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An FDA advisory committee has rejected Pfizer and BioNTech’s plan to deliver COVID-19 booster shots for individuals 16 years of age and older, saying they need more data.

The panel did, however, vote to recommend the booster shots to adults over 65 years of age and those at high risk of severe COVID-19, according to 

gton Post. The booster shots would be given at least 6 months after the primary doses.

The committee vote comes days before the 

 to begin releasing booster shots to the general public. Although the panel’s decision is nonbinding, it could strongly influence the final FDA decision expected soon.

Pfizer and BioNTech initiated rolling submission of the Biologics License Application for booster doses in individuals 16 years of age and older in late August, citing phase 3 data that a third dose induces significant neutralizing antibody titers. According to a 

, SARS-CoV-2 neutralizing titers against the wild-type strain 1 month after the booster dose was administered were 3.3 times higher than the titers found 1 month after the second dose.

These findings are from a phase 3 trial with 306 participants between the 18 and 55 years of age who received a third dose of the vaccine between 4.8 months and 8 months after the primary 2-dose regimen. Researchers had a median follow-up time of 2.6 months post-booster and the neutralizing titers after the third dose met a prespecified 1.5-fold non-inferiority criterion of success and were statistically superior. Notably, 99.5% of participants had a 4-fold response following the third dose, compared to 98% of participants after the second dose.

The trial also had a favorable reactogenicity profile within 7 days after the booster dose, with mostly mild to moderate reactions. The most common adverse events included injection site pain, fatigue, headache, muscle and joint pain, and chills. The frequency of reactogenicity was similar to or better than after dose 2 of the primary series.

Despite these findings, experts expected the advisory panel decision to be controversial. According to 

, 2 senior FDA officials involved in the meeting have said they are not convinced that every American who has received the Pfizer vaccine needs extra doses right now.1

In a paper published earlier this week in 

, experts from the FDA and World Health Organization said that according to current data, COVID-19 vaccines continue to be effective in protecting against severe SARS-CoV-2, including disease caused by the Delta variant. They added, however, that boosters could be appropriate for some individuals who may not have received adequate protection from the primary series, such as immunocompromised individuals.2

“Although the benefits of primary COVID-19 vaccination clearly outweigh the risks, there could be risks if boosters are widely introduced too soon, or too frequently, especially with vaccines that can have immune-mediated side effects,” the authors wrote. “If unnecessary boosting causes significant adverse reactions, there could be implications for vaccine acceptance that go beyond COVID-19 vaccines.”2

, some FDA committee members argued that booster doses should be limited to certain groups, such as individuals 60 years of age and older who are at a greater risk of severe disease. Some members specifically expressed concerns about the risk of myocarditis in younger people and said that more research is needed.

1. FDA panel rejects plan to administer Pfizer’s Covid booster doses to general public. CNBC; September 17, 2021. Accessed September 17, 2021. 

https://www.cnbc.com/2021/09/17/fda-panel-begins-voting-on-pfizers-covid-booster-doses-rejecting-shots-for-general-public.html

2. Krause P, Fleming T, Peto R, et al. Considerations in boosting COVID-19 vaccine immune responses. 

September 13, 2021. Accessed September 17, 2021. 

https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(21)02046-8/fulltext

Articles

The panel did, however, vote to recommend the booster shots to adults over 65 years of age and those at high risk of severe COVID-19. 

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FDA Panel Votes Against Recommending Pfizer COVID-19 Booster Doses for General Public

showed that a woman with immune thrombocytopenic purpura was resistant to treatment of high-dose steroids, intravenous immunoglobulin (IVIG), and rituximab, following receiving the second dose of Pfizer’s COVID-19 immunization but eventually responded to a thrombopoietin-stimulating agent.

As part of a double-blind, placebo-controlled, randomized trial of the Pfizer-BioNTech vaccine with 43,548 individuals, just 4 adverse events have been reported. None of the adverse reactions indicated immune thrombocytopenic purpura, leading investigators to think it could be seen after immunization with other vaccines.

Immune thrombocytopenic purpura had been reported to follow several vaccinations, but no causal relationship had been determined.

