Regulatory Insights: Flexible Regulatory Pathways Accelerating Access to COVID-19 Treatments

In this blog post, John Joines, senior manager of regulatory intelligence, policy and advocacy, and Christina Cocciardo, director of regulatory affairs, discuss some of the flexible and emergency use regulatory pathways facilitating fast-track access to potential or promising new COVID-19 medicines around the world. A recent post focused on similar pathways available in the U.S.

The need for flexible regulatory pathways to address COVID-19

Across the globe, regulatory authorities are supporting sponsors investigating potential treatments for COVID-19; many have acted with quick responses to scientific advice, informal commitments to expedite application review and facilitation of site selection in order to prioritize clinical trials and product approvals for potential treatments to address the pandemic. As of 12 June, there were reportedly 2,169 clinical trials globally with 777 candidates seeking to identify COVID-19 treatment.¹

Global regulatory pathways accelerating patient access to COVID-19 treatments

European Union

The European Medicines Agency (EMA) has set out extensive guidance to help accelerate the identification of potential treatments for COVID-19 and encourages contact as soon as possible to discuss regulatory strategy. Various rapid regulatory procedures are available, in line with the EMA’s plan for emerging health threats:   

• Rapid scientific advice
• Rapid agreement of pediatric investigation plans including rapid compliance check
• Streamlined timelines for review of marketing authorisations
• Extension of indication and extension of marketing authorisations
• Compassionate use
• Rolling data reviews

Full details for all procedures are available on the EMA website. Here we discuss some of the pathways being employed to support early access to COVID-19 treatment(s) in the EU: 

Compassionate Use

Compassionate use programs make products in development available to groups of patients who cannot enter clinical trials and who have no satisfactory authorized treatment available to them.

Compassionate use was established by Article 83 of Regulation (EC) No 726/2004, with programs coordinated and implemented by EU member states in line with their national legal framework. Strict criteria apply. For example, the medicine being requested must be undergoing clinical trials or have entered the marketing authorization application process, and patients must have a chronic or seriously debilitating or life-threatening condition to be eligible.

In March 2020, a number of EU member states requested the EMA’s opinion for compassionate use for an antiviral candidate treatment for COVID-19, thus making it accessible to patients.

Rolling Review

Rolling review procedures can be used by the EMA in a public health emergency to allow assessment of data for a promising medicine on a rolling basis as data becomes available.

For one potential treatment for COVID-19, the EMA’s Committee for Human Medicinal Products (CHMP) is utilizing this approach, reviewing preliminary data from clinical studies and supporting data from compassionate use programmes in addition to product quality and manufacturing.

At the conclusion of the first rolling review cycle, the CHMP invited the manufacturer to submit further data together with an application for conditional marketing authorization (CMA). On 8 June, the first CMA application was received by the EMA and its evaluation began — a first for a COVID-19 candidate treatment in the EU.

Depending on the robustness of data and request for further information, the EMA assessment opinion may be issued within weeks. Learn more about how EMA has used rolling review for potential COVID-19 treatments here.

Japan

In May 2020, the Japanese Regulatory Authority, the Pharmaceuticals and Medical Devices Agency (PMDA) utilized the “special approval for emergency” pathway to approve the first emergency treatment for COVID-19 in Japan. ²

The special approval pathway is an existing regulatory procedure detailed in article 14-3 of the Pharmaceuticals and Medical Devices Act. The article allows for approval of a medical product when it meets the following conditions: “1) an emergency situation requires an unapproved medical product to be used to prevent damage to public health caused by the spread of diseases, 2) such emergency situation cannot be managed appropriately by any means other than the use of the unapproved product, and 3) such product is legally available in a country with a regulatory system for medical products that is equivalent to Japan.”

Sponsors should be aware that approvals via the special approval pathway may be granted with associated conditions. For example, the following conditions apply for the product mentioned above:

• written informed consent is required prior to administration to patients
• a risk management plan must be implemented
• results of additional clinical studies must be submitted as soon as possible, within nine months
• surveillance/registry of all patients to be conducted during a designated period

India

The Indian Regulatory Authority, the Drug Controller General of India (DCGI) has also taken action to fast-track the availability of potential COVID-19 treatment to patients, authorizing the first product on 1 June.

Additionally, on 5 June, the Indian Ministry of Health & Family Welfare introduced a compassionate use scheme, by a draft amendment to the New Drugs and Clinical Trials Rules 2019. Under the scheme, applications for compassionate use must be made by hospitals or medical institutions and, as is the case with compassionate use schemes in other jurisdictions, specific criteria must be followed to obtain permission to import an unapproved medication.

Taiwan

In Taiwan, the Pharmaceutical Affairs Act (“The Act”) amendment 2016³ establishes processes that enable early access to potential treatments in emergency situations. Applications should be submitted to the Taiwan Food and Drug Administration (TFDA), and include a treatment plan, references to supporting literature, drug specifications, quantity of medicinal product and proof the product is approved in another jurisdiction.

Conclusion

Regulatory agencies around the globe continue to provide guidance, consistently update regulatory recommendations and support sponsors to address the COVID-19 pandemic, recognizing the need to streamline and fast-track potential treatments to protect the health of their citizens. We are witnessing an impressive response by the global biopharmaceutical industry to embrace these pathways to rapidly identify treatments for the growing number of patients in need and vaccines to prevent and limit the spread of COVID-19. Our regulatory intelligence policy and advocacy (RIPA) team continues to track and navigate these increasingly flexible and novel regulatory pathways to support our study teams, clients and most importantly, our patients.