Meds Pipeline Monitor 2021

Executive Summary

Meds Pipeline Monitor (MPM) is a horizon scanning report that features a selection of new medicines in the late stages of clinical evaluation that may have a significant impact on future clinical practice and drug spending in Canada.

Medicines in Phase III clinical trials or pre-registration are considered as candidates if they have the potential to address an unmet therapeutic need, offer a novel mechanism of action or therapeutic benefit over existing therapies, or treat a serious condition. The final selection features medicines that treat a broad range of therapeutic areas. In addition to identifying new medicines for inclusion in the list, medicines featured in the previous edition of the MPM are also reviewed to report on changes to their status in the pipeline. A section focused on Canada highlights potentially significant medicines currently under review by Health Canada.

This edition of the report also includes a section on COVID-19, which provides an overview of medicines undergoing Phase I, II, and III clinical trials or in pre-registration for the treatment and prevention of the novel coronavirus disease.

The report collects data from two main sources: GlobalData’s Healthcare database is used to identify medicines currently undergoing clinical evaluation, while Health Canada’s Drug and Health Product Submissions Under Review list provides information on new medicines under review in Canada.

Together with its companion publication Meds Entry Watch, this report series monitors the continuum of new and emerging medicines in Canada and internationally, providing key information to decision makers, researchers, patients, and clinicians, among other stakeholders.

List of Terms

For the purpose of this report, the following terms and associated definitions apply.

Clinical efficacy

The maximum response achievable from a medicine in research settings and the capacity for sufficient therapeutic effect in clinical settings.^{Footnote i}

Gene therapy

A technique for the treatment of genetic disease in which a gene that is absent or defective is replaced by a healthy gene, as defined by Health Canada.^{Footnote ii}

Market authorization

The process of approval for a medicine to be marketed in a given country. In Canada, market approval is granted following a substantive scientific evaluation of a product's safety, efficacy, and quality, as required by the Food and Drugs Act and Regulations.^{Footnote iii}

Medicinal ingredient

A chemical or biological substance responsible for the claimed pharmacologic effect of a drug product. Sometimes referred to as a molecule, active substance, or active ingredient.^{Footnote iv}

Medicine

A broad term encompassing both the final drug product and medicinal ingredient(s); this encompasses chemically manufactured active substances and biologics, including gene therapies. Medicines are reported at the medicinal ingredient level and can refer to a single ingredient or a unique combination of ingredients.

Medicine pipeline

A set of new medicine candidates under active research and development by biotechnology and pharmaceutical companies.

New medicine

A medicinal ingredient that has not previously received market authorization by a regulator.^{Footnote iv}

Orphan medicine

A medicine used to treat a rare disease. For the purposes of this study, orphan medicines are defined as having an orphan designation granted by the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA) for the relevant indication.

Introduction

This 11th edition of the Meds Pipeline Monitor (MPM) features a selection of medicines in Phase III clinical trials or pre-registration in 2021 that have the potential to significantly impact clinical practice and drug spending in Canada.

The methodology, which is detailed in the next section, uses a specific set of criteria to identify a list of pipeline candidates from the GlobalData Healthcare database, as well as a list of candidates currently under review in Canada from Health Canada’s Drug and Health Product Submissions Under Review (SUR) lists. Medicines reported in the previous edition are also reviewed in this report, including those that continue to qualify for the list of candidates as well as those that have since received market authorization. Likewise, the new medicines featured in this report will be monitored in future editions of the MPM to identify candidates that successfully enter the market.

To provide context for the selection of medicines, the MPM includes a snapshot of the entire pipeline, with an emphasis on the therapeutic breakdown of each phase of clinical evaluation. This edition of the report also highlights select vaccines and other medicines undergoing evaluation for the treatment and prevention of COVID-19, in global markets as well as in Canada. The medicines assessed for this portion of the analysis include new therapies as well as previously marketed treatments that have been repurposed.

Meds Pipeline Monitor is a companion publication to Meds Entry Watch, which analyzes the market dynamics of newly approved medicines in Canada and internationally. Together, these two PMPRB reports monitor the market continuum of late-stage pipeline medicines and new approvals, providing decision makers, researchers, patients, clinicians, and other stakeholders with information on the emerging medicines and evolving cost pressures.

Methodology

Meds Pipeline Monitor

The MPM focuses on new medicines in Phase III clinical trials or pre-registration in Canada, the United States, and Europe. Pipeline medicines are selected for inclusion using a two-stage process (Figure 1). The initial screening stage selects medicines in the late phases of clinical evaluation, while the analytic review stage involves a more rigorous appraisal of each potential candidate to identify medicines that may have a significant clinical and budgetary impact. The second stage considers a specific set of criteria, in addition to the results of a thorough review of clinical evidence and scientific literature.

