06/08/2026 | Ministry of Health

https://www.gov.br/saude/pt-br/assuntos/noticias/2026/junho/ministerio-da-saude-descontinua-temporariamente-estrategia-atual-de-vacinacao-do-butantan-contra-dengue 

The measure was adopted as a precaution until the conclusion of the investigation of 42 rare cases with warning signs that had not been observed in the clinical study.

The Ministry of Health announced, this Monday (June 8), the temporary discontinuation of Butantan-DV's current dengue vaccination strategy. With this, the use of the immunizing agent is paralyzed for the investigation of rare, unexpected events incompatible with the clinical study conducted for the vaccine's approval.

The measure, adopted as a precaution and based on consensus between the Ministry of Health and the National Health Surveillance Agency (Anvisa), comes after the registration of 42 cases with warning signs, such as intense abdominal pain, persistent vomiting, and bleeding. Among these, three were classified as serious, including two deaths. The identification of these episodes was made through pharmacovigilance – a standard monitoring procedure adopted whenever a new product of this type begins to be used in the Unified Health System (SUS).

These are rare events that correspond to 0.008% of a total of 500,000 doses administered up to May 30th – and there is still no conclusive result regarding their correlation with the vaccine.

"This discontinuation has a purpose. First, it is a precautionary measure that should always guide those who respect life and those who respect science, even more so when we are talking about vaccination. Second, it allows the Ministry of Health, Anvisa (Brazilian Health Regulatory Agency), and the Butantan Institute to deepen the investigation of the cases, especially the registered deaths, for which there is still insufficient information to establish a causal relationship with the vaccine," highlighted the Minister of Health, Alexandre Padilha.

The vaccination strategy with the Butantan vaccine was aimed at primary healthcare professionals and, more broadly, at the 15-49 age group in three cities – Botucatu/SP, Maranguape/CE, and Nova Lima/MG – and the Araguaína region in Tocantins. The action began in January of this year.

Surveillance

The identification of warning signs after the introduction of a vaccine into the healthcare system follows international pharmacovigilance protocols.

Before being incorporated into the Brazilian Unified Health System (SUS), the Butantan-DV vaccine underwent all the evaluation stages required by regulatory bodies, with results demonstrating its safety and efficacy.

The decision to temporarily suspend the application of the immunizing agent was made after discussions with the Interinstitutional Committee for Pharmacovigilance of Vaccines and other Immunobiologicals (Cifavi) and the Technical Advisory Chamber on Immunization (Ctai), bodies composed of representatives from the Ministry of Health, Anvisa (Brazilian Health Regulatory Agency), and specialists.

It is important to highlight that this measure does not invalidate the vaccine's efficacy nor alter the evidence of protection observed to date. Those who have already been immunized remain protected, and epidemiological surveillance continues to monitor the vaccinated population.

For those who have already received the vaccine, the recommendation is to monitor their health status for 21 days after application. In case of symptoms such as fever, intense abdominal pain, persistent vomiting, bleeding, dizziness, excessive drowsiness, signs of dehydration, or worsening of general condition, medical attention should be sought immediately.

From now on, health teams will reinforce surveillance of vaccinated patients who present dengue symptoms, paying special attention to recognizing warning signs and signs of severity. They should also intensify case reporting, activate local surveillance, and ensure immediate referral for clinical care when necessary.

Combating Dengue

The Ministry of Health maintains all other dengue control strategies in operation, with the aim of reducing virus circulation, preventing severe cases, decreasing hospitalizations, and avoiding deaths.

By the end of May of this year, the country had registered a 94% decrease in the number of cases compared to the same period in 2024. There were 365,000 probable cases, well below the 5.8 million recorded in the same period of 2024. Regarding deaths, the reduction was 97%, with 178 deaths recorded in 2026 and more than 6,300 in 2024.

Among the strategic measures adopted by the Ministry of Health are the strengthening of epidemiological surveillance, continuous monitoring of suspected and confirmed cases, identification and elimination of breeding grounds for the Aedes aegypti mosquito, permanent public awareness campaigns, and technical and financial support to states and municipalities for vector control actions.

The Ministry also maintains the distribution of insecticides, larvicides, and equipment to local surveillance teams, in addition to the continuous training of health professionals for early diagnosis and proper patient management. Another important front is the laboratory monitoring of circulating dengue virus serotypes, allowing for faster responses to increased transmission in certain regions.

Public participation remains fundamental to controlling the disease. The recommendation is to eliminate containers that can accumulate stagnant water, keep water tanks properly closed, clean gutters, dispose of waste correctly, and allow access for disease control agents when necessary.

