The path to regulatory approval can be long and arduous. It can also be especially difficult for smaller companies to navigate. But a new report finds that early input from health authorities can help make the process more straightforward.
The report is based on information gathered through interviews with small- and medium-sized biotechnology companies, the European Medicines Agency (EMA) and HTA bodies. The report finds early engagement with regulatory authorities and health technology assessment (HTA) bodies to be beneficial for small- and medium-sized enterprises (SMEs), but that the path to engagement is not always clear.
The report was sponsored by Deerfield, a healthcare investment firm, and the biotech industry group EuropaBio.
Starting the Conversation
Many regulators offer companies opportunities for early discussions about their products’ development and regulatory pathway.
The European Medicines Agency (EMA) offers SMEs access to administrative and procedural assistance, as well as fee reductions and waivers for scientific advice. The agency believes that providing these incentives to SMEs can support the development of innovative new products.
Some HTA bodies have recently begun to offer advice to companies. However, the survey found few instances of HTA bodies providing scientific advice, and even fewer instances of advice being provided to SMEs.
Engagement with Regulators
One of the main issues the report seeks to address is the fact that some drugs are not authorized—not because of their effectiveness, but because they failed to meet regulators’ data requirements. To address this issue, regulators offer scientific advice to companies to aid them in designing their clinical programs.
To measure the effect of scientific advice, the report looked at new drugs approved by EMA between 2006 and 2014. The analysis found an overall approval rate of 74.9% for the 371 drugs considered, whether they received scientific advice or not. Drugs that received scientific advice only one time during their development had the same overall approval rate. However, the analysis showed drugs that received scientific advice two or three times had higher approval rates—82.5% and 88.4% respectively.
Engagement with HTA Bodies
The report also looked at how companies engage with four HTA bodies in Europe. Many HTA bodies require different data than regulators for their review of a product, such as comparator studies and pharmacoeconomic data.
Companies often stumble at the HTA stage because their clinical programs are not designed with HTA requirements in mind. Engaging with HTA bodies early can help steer companies in the right direction and provide insights as to the data HTA bodies will be expecting.
The report finds that oftentimes “companies lack HTA skills,” which can make getting input from HTAs a difficult task and suggests that companies should put additional effort toward understanding HTA requirements.
The companies participating in the survey listed a number of benefits and challenges in obtaining advice from regulators and HTA bodies.
Companies generally found scientific advice helpful toward understanding the perspectives and needs of regulators, but said they faced internal and external hurdles in obtaining the advice.
Internally, companies reported difficulty getting buy-in from senior management on obtaining advice from regulators. Companies also said it can be difficult to determine the right questions to ask regulators, and found there were too few opportunities outside of pre-submission meetings to meet with reviewers.
In the survey, companies suggested that a more unified approach from regulators and HTA bodies could reduce the complexity of product development. Companies also found that HTA bodies often lacked specific experience with advanced therapies and rare conditions.