This methodology is reviewed annually and refined as required.

Figure 1. Selection process for medicines featured in the Meds Pipeline Monitor

Figure description

This is a flowchart describing the process used to select the listed medicines. The chart consists of two steps:

1. Initial Screening

This step begins with all medicines in Phase III clinical trials or pre-registration with the US Food and Drug Administration. Of these medicines, the next step includes only those with expected clinical trial end dates within three years of the analysis and drug geography including Canada, the US, and Europe. To qualify for the drug geography, a medicine must have Phase III clinical trials in Canada, the US, and/or geographic Europe (excluding Russia and Turkey).

2. Analytic Review

The analytic review step of the process is divided into two parts: one path for new medicines and the other for gene therapies.

New medicines must meet at least one of the following requirements to be included in the list:

• Demonstrates improved safety and efficacy
• Novel mechanism and/or first-in-class, with the addition of one or more of Breakthrough, Fast Track, and Priority Review designations

Gene therapies must demonstrate clinical effectiveness with an acceptable safety profile to be included in the list.

Stage 1. Initial screening

GlobalData’s Healthcare database is used to identify a list of medicines undergoing Phase III clinical trials or in pre-registration. These medicines serve as the basis for the initial screening stage.

The drug geography, defined as the geographical region or country in which the medicine is either marketed or in pipeline development, is restricted to Canada and other countries with similar regulatory and approval processes: the US and geographic Europe (excluding Russia and Turkey). Only new medicinal ingredients that have adequate data that supports increased efficacy and safety from clinical trials are considered as candidates for inclusion.

Medicines approved or sold in Canada, the US, or Europe for any other indication or in any other strength or formulation are excluded during the selection process, as are medicines whose clinical trials are inactive, suspended, withdrawn, or terminated.

The selection process groups pipeline candidates into two categories: (a) new medicines and (b) new gene therapies. As illustrated in Figure 1, the initial screening process for both groups is the same, but the analytic review stage is slightly different, as the available data for gene therapies is limited.

Stage 2: Analytic screening

Selection criteria

Following the initial screening, the second stage of the process considers a number of selection criteria to determine the final list of pipeline candidates. These criteria are detailed in Table 1.

Gene therapies are selected using a broader approach, as the clinical evidence available for this group is relatively limited. A gene therapy is retained on the list if the preliminary (or completed) results from Phase III trials suggest that there is evidence of clinical effectiveness with an acceptable safety profile.

Table 1. Selection criteria for the Meds Pipeline Monitor

Selection criteria
Improved safety and efficacy shown in clinical trials: a medicine that demonstrates increased safety, new outcome measures, or increased life expectancy or quality of life
Novel mechanism / First-in-class: a medicine that uses a new mechanism of biochemical interaction to produce a medical effect, or a medicine that is the first in its therapeutic class In addition, the medicine must fall into one or more of the three following FDA designations for expedited development and review: Breakthrough – medicines intended to treat a serious condition and for which preliminary clinical evidence indicates that they may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s) Fast Track – medicines used to treat serious conditions and fill an unmet medical need Priority Review – medicines that would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications Gene therapy: a technique for the treatment of genetic disease in which a gene that is absent or defective is replaced by a healthy gene

Additional descriptive information

A profile of each successful pipeline candidate is provided, including a brief outline of the indication and mechanism of action, as well as a summary of the applicable published outcomes from clinical trials. Specific attributes that may influence the potential uptake or cost of each medicine are also identified. Table 2 provides a detailed description of these key attributes.

Table 2. Key attributes of new medicines selected for the Meds Pipeline Monitor

Attribute	Relevance	Data sources
Phase III clinical trials in Canada	Medicines tested in Canada are likely to be of interest to Canadians	GlobalData Healthcare; Health Canada Clinical Trials Database; Health Canada Drug and Health Product Submissions Under Review; National Institutes of Health (NIH) Clinical Trial Registry
Rare or orphan designation	Medicines used to treat rare diseases or conditions that generally have high treatment costs and may result in substantial spending	GlobalData Healthcare
Biologic medicine	These complex molecules produced by living organisms are expected to have high costs, resulting in substantial spending
Add-on therapy	Medicines designed to be used in conjunction with existing medicines may increase the treatment cost and contribute to higher spending

The profile also provides details of potential cost implications, if available, which includes the forecasted global revenues reported by GlobalData.