Questions and Answers

Why is the Butantan Institute's dengue vaccine being discontinued?

The Ministry of Health has temporarily discontinued the current strategy for administering the Butantan-DV vaccine as a precaution. 501,000 doses were administered to primary healthcare professionals working on the front lines of the Brazilian Unified Health System (SUS), and to people aged 15 to 59 in the municipalities of Nova Lima (MG), Maranguape (CE), and Botucatu (SP), as well as the Araguaína region (TO). The epidemiological surveillance monitoring system functioned and detected an alarm signal that needs further investigation. The decision will allow for more in-depth investigations to assess the signals and does not invalidate the vaccine's effectiveness (clinical studies showed an overall efficacy of 65% and 80.5% for severe cases).

The Ministry of Health acted based on concrete data from the pharmacovigilance system—not on rumors, unfounded associations, or political pressure. It is the opposite of denialism: science being applied in real time, with transparency.

The Brazilian Unified Health System (SUS) continues to offer dengue immunization for children and adolescents aged 10 to 14 with the Qdenga vaccine, produced in Japan and offered in the public network since 2024. Approximately 8 million doses have already been administered in Brazil, with visible impacts on disease control.

What problem was found?

An alarm signal was detected by the pharmacovigilance of the health network. Of 501,000 people who received the vaccine, 42 cases of rare and unexpected reactions were recorded (corresponding to 0.008% of the total) that had not been identified in clinical studies nor predicted in the package insert. Three serious cases were also identified, two of which resulted in death. None of these occurred in the three cities and region where vaccination was expanded to the population.

It cannot be stated that the deaths were caused by the vaccine, but it was considered a warning sign that justifies a thorough investigation. Possible comorbidities, risk factors, and other situations that may have contributed to the deaths will be verified.

How was the vaccine approval process?

The Butantan Institute worked on developing the vaccine for approximately 20 years, having licensed the seed technology from the United States National Institutes of Health (NIH). Phase 1, 2, and 3 clinical studies were conducted, according to current protocols. More than 11,000 volunteers were vaccinated and monitored for 5 years to verify efficacy and safety. With this data, Anvisa authorized the production and commercialization of the vaccine in December 2025.

The vaccine showed an overall efficacy of 65% against the disease and an efficacy of 80.5% for more severe cases.

Is the vaccine safe?

Of more than 500,000 doses administered, rare and unexpected effects appeared in 0.008% of people who received the vaccine. The vaccine went through all the required stages before being approved: pre-clinical and clinical development with 11,000 vaccinated individuals, safety, immunogenicity, and efficacy studies, including follow-up of up to 5 years of vaccinated individuals.

The vaccine does not cease to be safe when a signal is detected. It is an alert that needs to be investigated more thoroughly. This identification after application is part of the process and is being analyzed with the utmost rigor.

Therefore, continuous monitoring is essential, and it works.

Are those who have already received the vaccine at risk?

Those who have received the vaccine are protected against the four types of dengue: the decision does not invalidate the vaccine's effectiveness. Vaccines, like any other medications, are not exempt from rare and unexpected events. The identified risk is rare. More than 90% of vaccinated individuals did not experience side effects after vaccination. The vast majority of reported effects are mild to moderate, disappearing after a few days.

The recommendation remains the same for those who have been vaccinated: monitor your health for up to 21 days after the dose. Seek immediate medical attention if you experience fever, severe abdominal pain, persistent vomiting, bleeding, dizziness, excessive drowsiness, signs of dehydration, or a worsening of your general condition.

After 21 days, no active component of the vaccine is detectable in the body.

What are the most common adverse effects?

According to the vaccine leaflet, the expected adverse effects in some vaccinated individuals are: headache, body aches, eye pain, skin rashes, extreme fatigue, itching, nausea, sensitivity to light, and chills.

Very rarely, cases similar to dengue fever with fever have been detected.

Does this mean the vaccination strategy failed?

No. On the contrary. The identification of cases shows that the vaccine monitoring and safety mechanisms work and can quickly detect possible warning signs.

Was there a problem with the storage or application of the vaccine?

There is no evidence of failures in the storage, transport, or application of the doses. These hypotheses continue to be verified during the investigations.

What will be done with the doses that are already in the health centers?

The vaccines should not be discarded or destroyed. They should remain stored in the cold chain until the investigation is completed.

Are vaccines still important?

Yes. Vaccines remain one of the main tools for preventing diseases and saving lives. The investigation of a possible rare and unexpected adverse event demonstrates that the surveillance system is working.

