement, so understanding cross country HTA needs and developing an early value package can add to the investment potential for new therapies and further attract potential investors.
Implementing HTA Requirements in the Reimbursement Strategy
Key elements of an early value package include a target product profile for market access, key endpoints and outcomes assessment, early health economic modelling and value argument development (Value dossier), as well as scientific HTA advice seeking program (NICE, payer, clinician) and an early assessment of the pricing potential.
Investors are increasingly becoming aware of market access and asking the key questions around (patient focused) value and value for money before investing in new therapies and companies.
Therefore, it is essential for personnel in small and medium biotech/pharma companies to gain an understanding of payer and HTA requirements and how to implement those into their own programs for their own products to optimize their chances to get reimbursement, sales and thus return of investment. Therapeutic Value evidence generation is a key phrase that needs to be understood and implemented in order for optimal market access to be a possibility.
The Vienna School of Clinical Research (VSCR) and the Sigmund Freud University Wien (SFU) are holding international Diploma Programmes on Health Economy, Health Technology Assesement (HTA) and Decision Making for Pricing and Reimbursement. The first module takes place in Vienna from November 15th to 18th. Head lecturers are Keith Tolley, BA, MPhil, Director of Tolley Health Economics, University of York, UK and Lieven Annemans, MSc, PhD Senior Professor of Health Economy, University Ghent, Belgium, past president ISPOR