The indications and therapeutic areas of the featured medicines correspond to their Phase III clinical trial or pre-registration stage. A single clinical trial may assess multiple indications within the same therapeutic area. These medicines may also have additional indications at various phases of clinical evaluation that are not mentioned in this report. The scientific description and key attributes provided are focused on the specified indication(s) for the selected medicines.

Medicines reported for a given year are reassessed for each following edition of the MPM. They may be retained on the MPM list if they continue to meet the selection criteria, or they may be removed if they have been granted market authorization through the US FDA, the EMA, or Health Canada. Medicines for which clinical trials have been discontinued or for which the selection criteria is no longer met are not reported in subsequent editions.

Limitations

This analysis captures a snapshot of the pipeline over a specific time period. Although it is assumed to be representative of the composition of medicines over the entire year, the pipeline is fairly dynamic and the share of medicines in any particular therapeutic area will vary.

This assessment is restricted to medicines under development for market in Canada and other countries with similar regulatory and approval processes: the US and Europe (excluding Russia and Turkey). Medicines that have not yet received market authorization in these countries were considered as potential pipeline candidates, even if they have been approved elsewhere in the world.

Some of the selected medicines may be undergoing clinical trials for additional indications; this analysis only reports on indications in the late stages of development—that is, in Phase III clinical trials or pre-registration with the US FDA—that satisfy the selection criteria set out in the methodology.

For each selected pipeline medicine, the primary manufacturer(s) and trade name, if available, are given along with the indication. In some cases, additional manufacturers, including subsidiaries, may also be involved in the development of the medicine with the primary companies, or other manufacturers may be developing the same medicine for other indications.

Although this report attempts to identify the most important pipeline medicines, the selection is not exhaustive and some medicines that are not included in this selection may have a significant impact on future clinical practice and drug spending in Canada.

Unless otherwise specified, the featured lists capture the composition of the pipeline as of September 2021 and are validated as of March 2022. Due to the unpredictability and fast-moving nature of pipeline medicines entering the market, some of the medicines listed in this edition may have been approved or marketed in Canada, the US, or Europe following this date. Pipeline medicines that have not been included in this report due to the timing of the selection may presently meet the selection criteria; these, along with the rest of the drug pipeline, will be considered for the next edition of the report.

Snapshot of the 2021 Pipeline

Pharmaceutical innovation is transforming the development and application of medical treatments worldwide. Nearly 8,500 new medicines were in clinical evaluation or pre-registration in 2021.

Figure 2 provides a snapshot of the pipeline in 2021, including the number of new medicinal ingredients in each phase of clinical evaluation. Of the 8,460 new medicines, 1,145 (13%) were in Phase III clinical trials or pre-registration.

Figure 2. Number of new medicines in each phase of clinical evaluation, 2021

Figure description

This stacked bar graph illustrates the number of new medicinal ingredients in the pipeline by their highest phase of clinical evaluation in September 2021. There were 8,460 new medicines across the pipeline, of which 3,582 were in Phase I clinical trials, 3,733 were in Phase II, 991 were in Phase III, and 154 were in pre-registration.

Data source: GlobalData Healthcare database (accessed September 2021); IQVIA MIDAS© Database.

Figure 3 illustrates the distribution of new medicines by therapeutic area from Phase I through pre-registration. Although the findings show that pipeline medicines represented a wide range of therapeutic areas in 2021, cancer treatments dominated the therapeutic mix across the pipeline, accounting for over one third (35%) of medicines in all phases of clinical evaluation. Other important pipeline therapies include those for infectious diseases (such as COVID-19) and central nervous system therapies.

Figure 3. Therapeutic class distribution of pipeline medicines by phase of clinical evaluation, 2021

Figure description

A stacked bar graph shows the distribution of new medicines in the pipeline by their therapeutic area. The distribution is given as a percentage of all new medicines in each phase of development, as well as a total share for all phases.

Therapeutic area	Phase I	Phase II	Phase III	Pre-registration	All phases
Oncology	39%	35%	21%	23%	35%
Infectious Disease	13%	13%	21%	17%	14%
Central Nervous System	11%	10%	9%	11%	10%
Metabolic Disorders	5%	5%	6%	8%	6%
Immunology	5%	5%	4%	4%	5%
Cardiovascular	4%	4%	5%	6%	4%
Gastrointestinal	5%	5%	4%	1%	5%
Respiratory	3%	3%	4%	3%	3%
Ophthalmology	2%	3%	6%	6%	3%
Dermatology	2%	3%	3%	6%	3%
Genetic Disorders	1%	3%	3%	4%	2%
Other	9%	10%	13%	13%	10%

Data source: GlobalData Healthcare database (accessed September 2021). 

