#BIOSPAIN2023Interview | "Regulatory agencies are aware of the need for new standards in the field of advanced therapies and personalized medicine"

The 72nd World Health Assembly, held in May 2019 by the World Health Organization (WHO), established two documents that currently stand as the main strategies to advance in the elimination of avoidable harm to patients in healthcare. We are talking about the "Global Action on Patient Safety" and the "Global Patient Safety Action Plan 2021-2030".

Raising awareness and expanding knowledge worldwide regarding patient safety is one of WHO's goals, aiming to reduce the damages that can arise from malpractice or negligence in healthcare. With this backdrop, World Patient Safety Day was celebrated on September 17th with a clear message.: giving voice to patients to ensure their participation in policy formulation, joint design of safety strategies, and promoting their involvement in the care they receive.

One of the historical demands of patients refers to excessive waiting times to access innovative medications that have been approved by relevant regulatory agencies. According to the results of the W.A.I.T. (Waiting to Access Innovative Therapies) Report, published in April 2023, Spaniards have to wait an average of 629 days to access innovative medications. The report highlights the growing inequities among different European countries.

Reducing waiting times for access to innovative medications is a problem that directly affects patient safety and limits their options and prognosis for certain diseases. Understanding and evaluating the regulatory and bureaucratic barriers that exist both at the national and international levels is crucial for developing more efficient models for evaluating innovative medications and establishing the foundations of new regulatory frameworks.

On this World Patient Safety Day, we analyze this situation alongside Oriol Penon, Director of Strategy and Innovation at Asphalion, an international consulting firm specializing in regulatory and scientific affairs and a Silver Sponsor of BIOSPAIN 2023.
 

AseBio. One of the main problems affecting innovative medications is the long period of time that elapses from their authorization to their availability for patients. Why does this happen?

Oriol Penon. This extended period is due to a combination of various factors. Innovative medications, for example, must be directly registered through the European Medicines Agency (EMA) using a procedure called "centralized."

The centralized procedure allows for the authorization of a medication based on a single evaluation valid throughout the EU. However, once the medication is authorized by the EMA, before it can be available to patients in a specific EU country, it is necessary to negotiate the price and reimbursement at the national and regional levels within the context of each country's national healthcare system. This local negotiation process is one of the factors that can often prolong the access process for patients since dealing with "payers" may not always be a swift process.

Another factor to consider is the distribution and logistics processes, which can sometimes take time to set up, in addition to the need to comply with quality standards and specific regulations for each country.

AseBio. There are many voices criticizing the use of outdated procedures for evaluating the viability and safety of innovative medications. What are the main regulatory barriers that innovative medications face in Spain and Europe?

Oriol Penon. One of the main barriers is the lack of specific regulatory guidelines that help define the regulatory pathway for these types of medications. For example, in the field of advanced therapies, which includes gene and cell therapy products, there are still no guidelines that standardize and define how to demonstrate efficacy and safety data for certain types of products.

This fact means that researchers and companies developing these medications do not have guidelines to follow, requiring greater support from agencies to ensure that the product can meet the regulatory requirements for authorization, always considering the benefit-risk of the product.

AseBio. Are there regulatory agency procedures that can accelerate patient access to these types of medications?

Oriol Penon. Yes, there are special programs implemented by agencies such as the EMA or the FDA to expedite the market access of innovative medications.

For instance, the PRIME program stands out, through which the EMA offers early and proactive dialogue to drug developers dealing with serious diseases or medical needs that are not met. This support helps optimize the generation of robust data on the benefits and risks of the medication, allowing for an accelerated review process and faster availability of the medication to patients. Another noteworthy procedure is the "Rolling Review", designed for situations of health emergency, which has been used for the approval of Covid-19 vaccines, reducing evaluation times.

Other programs like compassionate use or hospital exemption allow patients to access medications that have not yet been approved but are in advanced stages of clinical development. This can be particularly relevant for patients with severe diseases or those with no treatment options.

Lastly, it's worth mentioning the importance of specific programs existing both from the EMA and the FDA for the development of orphan drugs. These programs incentivize the development of drugs targeting very small numbers of patients by offering certain benefits.

AseBio. Asphalion, through its consultants, is in direct contact with the European Medicines Agency and national competent authorities for the development of new regulatory standards. What steps are being taken in this regard? What are the main challenges?

Oriol Penon. The Agencies are highly committed and aware of the need for the development of new regulatory standards, especially in the field of advanced therapies and personalized medicine. Collaboration between regulatory agencies, the pharmaceutical industry, researchers, and patients is essential to address these challenges and continuously improve regulatory standards.

For example, Asphalion is participating in the ARDAT project, co-funded by the European Commission. This project brings together a total of 34 partners from academia, the pharmaceutical industry, and small and medium-sized enterprises with the aim of developing and providing tools that advance knowledge in the field of gene and cell therapy and, in turn, accelerate the development of new treatments for rare diseases.

AseBio. What does Asphalion expect from BIOSPAIN 2023?

Oriol Penon. After a very challenging period with hardly any in-person events, the Asphalion team is eager to reconnect with the entire sector and continue sharing experiences and new challenges in the future.