Clarivate Plc (NYSE: CLVT), a global leader in connecting people and organizations to intelligence they can trust to transform their world, announced the release of its new report, Making gene therapy work for patients, payers, regulators and developers: Five key trends in gene therapy approvals and access. The report explores the challenges and triumphs of gene therapy, with an eye on how developers are addressing risk and the need for multiple modalities and how payers are exploring alternative pay methods.
Nearly 50 years since scientists first proposed the idea of modifying defective genes to treat genetic illnesses, the gene therapy revolution has evolved to include treatments that offer the promise of better health outcomes and/or quality of life for patients with rare diseases. Yet, there is a major gap between the needs of developers and payers faced with absorbing the costs of a wave of medical innovation elsewhere. Similarly, regulators are wrestling with policies and processes that were not designed with these treatments in mind.
The new report explores five key trends in gene therapy approvals, industry challenges and ways biopharmas can begin to address them. Trends covered in the report are:
- Longevity of gene expression and immune evasion remain challenging for developers
The duration of therapeutic effects and questions about long-term safety persist as important uncertainties with gene therapies.
- As drugmakers target broader populations, patients face barriers to access
For companies marketing gene therapies, pricing strategies will, as ever, need to account for unmet need, availability of alternative therapies and their cost.
- Costly therapeutics demand new metrics and payment models
The prospect of paying $2 million or more for a one-time administration of a gene therapy to a patient with a rare disease has created financial challenges for health insurers as the pipeline grows for these potentially life-saving cures. Payers are responding by exploring alternative financing arrangements and controls to manage drug utilization.
- The trend toward outcomes-based contracts takes on new urgency
The inordinately high upfront costs of gene therapy have rendered the traditional reimbursement models (e.g., buy-and-bill) unfeasible for many insurers. Payers are exploring alternative payment models, such as outcomes-based contracts between payers and manufacturers, among others.
- In the U.S., regulators wrestle with accelerated approvals model
The number of accelerated approvals granted by the U.S. Food and Drug Administration (FDA) has been increasing in recent years, with several parties expressing concerns about safety and efficacy, drug price inflation and drug approvals on weak evidence. Potential legislative actions could impact this pathway further.
Mike Ward, Global Head of Life Sciences and Healthcare Thought Leadership, Clarivate:
“Gene therapy remains a novel and disruptive field which presents significant challenges to traditional models of market approvals and payment. Even as the number of therapies in pipelines and available to patient begins to accelerate, the picture on market access and commercial launch for these products remains uncertain. To be successful, companies developing gene therapies will want to consider the patient journey as well as the market, competitive, regulatory and payer landscapes.”
Read the report, Making gene therapy work for patients, payers, regulators and developers: Five key trends in gene therapy approvals and access, here.
Clarivate draws on deep industry expertise and industry standard data to help life science partners identify market opportunities, anticipate barriers in order to deliver innovative treatments to patients and create a healthier tomorrow. To learn more, please visit www.clarivate.com.