Orphan medicines, as designated by the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA), accounted for a notable proportion of the total medicine pipeline in 2021. Figure 4 provides the shares of orphan and other medicines in the pipeline from Phase I to pre-registration. Orphan medicines made up a greater share of medicines in the later stages of clinical evaluation, accounting for 8% of pipeline medicines in Phase I clinical trials and 30% of those in pre-registration.

Figure 4. Share of orphan medicines in the pipeline by highest phase of clinical evaluation, 2021

Figure description

A stacked bar graph gives the share of orphan medicines in each phase of development (Phase I, Phase II, Phase III, and pre-registration) as of September 2021. Orphan medicines were defined as pipeline medicines that have been granted an orphan designation by the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA). 

	Phase I	Phase II	Phase III	Pre-registration
Orphan	7%	21%	36%	30%
Non-orphan	93%	79%	64%	70%
Total number of medicines	3,582	3,733	991	154

Note: Includes all pipeline medicines with a highest development stage of Phase I to pre-registration that are being developed for market in Canada, the United States, or geographic Europe (excluding Russia and Turkey). Orphan medicines were defined as pipeline medicines that have been granted an orphan designation by the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA).
Data source: GlobalData Healthcare database (accessed September 2021).

Meds Pipeline Monitor 2021

The following tables list the selection of new pipeline medicines in 2021, those retained from earlier editions of the Meds Pipeline Monitor, as well as medicines featured in previous editions that have since gained market authorization. These pipeline medicines will continue to be monitored in future editions of this report.

Applying the screening criteria described in the Methodology section, 31 of the 1,145 pipeline medicines in late stages of clinical evaluation were selected for inclusion in the 2021 new medicines list (Table 4).

Of the new medicines featured in the 2020 report, 10 were retained as recent evidence continues to support promising clinical benefit and satisfies the selection criteria (Table 5). Nine of the 2020 pipeline medicines had received market authorization in the US, Europe, or Canada as of December 2021 (Table 6), while eight were removed from the list as their clinical trials were discontinued or they no longer fulfill the selection criteria.

Emerging COVID-19 Therapies

This section of the Meds Pipeline Monitor includes an overview of new and existing pipeline medicines that are under evaluation for indications related to the prevention and treatment of COVID-19. An analysis of global markets provides information on COVID-19 medicines in all phases of clinical trials and pre-registration.

Global markets

There have been significant strides in the COVID-19 drug pipeline worldwide. However, published information to confirm safety and efficacy of the various treatments for COVID-19 is continuously evolving.

In addition to the wide variety of vaccines under development, many novel and repurposed medicines are currently being evaluated in clinical trials for their potential benefits in the treatment of COVID-19. These include antivirals, monoclonal antibodies, mesenchymal stem cells, convalescent plasma, and cytokine adsorbers.²⁰²

A breakdown of COVID-19 pipeline vaccines and treatments by phase of clinical evaluation is given in Figure 5. For this snapshot, data was extracted for medicines indicated for the treatment of COVID-19 with a development stage of Phase I, II, III, or pre-registration. These medicines are presented in three categories: vaccines, which are used to prevent infection of the novel coronavirus; COVID-19 treatments (new), which are new medicines used for the prevention or reduction of some of the complications associated with COVID-19 (e.g., pneumonia or respiratory complications and hyperinflammation); and COVID-19 treatments (existing), which are previously marketed medicines that have been repurposed to treat COVID-19 or its symptoms.

Brief Insights

The pipeline for COVID-19 medicines is growing rapidly, with clinical investigations of novel and existing drugs:

• Current approaches to COVID-19 therapies generally fall into two categories: antivirals, which prevent the virus from multiplying; and immune modulators, which help the immune system to fight the virus.
• In 2020, there were 53 trials for COVID-19 categorized as pivotal/registration studies, whereas in 2021, this decreased to 41 trials. A pivotal or registration clinical trial is a trial seeking to prove the efficacy of new therapeutics and vaccines.
• As new strains of COVID-19, such as the Omicron variant, continue to raise the number of cases around the world, focus is placed on the efficacy of booster vaccinations.
• The 2020 pipeline had more ongoing and completed clinical trials while 2021 had a greater number of planned clinical trials.
• Following the approvals of the four most common vaccines used in Canada and the US—Pfizer/BioNTech’s Comirnaty, Moderna’s Spikevax, AstraZeneca’s Vaxzevria, and the Janssen vaccine—sponsors continued to focus testing on different age groups in 2021 (e.g., authorization for Pfizer/BioNTech’s Comirnaty vaccine for children aged 5 to 11 years).
• Vaccine manufactures have estimated that by the end of 2021, 12 billion COVID-19 vaccine doses will have been produced globally.