As part of a medical study, the 67-year-old Hispanic woman was admitted to a hospital following her second dose of the Pfizer vaccine, and all her vaccinations were up to date.

She developed mild rashes on her chest and legs about 2 weeks after her first dose but denied any adverse effects. Two days following her second dose, she experienced bleeding of the gums when brushing her teeth, an episode of epistaxis, and that the rash had spread.

The woman then developed a headache on the right side, hemorrhagic lesions of the buccal mucosa and tongue and subconjunctival hemorrhage in her right eye.

Her platelet count was low, so she was given oral Prednisone and IVIG at 400 mg/kg a day, but her platelet count did not improve, and she was transfused with 1 unite of platelets.

She started on aminocaproic acid of 5 g to start and continued with 1 g twice daily. Her platelet levels rose slowly, and she received IVIG with a high-dose steroid for 3 more days.

She received 4 doses of Rituximab at a Hematology/Oncology clinic following her release from the hospital. She continued to be treated with a TPO agent, Eltrombopag, to help increase and maintain her platelet levels.

Vaccine-induced thrombocytopenia, investigators noted, seem to occur 4 to 16 days after exposure to the vaccine, However, they can become present earlier.

There had been several reports of immune thrombocytopenic with COVID-19 vaccinations, but they have not been widely reported following the Pfizer vaccine, so there are not enough cases to attribute to safety concerns.

Jasaraj RB, Shrestha DB, Gaire S, Kassem M. Immune thrombocytopenic purpura following Pfizer-BioNTech COVID-19 vaccine in an elderly female. 

. 2021;(8):e16871. doi:10.7759/cureus.16871

Study results showed that an individual with the blood disorder did not respond to treatment, including intravenous immunoglobulin, following their second dose of the Pfizer vaccination.

IVIG

Immune Thrombocytopenic Purpura Is Resistant to IVIG Following COVID-19 Vaccine

Research is ongoing for the treatment of amyloidosis in multiple myeloma to prove efficacy in decreasing amyloids, according to a presentation at the Society of Hematologic Oncology (SOHO) 2021 Annual Meeting.

Suzanne Lentzsch, MD, PhD, professor of medicine and director of the Multiple Myeloma and Amyloidosis Program at the College of Physicians and Surgeons of Columbia University and at New York Presbyterian Hospital in New York, gave an overview of treating relapsed disease and how a frequent assessment of response as a health care provider is important.

“I usually see my patients every 3 months and make sure they are in a complete remission of at least complete response (CR) or very good partial response (VGPR) with organ response,” Lentzsch said. “And if the patients lose VGPR or stay in VGPR with organ progression, then we consider restart or change in treatments.”

Lentzsch discussed FISH Cytogenetics in amyloid light chain (AL) amyloidosis prognosis, such as translocation, high risk aberrations, and gain of 1q21 and their impact on patients who are treated with bortezomib.

Although she mentioned that there are not many trials in this area of multiple myeloma, some have shown significant data. For example, the phase 3 TOURMALINE-AL1 of ixazomib-dexamethasone versus physician’s choice in relapsed/refractory AL did not meet the primary outcomes, but it did show the ixazomib-dexamethasone hematologic CR rate was higher, as it was the first combination to get approved by the FDA in this area, according to Lentzsch.

Treatment of AL amyloidosis occurs in a hematologic response (monoclonal plasma cells and immunoglobin free light chains) and an organ response (amyloid fibrils and tissue damage), which is being explored in recent studies. However, Lentzsch mentioned how lenalidomide, which is part of the previous anti-amyloid antibodies, has a bad reputation because it can cause cognitive failure in patients. Recent studies have instead used the monoclonal antibody ch11-1F4 mAb, for example, which established a well-tolerated response in the overall median survival time.

Lentzsch reiterated that ixazomib plus dexamethasone improves CR and delays organ deterioration, but the combination failed to achieve the primary endpoint of overall response rate, which makes other options superior, such as autologous stem cell transplant (ASCT). As for other treatments, bendamustine was well-tolerated with 30% VGPR, in addition to venetoclax alone or in different combinations.

