Regulation optimisation could save thousands of life years

Embracing a “once-in-a-generation opportunity” to improve access to oncology treatments could save thousands of years of life across the European Union.

Without “decisive action” cancer deaths across the European Union (EU) will increase by almost a quarter by 2035, making it the bloc’s leading cause of death.

Expediating access to new treatments is key to avoiding this devastating outcome, according to a new publication from the European Federation of Pharmaceutical Industries and Associations (EFPIA).

Every Day Counts: Improving Regulatory Timelines to Optimise Patient Access to Innovative Oncology Therapies in Europe says optimising regulatory pathways is not only desirable, but, as the global response to COVID-19 has shown, it is possible.

Long and winding process

The report, which was carried out by Vintura on behalf of the EFPIA Oncology Platform, is a follow up to 2020’s Every Day Counts – Improving Time to Patient Access to Innovative Oncology Therapies in Europe.

The first publication focused on market and patient access, whereas the new report concentrates on the final part of the route to marketing authorisation: from the final scientific opinion of the Committee for Medicinal Products for Human Use (CHMP) to the final decision of the European Commission (EC).

Said the authors: “The process officially takes 67 days, which includes a linguistic phase and a decision-making phase. In the last five years, the total process has taken a median of 60 days for oncology products, although the range extends from 33 to 198 days.

“The experience of marketing authorisation being granted for COVID-19 vaccines within one day of the final CHMP opinion has shown clearly that the European community of stakeholders is able to work together to optimise this process greatly.”

While the report does not suggest this one-day process become the norm, it does point out how it highlights the huge potential to reduce time to market access.

Tide of change

The current timelines between CHMP opinion and EC decision come at a cost, said the authors, pointing to an analysis of 11 recently authorised oncology treatments.

It showed that the regulatory steps between CHMP opinion and EC decision accounted for 18,600 years of potential life lost. The full extent of this figure, however, is likely to be far greater when considering all oncology indications.

“In addition, optimisation is necessary to increase efficiency and future-proof the system, which is currently overburdened as a result of challenges such as Brexit, COVID-19, and the rising number of new medicine assessments,” said the report, which was developed with the input of 35 organisations. These included health technology assessment (HTA) bodies, healthcare professional associations, patient organisations, policy makers, academics, payers, and pharmaceutical companies.

“There is also momentum for change, as the EC is currently considering changes to pharmaceutical legislation as part of the EU pharmaceutical strategy; a process which only occurs once in a generation. Even if only small improvements are made, they could have a significant impact on patients.”

Options for change

The paper sets out three potential solutions, based on an extensive review of grey and academic literature, and assessment of the health- and socio-economic gains of reducing the time to access.

The first is conducting the decision-making and linguistic phases in parallel, which could accelerate timelines by 12 days and save 3,300 life years.

Secondly, the increased use of digital tools during the linguistic phase, could save 10 days and 2,800 life years.

“Improvements are needed to future-proof today’s overburdened system and reduce the potential for future delays,” said the report.

“Greater digitalisation and an increase in the number of human resources should be considered to reduce the workload pressure associated with today’s process, and may further reduce timelines.”

In addition, shortening the written procedure in cases where EU member states foresee no objections could accelerate timelines by 15 days, and save 4,200 life years.

“Improving the transparency and predictability of the process between the final CHMP opinion and the EC decision would allow for better planning of the subsequent pricing and reimbursement (market access) process.

“Further optimisation” could be achieved by starting national HTA processes immediately following a positive CHMP opinion as the final EC decision-making process continues, said the authors.

In the last 10 years, they said, EC decisions have always aligned with CHMP opinions. Starting the HTA process earlier, then, would be a pragmatic way to avoid more than 60 days of delay to patient access.

Every day counts

The report makes it clear that now is a time for change, and that it is the responsibility of stakeholders across the health and life sciences ecosystems to embrace the opportunity – for all patients.

“Combining these solutions can save thousands of years of life across the European Union, especially since these solutions are not limited to oncology therapies.

“Together, let’s change this process and make use of the once-in-a- generation opportunity of the review of pharmaceutical legislation. Because for patients, every day counts.”

To read the full report, click here.