Source: Pharma COVID-19 Bulletin, GlobalData (December 30, 2021); Health Canada (December 2021).

Figure 5 illustrates the breakdown of vaccines and new or existing treatments for COVID-19 by highest development stage. The majority of treatments undergoing clinical evaluation are new medicines with an increasing proportion of existing medicines in the later stages of clinical development.

Figure 5. Number of medicines indicated for the prevention and treatment of COVID-19 by stage of development, 2021

Figure description

This stacked bar graph illustrates the number of COVID-19 treatments and vaccine in the pipeline by their highest phase of clinical evaluation in September 2021. Totals are given for vaccines and COVID-19 treatments, with separate totals for new and existing medicines.

	Phase I	Phase II	Phase III	Pre-registration
Vaccine	33	55	43	5
COVID-19 treatment, new medicine	74	183	106	4
COVID-19 treatment, existing medicine	12	42	73	33
Total	119	280	222	42

Figure 6 breaks down the COVID-19 vaccines by mechanism of action and highest development phase.^{Footnote vii} Vaccines are categorized into various vaccine types based on their mechanism of action; for example, while live attenuated vaccines target the whole virus, subunit and recombinant vaccines target one specific part of the virus.

Figure 6. Distribution of COVID-19 vaccines by mechanism of action and phase of clinical evaluation, 2021

Figure description

A stacked bar graph gives the distribution of COVID-19 vaccines in each phase of clinical evaluation by vaccine type, as of September 2021.

	Subunit vaccine	Inactivated vaccine	mRNA vaccine	Recombinant vector vaccine	DNA vaccine	Conjugate vaccine	Live attenuated vaccine	Vaccine (prophylactic)	Total
Phase I	10	3	4	6	6	–	1	1	31
Phase II	21	6	11	7	5	–	–	5	55
Phase III	16	13	4	5	3	1	1	–	43
Pre-registration	–	1	2	2	–	–	–	–	5

Canada

As COVID-19 disease activity continues to accelerate, Canada’s health authorities have committed to the immunization response plan published in December 2020. Key elements of Canada’s immunization plan include securing sufficient supply; regulatory authorization for safety and efficacy; managing allocation and distribution of vaccines efficiently and securely; administering vaccines rapidly and equitably; and continuing to monitor vaccine safety, effectiveness and coverage. As of December 2021, the eligibility regulations for booster doses have permitted individuals 18 years and older residing in many Canadian provinces to receive a third shot.

Table 8 gives the number of medicines approved by Health Canada for the prevention and treatment of COVID-19, while Table 9 gives the number of COVID-19 medicines currently under review. As the COVID-19 pandemic remains a top priority in Canada, the current submissions undergoing Health Canada’s review process for COVID-19 are being reviewed under expedited approval timelines. This effort has been supported by the use of interim orders intended to put temporary regulations into place in order to make drugs available to address large-scale public health emergencies. For example, the Interim Order Respecting the Importation, Sale and Adversiting of Drugs for Use in Relation to COVID-19, approved on May 23, 2020, introduced an alternate pathway to facilitate clinical trials for potential COVID-19 drugs and medicinal devices, while upholding strong patient safety requirements and validity of trial data.

Table 8. COVID-19 treatment and vaccines approved by Health Canada, 2021

* The Interim Order Respecting the Importation, Sale and Adversiting of Drugs for Use in Relation to COVID-19, approved on May 23, 2020, introduced an alternate pathway to facilitate clinical trials for potential COVID-19 drugs and medicinal devices, while upholding strong patient safety requirements and validity of trial data.
Data source: Drug and vaccine authorizations for COVID-19: List of applications received, Health Canada (accessed February 2022):
https://www.canada.ca/en/health-canada/services/drugs-health-products/covid19-industry/drugs-vaccines-treatments/authorization/applications.html 

Table 9. COVID-19 treatment and vaccines under review by Health Canada, 2021

* The applicant has filed a new drug submission under the Food and Drug Regulations to transition this product from the interim order. The product continues to be approved for sale in Canada during this transition period.
Data source: Drug and vaccine authorizations for COVID-19: List of applications received, Health Canada (accessed February 2022): https://www.canada.ca/en/health-canada/services/drugs-health-products/covid19-industry/drugs-vaccines-treatments/authorization/applications.html 

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