“When using Revlimid, do it judiciously, as it can cause high cardiotoxicity,” Lentzsch said. “Anti-amyloid immunotherapy is a tantalizing matter but remains to be proven in decreasing amyloids.”

Lentzch S. Salvage Therapy for Amyloidosis: Agents and Anti-Fibril Therapy. Presented at: the Society of Hematologic Oncology (SOHO) 2021 Annual Meeting; virtual. September 9, 2021. Accessed September 17, 2021.

A presentation at the Society of Hematologic Oncology 2021 Annual Meeting reviewed the treatment of amyloidosis in multiple myeloma.

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Hematology

Oncology

Multiple Myeloma

Salvage Therapy for Amyloidosis in Multiple Myeloma: Agents and Anti-Fibril Therapy

Shereen Stutz and Mark Alwardt, of McKesson, said the rise in oral oncolytics during the COVID-19 pandemic allowed pharmacists to be more innovative and to become an even more integrated part of the oncology care team.

In the interview, Stutz and Alwardt discussed the necessity for close monitoring when patients receive oral oncolytics, potential concerns about adherence, and how telemedicine enabled this care and monitoring. Stutz and Alwardt also co-authored an article on the subject, which will be featured in the October issue of 

Videos

Shereen Stutz and Mark Alwardt, of McKesson, said the rise in oral oncolytics during the COVID-19 pandemic allowed pharmacists to be more innovative and to become an even more integrated part of the oncology care team. 



COVID-19 Pandemic Provides Opportunities to Innovate With Oral Oncolytics, Medically Integrated Dispensing

The results of a review by the Murdoch Children’s Research Institute (MCRI) published in the 

 showed that children who have pre-existing conditions are more likely to experience severe COVID-19 symptoms than those without.

Investigators studied the effects of COVID-19 on pediatric individuals, because of a lack of research on the subject.

“Current studies lack a clear case definition and age-related data, have variable follow-up times, and rely on self- or parent-reported symptoms without lab confirmation,” Nigel Curtis, professor at MCRI, said in a statement. “Another significant problem is that many studies have low response rates, meaning they might overestimate the risk of long COVID.”

They found that pediatric individuals with pre-existing conditions are at greater risk of severe COVID-19. Pediatric individuals with pre-existing conditions in the review reported severe symptoms at 5.1% compared with 0.2% without pre-existing conditions.

The review analyzed 14 international studies with a combined 19,426 pediatric individuals who reported persistent symptoms following COVID-19 infections.

The most common symptoms reported after 4 to 12 weeks were abdominal pain, difficulty concentrating, fatigue, and headaches, investigators found.

“The low risk posed by acute disease means that one of the key benefits of COVID vaccination of children and adolescents might be to protect them from long COVID,” Curtis, professor of pediatric infectious disease at the University of Melbourne and head of infectious diseases at The Royal Children’s Hospital, said in the statement. “An accurate determination of the risk of long COVID in this age group is therefore crucial in the debate about the risks and benefits of vaccination.”

The review also confirmed research gaps around the Delta variant in pediatrics.

Australia has not reported any COVID-19 deaths in pediatrics younger than aged 10 years and just 1 death of an adolescent.

Long COVID symptoms in children rarely persist beyond 12 weeks. EurekAlert. News release. September 16, 2021. Accessed September 17, 2021. 

https://www.eurekalert.org/news-releases/928599

The results of a review by Murdoch Children’s Research Institute finds that pediatric patients who are vaccinated get protection from the long-term effects of the disease.

Pediatrics

Immunization

Children With Pre-Existing Conditions Experience More Severe COVID-19

Investigators at the IMT School for Advanced Studies Lucca proposed new criteria for establishing the priority administration of COVID-19 vaccines, because of the results of a new study surrounding the COVID-19 crisis that Italy faced.

“We conclude that lockdowns should be avoided in the future by prioritizing the vaccination of essential workers and people more exposed to unemployment risk among the healthy and active population,” Massimo Riccaboni

 professor of economics at the IMT School, said in a statement.

