Duchenne UK Collaborates with Industry to Speed Payer Value Assessments

Rare Daily Staff

Duchenne UK has launched a collaboration with five pharmaceutical companies developing treatments for Duchenne muscular dystrophy to create a “robust and relevant evidence-based” in the hopes of accelerating payers’ decisions on which medicines to fund to treat the rare and fatal progressive muscle disease.

Dubbed Project HERCULES, the organization has committed $263,000 (£200,000) to fund the initial stages of the project. The intent is to provide a central core of data to allow for informed health technology assessments for DMD therapies to speed the process. It will enable drug developer to work together to create evidence to be used by drug pricing watchdogs, such as The United Kingdom’s National Institute of Health and Care Excellence.

The effort comes as the first of a new generation of DMD therapies have come to market and a growing pipeline of experimental treatments are making their way through clinical development. The group said the aim of HERCULES is to address the challenge after medicines reach regulatory approval—how to get governments and health services to pay for these medicines.

“In order to persuade government bodies to pay for new medicines, companies often have to provide complex data analysis and burden of care studies which are costly and time-consuming,” said Emily Crossley, co-founder and joint CEO of Duchenne UK. “The aim of HERCULES is for companies to collaborate together on building such models, to save time, money, and ensure that the collaboration speeds up access for patients.”

Duchenne UK notes that the pace of innovation in treatments and medicines is outstripping the time it takes to approve them for use in the U.K.’s National Health Services. The lack of evidence available for therapies to treat rare diseases like DMD to meet the requirements for access and pricing decisions compounds the delays.

To win a positive recommendation from NICE for the NHS to fund a drug, companies must do a large amount of specialized and detailed work that includes data collection, economic modelling, and quality of life measurements. That can be difficult, time consuming, and expensive, the group said.

HERCULES seeks to improve modelling, build a broader evidence base, reduce duplicative or inconsistent efforts, improve engagement with patients, lower costs and management time, and make better use of patient input. Companies work together to develop a shared model that will focus on developing common tools and practices.

The first phase of the project is underway. Working with leading academic groups it focuses on synthesizing the current evidence base, the cost and burden of DMD and lives lost to it, and current measures used in DMD clinical trials. The project will also look at how quality of life for boys with DMD is measured and seek to develop better measures.

The second phase of the project will map the impact of DMD on patients, families, and careers, and develop a disease level economic model to support pricing and reimbursement decisions. The second phase of the project will be announced in 2018.

“HTA agencies can struggle with decisions in the absence of a solid evidence base, be that data on the burden of the disease or the likely disease progression. Through its collaboration with patient organizations, industry, universities, and other partners, said Josie Godfrey, former director of NICE, who is heading the HERCULES Project. “Hercules will ensure all companies can produce the best possible evidence for HTA agencies—making decisions easier, fairer and hopefully enabling faster access to the best new treatments for DMD.”