The development of biologics and advanced-therapy medicinal products (ATMPs) is transforming medicine by introducing novel breakthrough therapies for previously uncurable diseases. However, several barriers stand between these therapies and widespread access. High treatment costs pose significant obstacles for patients and other stakeholders, including employers, government-funded healthcare programs, commercial payers, healthcare providers, and drug manufacturers.
Global initiatives have been established to support biopharmaceutical development and improve patient access. The US White House Office of Science and Technology Policy presented goals for the US bioeconomy in 2023, aiming to increase manufacturing scales of cell and gene therapies (CGTs) and reduce manufacturing costs tenfold over the next 20 years. The UK Life Sci for Growth package includes several investments to enhance commercial clinical trials, expand biological data banks, and encourage pension schemes to invest in science and technology companies.
The AGORA Initiative, a consortium founded by researchers at Great Ormond Street Hospital for Children, brings together international stakeholders to create sustainable solutions for children with rare and ultrarare diseases to access novel gene therapies. The Global Gene Therapy Initiative (GGTI) enables access to curative medicines for conditions such as sickle-cell disease and HIV infection in low- and middle-income countries.
The Role of Health-Technology Assessments
Regulatory approval does not guarantee a drug’s successful launch or patient access. Effective go-to-market strategies must incorporate critical factors, including high costs and unique financial challenges posed by curative treatments. Reimbursement processes for biopharmaceuticals vary significantly by geographic location, often influenced by whether payers require health-technology assessments (HTAs).
HTAs inform decision-making related to drug pricing and reimbursement, particularly in countries with universal-coverage healthcare systems. They rely on efficacy and safety data, patient-health-related quality of life measurements, life-expectancy impacts, and cost data. Incremental cost-effectiveness ratios (ICERs) compare new drugs to standards of care, enabling decision-makers to determine monetary values of new technologies relative to existing treatments.
Strategic Market-Access Challenges and Opportunities
As patients seek rapid access to newest biopharmaceutical therapies, regulatory and payer decisions are becoming more complex. A successful market-access plan includes several factors, such as incorporating market-access plans early into drug-development programs and generating data through real-world evidence (RWE).
RWE provides insights into real-world drug performance and helps drug developers demonstrate product values to payers and regulators. It can be used to fulfill postapproval requirements and support ongoing market access and reimbursement. Companies need to conduct real-world analyses on their product values and comparative effectiveness while focusing on specific subpopulations.
Addressing Global Variations in Reimbursement Approvals
Market-access strategies vary by country and payer model. Obtaining regulatory approval does not guarantee that a drug can be prescribed until it is included in each country’s reimbursement catalog. Market-access teams guide programs with a process incorporating attributes such as early visibility into total health-system costs and favorable positioning on drug formularies.
Market-Access Success Needs Experience and Expertise
Technological advances are changing the biopharmaceutical landscape, and companies must update their global market-access strategies to support emerging regulatory changes and payer requirements. A successful global market-access strategy must account for varying definitions of value across geographies. Many companies are outsourcing market access to experienced professionals who can navigate complex challenges and build evidence-based value propositions.
Spotlight on Cell and Gene Therapy Commercialization
Cell and gene therapies offer potential cures for previously untreatable conditions. Despite growth, CGT manufacturers face commercialization challenges, including complex and costly manufacturing processes. Specialized equipment, high-cost raw materials, and skilled-labor needs drive up production costs and patient pricing. Prominent agencies offer expedited pathways for CGT approval, such as priority review and accelerated assessments.