Investigators contend that vaccine campaigns should focus on beneficiaries of wage guarantees, employees who face a high unemployment risk, and essential workers, because it would help individuals return to work while also helping fix the economy.

Results of the study in Italy showed that, with a lockdown, there was a 1% drop in mobility, implying a 0.6% drop in excess deaths the following month.

However, a 1% drop in mobility also corresponded to a 10% increase in the Wage Guarantee Fund the next month.

Investigators also said that a COVID-19 certificate, allowing European Union individuals to return to school and travel, could also be required for workers soon.

They said that delivering vaccines to workers is getting more relevant, because of the unemployment risk this imposes, the investigators said.

Investigators will further research how the mobility patterns may influence employment risk in other countries and across different sectors of the economy.

COVID-19, who to vaccinate first among workers. EurekAlert. Press release. September 17, 2021. Accessed September 17, 2021. 

https://www.eurekalert.org/news-releases/928643

Investigators at the IMT School for Advanced Studies say that future rollouts should focus on workers, which would help avoid another lockdown.

Infectious Disease

Economy May Benefit From Making Employees a Vaccine Priority

Rabies is an acute, fatal, progressive viral disease that claims nearly 60,000 lives a year.

Most deaths occur in Africa and Asia, with approximately 80% in rural areas. Human rabies is rare in the United States because of animal control programs and veterinary vaccination.

 Any mammal can be infected by the rabies virus and may become a carrier, but the most common wild reservoirs include bats, foxes, raccoons, and skunks. The most common domestic reservoirs include cats, cattle, and dogs.

 Contact with infected bats is the leading cause of human rabies in the United States. Worldwide, contact with infected dogs is the leading cause of human rabies death.

The rabies virus is transmitted via direct contact with infectious tissue or fluids of infected mammals. This includes nervous tissue, saliva, respiratory tract fluids, and tears. Direct contact implies broken skin or mucous membranes of the eyes, mouth, or nose.

 The incubation period is between 20 and 60 days but can span several years before the virus enters the central nervous system. The disease manifests as furious rabies, causing hyperactivity, hydrophobia, and sometimes aerophobia, or as paralytic rabies, causing paralysis starting at the site of transmission. Once clinical symptoms appear, survival is rare and associated with permanent disability and severe brain damage.

 Fortunately, vaccinations have nearly eliminated death from rabies.

Recommendations for Prophylaxis and Vaccinations

Per CDC guidelines, the United States population at large does not need vaccinations. Travelers to other countries, particularly Africa and Asia, should visit the CDC Poxvirus and Rabies Branch website (https://www.cdc.gov/rabies/resources/countries-risk.html) to evaluate their risk of rabies exposure and assess their need for postexposure prophylaxis (PEP).

 PEP consists of 1 dose of rabies immune globin and 4 doses of rabies vaccines on days 0,3, 7, and 14.

Travelers should consider preexposure prophylaxis (PrEP) vaccination if planned activities include encounters with domestic or wild animals, if staying longer than a month in an area where dog rabies is common, or if visiting remote areas where medical care may be delayed.

 The CDC categorizes risk into 3 categories: continuous, frequent, and infrequent. The degree of risk is determined by the extent of contact with the rabies virus, such as working in a research lab or in vaccine production (continuous risk), working with animals in endemic rabies areas (frequent risk), traveling to an endemic area, or working with animals in areas where rabies is not endemic (infrequent risk).

In addition to these categories, people who are more likely to encounter rabid animals include adventure travelers, campers, cave explorers, and visitors to rural areas. Consider immunizing children because they may not report bites or may receive more severe bites.

PrEP vaccination was recently updated for immunocompetent individuals 18 years and older to 2 doses on days 0 and 7 rather than 3 doses on days 0, 7, and 21 or 28. The Advisory Committee on Immunization Practices further recommended an intramuscular booster dose of rabies vaccines in lieu of a titer check for immunocompetent individuals 18 years and older with elevated and sustained risk of rabies exposures. This booster dose should be administered no sooner than day 21 and no later than 3 years after the 2-dose PrEP vaccinations.

