Expedited approvals and eased requirements for COVID-19 research and treatment speed time to market and spotlight potential of standardized accelerated access pathways across wider range of groundbreaking therapies
- Accelerated approval pathways for clinical trials and research projects related to COVID-19
- Simplifying market authorization and reliance on expedited/priority review pathways
- Access via compassionate or emergency use exemptions
While authorities grappling with the pandemic have faced inevitable barriers to re-orienting their health systems at such a rapid pace, one immediate tool at their disposal has been simplifying health product market authorization procedures.
Regulators are thus widely looking at ways to accelerate access to potential COVID-19 treatments both in terms of drug development and licensing.
Accelerated approval pathways for clinical trials and research projects related to COVID-19
With many treatments in preclinical or clinical stages, regulators have emphasized easing regulatory requirements surrounding conducting clinical trials related to COVID-19. This approach has manifested in a number of areas, including accelerated pathways for evaluating research projects and ethics approval processes related to COVID-19. Regulators such as the U.K.’s MHRA are also offering expedited scientific advice and rapid reviews of clinical trial applications to support manufacturers and researchers investigating potential COVID-19 treatments. Regulatory authorities around the world have likewise reduced consultation fees related to COVID-19 to ease the approval process.
The EMA has released harmonized guidance on conducting clinical trials during the pandemic, introducing a number of flexibilities and adjustments to standard processes. The EMA has asked researchers to draw on the European Network of Centers for Pharmacoepidemiology & Pharmacovigilance (Encamps) Guide on Methodological Standards in Pharmacoepidemiology when designing studies and has designated EMA-ENCePP COVID-19 response group to facilitate robust but responsive clinical development during the pandemic. The European Commission has also committed to an EU Strategy for COVID-19 Vaccines, whereby it will fund vaccine manufacturing in order to secure supply of any future treatments
EU member states have followed with their own further policies amending standard processes to ease COVID-19 related investigations. Regulatory authorities such as Germany’s BfArM and the U.K.’s MHRA are prioritizing drug development, clinical trial applications and other research projects related to COVID-19. This includes expedited scientific advice and rapid review for trial applications as well as for research ethics approval processes, such as parallel review of applications where sign-off from multiple decision-makers is usually required (e.g., as required from both Colombia’s INVIMA Clinical Research Group and Research Ethics Committees). Similarly, trial reporting requirements have been widely simplified in the interest of flexibility amid the rush for a vaccine or other effective treatments.
Simplifying market authorization and reliance on expedited/priority review pathways
Further regulatory changes around simplifying drug registration procedures and selectively easing some evidence requirements, including post-approval data submissions, have been seen around the world as regulators consider the next step in market access.
With the potential for proven treatments to come to market, authorities are looking at ways to expedite standard market authorization procedures, both through drawing on the increasing proliferation of expedited access programs globally as well as through new special mechanisms rolled out during the pandemic. Authorities have also indicated that they will prioritize COVID-19 treatments for reimbursement review – for instance, NICE’s commitment to prioritize COVID-19 therapies in its assessments – but as available treatments are relatively few this has not been addressed as meaningfully across the range of global payers.
As part of a larger revision of drug authorization processes to facilitate expedited review pathways in 2020, such as for breakthrough therapies, Mainland China’s Center for Drug Evaluation (CDE) has also moved to speed authorization of COVID-19 vaccines or therapies under Special Review and Approval Procedures-" for emergency use.
Applicants with promising clinical and safety data will be subject to review times expected to be far shorter than traditional approval timelines through potentially waiving the need to complete requirements such as bioequivalence testing or other evidentiary benchmarks prior to authorization.
Brazil has similarly sanctioned temporary expedited registration for COVID-19 treatments, including measures such as potentially approving registrations conditionally with further data and/or additional evidence allowed to be submitted under agreed to follow-up plans. The French National Authority for Health (HAS) has further sped reviews as well, as discussed in our assessment of the impact of COVID-19 on HTA processes.
Access via compassionate or emergency use exemptions
Finally, governments are turning to compassionate/expanded use or other emergency authorization programs for early patient access to select investigational medicines outside of clinical trials prior to approval. These programs allow for access to unapproved medicines to selected patient populations on compassionate or emergency grounds, in this instance the urgency of the pandemic and lack of available proven alternatives. Patient costs are most often covered by a combination of public and private payers or manufacturers themselves.
In the most common illustration of this trend, Gilead’s remdesivir has seen wide early adoption through these programs. In line with regulators around the world, the U.K.’s MHRA offered a positive recommendation through the Early Access to Medicines Scheme (EAMS) that will see the drug see early access among select populations in the most need.
This was followed by the EMA’s human medicines committee (CHMP) recommending compassionate use beyond patients on mechanical ventilation in May 2020. In the US, the drug received Emergency Use Authorization in May 2020, following earlier usage through expanded and compassionate use programs. Notably, in October 2020, the FDA granted remdesivir proper approval for use in select hospitalized adult and pediatric COVID-19 patients, the agency’s first such approval for a COVID-19 treatment. It remains to be seen to what extent other markets may follow the U.S. regulator as mixed clinical data has seen divided opinion among regulators. In any case, for markets lacking formal approval, Gilead has commonly looked to transition to wider expanded use programs over more individualized compassionate use in order to ensure adequate supply.
Since the advent of the novel coronavirus, a few select drugs have received authorization in this manner. The table below provides a representative illustration of compassionate use approvals for COVID-19, drawn from Italy’s AIFA:
Drugs Approved for COVID-19 Compassionate Use, Italy, June 2020
|Manufacturer ||Drug ||Compassionate Use Program |
|Novartis ||Ruxolitinib ||Treatment of severe / very severe lung disease associated with COVID-19 |
|Novartis ||Canakinumab ||Treatment of cytokine release syndrome (CRS) in patients with COVID-19-induced pneumonia |
|Gilead ||Remdesivir ||Treatment of SARS-CoV2 (CoV) Infection |
|APEPTICO Forschung und Entwicklung GmbH ||Solnatide ||Treatment of pulmonary permeability edema in patients with COVID-19 with acute pulmonary insufficiency |
|Bausch Health ||Ribavirin for inhalation solution ||Treatment of hospitalized adult patients with respiratory distress due to COVID-19. |
Source: Decision Resources Group. https://www.aifa.gov.it/en/programmi-di-uso-compassionevole-covid-19 (accessed October, 2020).