Those considering PrEP vaccination do not need to delay for minor illness, such as colds. 

Consider delaying vaccination for those with AIDS/HIV, allergic reactions to the rabies vaccine in the past, cancer, or severe allergies, or individuals who are breastfeeding, pregnant, or taking immunocompromising medication. After rabies exposure, all patients should be vaccinated, regardless of any of the above conditions.

 Adverse effects of the vaccine are usually mild and may include dizziness, headache, muscle aches, pain, redness and swelling at the injection site, stomach pain, and upset stomach. Symptoms are self-limiting and resolve in a few days.

The same vaccine is used before and after exposure. The 2 rabies vaccines available in the United States are the human diploid cell vaccine (Imovax Rabies; Sanofi Pasteur SA) and the purified chick embryo cell vaccine (RabAvert; GlaxoSmithKline).

 Both are administered intramuscularly and contain inactivated rabies virus.

 In the event of a shortage or if one vaccine becomes unavailable, the vaccines are interchangeable. If a patient started a series with one vaccine, the other may be used to complete both a pre- and postexposure series.

PrEP is something to consider if traveling to areas with a high probability of encountering domestic or wild animals or if working in a high-risk field. PrEP vaccination does not eliminate the need for treatment after exposure, but it will simplify treatment and provide protection to those unable to seek higher-level care quickly. 

is a clinical pharmacist of infectious diseases at Olathe Health in Kansas City, Missouri.

is chief of hematology oncology pharmacy at Tripler Army Medical Center in Honolulu, Hawaii. 

1. Wallace RM, Petersen BW, Shlim DR. Travel-related infectious diseases - rabies. In: 

. Oxford University Press; 2019:169-392. Accessed August 13, 2021. 

https://wwwnc.cdc.gov/travel/yellowbook/2020/travel-related-infectious-diseases/rabies

2. Rabies and your pet. American Veterinary Medical Association. Accessed August 14, 2021. 

https://www.avma.org/resources/public-health/rabies-and-your-pet

3. What kind of animal did you come in contact with? CDC. June 11, 2019. Accessed August 13, 2021. https://www.cdc.gov/rabies/exposure/animals/index.html

4. Rabies. CDC. Updated September 25, 2020. Accessed August 13, 2021. 

5. Manning SE, Rupprecht CE, Fishbein D, et al; Advisory Committee on Immunization Practices Centers for Disease Control and Prevention (CDC). Human rabies prevention--United States, 2008: recommendations of the Advisory Committee on Immunization Practices. 

6. Rabies. World Health Organization. May 17, 2021. Accessed August 14, 2021. 

https://www.who.int/news-room/fact-sheets/detail/rabies

7. RabAvert. Prescribing information. GlaxoSmithKline; 2018. Accessed August 14, 2021. 

https://www.fda.gov/files/vaccines%2C%20blood%20%26%20biologics/published/Package-Insert---RabAvert.pdf

8. Vaccines by disease. US Department of Health & Human Services. Updated April 29, 2021. Accessed August 14, 2021. 

9. Preexposure vaccinations. CDC. Updated April 22, 2011. Accessed August 14, 2021. 

https://www.cdc.gov/rabies/specific_groups/travelers/pre-exposure_vaccinations.html

10. ACIP recommendations. CDC. Updated July 13, 2021. Accessed August 14, 2021. 

https://www.cdc.gov/vaccines/acip/recommendations.html

11. Imovax Rabies. Prescribing information. Sanofi Pasteur SA; 2019. Accessed August 14, 2021. 

PrEP simplifies management of the virus by eliminating the need for immune globulin and decreasing the number of doses needed.

Hit Me With Your Best Shot: Rabies Pre-exposure Vaccination

A low dose of the COVID-19 vaccine from Moderna elicits immunity for at least 6 months, according to researchers at the La Jolla Institute for Immunology. Notably, the researchers also found no indications that vaccinated people will need a booster shot.

Although the data found that the vaccine elicited strong CD4-positive T cells, CD8-positive T cells, and antibody responses for at least 6 months after vaccination in a clinical trial, the researchers said immune responses would likely last much longer. This strong immune memory lasted in all age groups tested, including individuals over 70 years of age who are especially vulnerable to COVID-19.

“This time point is critical because that is when true immune memory has formed,” said study co-leader Daniela Weiskopf, PhD, in the press release.

Investigators compared data from recovered COVID-19 patients to vaccine trial participants who received a 25-microgram dose of the Moderna vaccine during phase 1 clinical trials. This is a quarter of the 100-microgram dose given emergency use authorization by the FDA, and although the researchers do not know whether this small dose is as effective as the larger dose, their findings showed that the T cell and antibody response is still strong.

“We wanted to see if a quarter of the dose is able to induce any immune response,” said study first author Jose Mateus Trivino, PhD, in the press release. “We had the opportunity to receive the samples from the original Moderna/[National Institutes of Health] phase 1 trial participants who had received 2 25-microgram injections of the vaccine, 28 days apart.”

According to the press release, the researchers found that the vaccine induces an adaptive immune response to the SARS-CoV-2 spike protein, which is nearly identical to the immune system’s response to a natural SARS-CoV-2 infection. The researchers added, however, that the findings do not show that a lower dose of the Moderna vaccine provides the same protection as the standard dose.

“It would take a clinical trial to tell you how protective the lower dose is,” said study co-leader Shane Crotty, PhD, in the press release.

Interestingly, however, the research did demonstrate the power of “cross-reactive” T cells. A 2020 study found that T cells in individuals who recovered from common cold coronaviruses could respond to SARS-CoV-2, although at the time researchers did not know whether this could actually protect against COVID-19.

“Understanding the role of cross-reactive T cells is important because T cells play an important role in the control and resolution of COVID-19 infections,” said study co-leader Alessandro Sette, Dr. Bio. Sci, in the press release.

In the new study, the team found that individuals with cross-reactive T cells had significantly stronger CD4-positive T cell and antibody responses to both doses of the vaccine. Sette explained that with this immune reactivity, the immune system may work faster against the virus.

The study is also one of the first to demonstrate effective CD8-positive T cell data in COVID-19 vaccine research. They found a strong CD8-positive T cell response to the low-dose Moderna vaccine, similar to the response after a patient fights a natural SARS-CoV-2 infection.

“We know naturally infected and recovered people develop excellent CD8-positive T cell responses against SARS-CoV-2; however, there was concern about the generation of CD8-positive T cells by mRNA vaccines,” Trivino said in the press release.

Future research will investigate whether the same vaccine durability holds true for other types of COVID-19 vaccines. Based on current hospitalization rates among unvaccinated individuals, Weiskopf said real-world data suggest that this immune memory does last.

Moderna COVID-19 Vaccine Spurs Lasting Immune Response. News release. La Jolla Institute for Immunology; September 14, 2021. Accessed September 16, 2021. 

https://www.lji.org/news-events/news/post/moderna-covid-19-vaccine-spurs-lasting-immune-response/

Strong immune memory lasted in all age groups tested after receiving the Moderna vaccine, including individuals over 70 years of age who are especially vulnerable to COVID-19.



Study Finds Moderna COVID-19 Vaccine Elicits Lasting Immune Response

The United States, the European Union (EU), and Canada are currently the world leaders in specialty pharmacy. IQVIA’s global use of medicine report states that by 2023, developed markets will see a growth in specialty spending from $336 billion in 2018 to nearly $505 billion and the specialty share of total pharmaceutical spending will reach 50%, since the majority of new drugs have been and will continue to be in specialty groups.

According to IQVIA’s 2018 market prognosis report, 10 developed markets including the United States, EU (UK, Germany, Italy, France and Spain), and other major developed countries (Japan, Canada, Australia, and South Korea) account for 66% of global spending. Specialty share of spending across these countries grew from 42% in 2018 to 50% in 2023.

The 5 largest specialty therapeutic groups will drive the majority of growth: oncology, autoimmune, immunology, HIV, and multiple sclerosis. Over the 5 years, these groups will contribute 74% to specialty growth.

The question is: “Do we expect to see specialty pharmacy expanding beyond these regions”?

As specialty drugs do not have a universal definition yet, United Arab Emirates (UAE) has its own definition for what a specialty drug is. Daman, the largest health insurer in Abu Dhabi and third largest in Dubai, defines specialty medications as “drugs used to treat chronic health conditions where they are continuously monitored and are subject to special evaluation requiring full medical documentation for authorization.”

Hence, they created a list to ensure that those medications are prescribed to the right population. Daman emphasized that the turnaround time for approving specialty drugs might take longer than normal. Daman’s specialty drugs list in 2019 included 37 medications, which increased to 57 in 2021.

As you can see, the number of specialty drugs has been on the rise, which shows the huge interest the UAE has in making these specialty medications available for patients in need.

The overall orphan disease prevalence in the MENA region is 2.97 million patients. According to a white paper published by IQVIA in 2018, it is estimated that 50,000 patients out of the 9.16 million total population are diagnosed with an orphan disease in the UAE.

The Ministry of Health and Prevention in the United Arab Emirates (MOHAP) is committed to increasing patient access to treatments by prioritizing the delivery, assessment, and registration of breakthrough and orphan medications.

Fast Track Registration of Specialty Drugs

UAE drug authorities have been putting lots of efforts into fast-tracking registration for innovative products directly after FDA or EMA approval. In 2018, MOHAP issued a Ministerial Decree for the Fast Track Registration of 1) drugs containing a, completely or partially new active ingredient and whose owner holds a patent (innovative drugs) and 2) drugs that are used for the treatment, diagnosis or prevention of rare diseases (orphan).

Even if final approval has not yet been granted by any of the relevant accredited international regulatory bodies, an innovative or orphan drug can be included in the Fast Track process following a favorable opinion issued by the regulatory bodies, as long as a certificate of pharmaceutical product is submitted to MOHAP once available.

As part of the process, the MOHAP Drug Registration Committee reviews an innovative drug or orphan drug application within 15 working days of submission, after which it will accept or reject the application within 10 working days the evaluation. Most of the members of the committee must vote in favor of approval.

The price is then decided after the committee has given its approval, and a certificate of registration and pricing for the medication will be issued.

In the past few years, UAE has increased its efforts in developing the health care sector in several ways. One of the ways was to introduce mandatory health insurance to all UAE nationals and expatriate residents and their families.

The implementation of compulsory health insurance in Abu Dhabi nearly a decade ago, and more recently in Dubai and Sharjah, has resulted in substantial growth in health insurance penetration. Authorities believe this mandate will help increase access to care and reduce costs concomitant with routine illnesses, check-ups, emergency treatments, and several other medical requirements.

Even with the insurance mandate in place, some specialty medications such as biologics can be quite expensive. Many patients have basic insurance with cap on annual spend on medications, while others are unable to afford co-payments for these types of medications.

Other medications are only covered for inpatients use but not for outpatient prescriptions such as oral oncology drugs. Therefore, a global pharmaceutical company launched a joint payment access program to improve access to these medications, and we can see many pharma companies following the same path too. 

To address this challenge, organizations such as Axios International and IQVIA were engaged back in 2016. Axios International had previously concentrated on uninsured patients until November 2015, when it started to expand its services to include covered patients who were having some challenges in paying their co-payments.

In addition to independently funded government patient assistance programs (PAP), Axios International and IQVIA are currently the only privately held organizations in the UAE offering PAPs. These programs are run together with the pharmaceutical companies to provide financial assistance to help patients access medications for little or no cost.

Clearly, not everyone qualifies, but many patients have been assisted. So, how do we know whether a patient qualifies? Axios uses its validated Patient Financial Eligibility Tool to evaluate a patient's ability to pay for treatment.

The objective of this tool is to provide a complete picture of the patient’s income sources by taking 3 key metrics into account: income level, standard of living, and assets.

Specialty medications are usually classified as high-cost and high-complexity pharmaceuticals, which means it is not just a matter of cost. These drugs require specialized handling, administration, and storage due to their complex nature.

There are many other complexities associated with specialty drugs and patients are the ultimate victims of all these complexities. That is why such organizations not only provide financial assistance services but offer many services just like hub service providers in the United States, where they act as intermediaries between involved parties (patients, physicians, pharmacists, and pharmaceutical manufacturers). These services include claims support, prior authorization, patient education, adherence support services, drug administration, and home-based clinical support.

The above-mentioned patient services allow pharmaceutical manufacturers to positively affect the patient journey by ensuring efficient distribution of medication and improving adherence rate and outcomes. When patients are engaged, and followed up with during their treatment process, they will gain a greater understanding of their own journey, resulting in more favorable results.

Non-adherence is a growing concern; its consequences not only include health-related consequences (e.g., treatment failure, increased hospital admissions), but also financial ones.

These hub service providers have taken a stand in this concern, committing to providing tailored communication to patients through both new and conventional methods—such as online web portals and telephone outreach—to promote medication adherence among patients.

Specialty Medications Providers and Availability

Many of the specialty medications require high-touch patient care management, which is why they are only available via limited distribution networks, such as hospitals or big pharmacy chains. For example, Luxturna, the first FDA-approved gene therapy for patients with inherited renal disease, can only be supplied to qualified centers/hospitals that meet certain criteria set by the European Medical Association (EMA).

These criteria might include the requirements of trained and competent physicians, as well as supply chain capability to transfer and store the medication, which requires a specific type of transportation (dry ice) and a specific type of freezer (-65 C). In addition, pharmacists must be trained and competent to handle, prepare, and deliver the medication in a ready-to-administer form, with the availability of the facility and equipment necessary for sterile preparation of the medication.

These requirements are not only limited to Luxturna, but to many of the specialty therapies available today. They typically require specialized handling to ensure the medication's quality and proper administration. This can demand complex handling, cold chain storage, and transportation in highly regulated environments, such as temperature-controlled areas, to ensure product integrity is maintained.

With the UAE becoming the third country to approve Luxturna, one of the most advanced gene therapies available worldwide, it demonstrates the country's commitment to bringing innovative and groundbreaking specialty medications to the market early on. The availability of this breakthrough medication was met with the enthusiastic support of the Ministry of Health, where the product’s registration took between 2 to 3 months.

Specialty medications will account for nearly two-thirds of new product launches over the next 5 years, up from 61% in the previous 5 years.

 With the increasing availability of specialty drugs, there must be a specialized space for better scrutiny and monitoring of these medications.

These complex, sensitive, and highly delicate therapies necessitate the formation of new distribution models to link patients to these costly therapies. Patients with complex and rare diseases receiving these therapies require more attention to ensure adherence to the therapy regimen and to effectively manage adverse effects.

There are currently no specialty pharmacies in the UAE that are fully dedicated in dispensing specialty medications with designated hub services. Presently, there are patient access organizations started in the country; however, they don’t offer the other services of specialty pharmacies, which is why there is a great opportunity to establish specialty pharmacy in the region.

Overcoming local regulations, insurance company acceptance, and pharmaceutical company support to the idea is imperative to the success of starting a specialty pharmacy in the UAE.

Zakieh Abuelkhair earned her Bachelor Degree of Pharmacy from the University of Sharjah in the UAE and her Master of Pharmacy Business Administration (MPBA) program at the University of Pittsburgh, a 12-month, executive-style graduate education program designed for working professionals striving to be tomorrow’s leaders in the business of medicines. In the past 2 years, Zakieh has worked at Novartis Pharmaceuticals as a Disease Education Specialist and completed her 6-month pharmacy internship at Cleveland Clinic Abu Dhabi. Her current role is working as Pharmacy Operations Manager at Al Thiqa Specialty Pharmacy in an effort to transform the practice of Specialty Pharmacy in the Middle East.

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The 5 largest specialty therapeutic groups—oncology, autoimmune, immunology, HIV, and multiple sclerosis—will account for 74% of specialty spending growth over the next five years.

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