According to the WHO, vaccines have saved 154 million lives in the last 50 years, about 3 million per year. In Brazil, vaccines have helped eradicate polio (infantile paralysis) and rubella, and in the case of measles, the country is free of the disease.

When will there be a decision on resuming vaccination?

The decision will depend on the results of ongoing investigations. The Ministry of Health and Anvisa will release new guidelines as soon as there are sufficient technical conclusions. Anvisa will have a panel of experts to further investigate the epidemiological situation regarding this vaccine.

What is the current dengue scenario in Brazil in 2026?

This year, up to May 30th (week 21), Brazil registered a 97% reduction in deaths and a 94% reduction in probable cases compared to the same period in 2024.

However, dengue fever remains the country's most endemic disease. Vaccines continue to be fundamental weapons in this fight and will help achieve even better results.

Ministry of Health

06/08/2026 | Michael Esquer and Vitória Nascimento | Valor Econômico/p/Abrafarma

https://www.abrafarma.com.br/noticias/farmaceuticas-globais-ampliam-investimento-em-pesquisas-de-doencas-raras-no-brasil 

The role of the SUS (Brazilian Public Health System), new legislation, and genetic diversity have attracted the interest of multinational companies.

Influenced by recent regulatory advances, the country's genetic diversity, and the potential role of the Unified Health System (SUS) as a source of data and a future buyer of medicines, large global pharmaceutical groups have prioritized Brazil as a market for clinical research in rare diseases. This segment, also called "high-cost," involves treatments that approach millions of dollars in some cases, and even exceed that in others.

According to a survey by Interfarma, which brings together multinational companies in the sector present in the country, along with IQVIA, Brazil ranks 20th globally in clinical studies, but could reach 10th place. Although there is no segmented data for rare diseases, companies affirm that the country, where these types of diseases affect approximately 13 million people, has been a priority market for such investments, a trend that is expected to grow with the Clinical Research Law, regulated in 2025.

“The regulatory framework is improving and encouraging international companies to use the country as a platform for product development and clinical research,” said Nelson Mussolini, president of Sindusfarma, the pharmaceutical industry union.

The country attracts attention due to the size of its population and the quality of data from the SUS (Brazilian public health system), which facilitates the tracking of patients with rare diseases, said Rubens Granja, partner at the law firm Lefosse, a specialist in the health sector. The expectation of government purchases of future innovations also sparks interest. “The government is an excellent client and buyer, and companies operating in this segment at some point develop strategies for incorporating these products into the SUS (Brazilian Public Health System), which involves conducting economic studies to show that this is advantageous.”

“National companies cannot make this investment due to a lack of technology.” — Nelson Mussolini

Mussolini, from Sindusfarma, explains that multinational companies are the ones that invest the most in the segment, since national companies focus on biosimilars and synthetics. “National companies cannot make this investment, due to a lack of technology and because they don't have the working capital to invest R$ 3 billion in products that may turn out not to have the expected effect.”

The profile of pharmaceutical companies participating in this movement ranges from those that have been operating in the field since their founding to those that have undergone acquisition processes or portfolio segmentation to focus on more complex areas, such as rare diseases. “There’s a question of global positioning for these companies as innovation pharmacies,” said Granja, from Lefosse.

The Anglo-Swedish company AstraZeneca acquired the American company Alexion during the pandemic, which became its rare diseases division. In Brazil, the unit accounts for approximately 17% of the company's revenue, a level similar to the global average. Considering AstraZeneca's Brazilian revenue, around US$1.4 billion, the division's revenue would be approximately US$224 million.

“Brazil is a unique country in terms of population diversity. “With a [research] center in the country, there is already a considerable diversity of population,” said Marc Dunoyer, global president of Alexion and former CFO of AstraZeneca, to Valor. The pharmaceutical company invested R$ 10 million in clinical research on rare diseases in Brazil in 2025 and expects to reach R$ 12 million this year. Considering other areas, the total investment was R$ 315 million.

The American company Biogen has about 60% of its revenue in the country anchored in rare diseases and plans to invest R$ 165 million in clinical research in Brazil this year, a considerable portion for this segment. “In this market, we have less than 20% of therapies discovered.” "In other words, there are still 80% of diseases without innovation," stated General Manager Rogério Frabetti. He said the field is investment-intensive, but tends to have higher margins due to "good discoveries."

The Swiss company Roche integrates this field within divisions such as neurology, hematology, and immunology, and in Brazil, invests approximately 20% of its total investment in clinical research for rare diseases, which would correspond to about R$ 118 million considering the R$ 590 million investment planned for 2025. "Brazil is considered one of the priority countries, even due to its strategic relevance. And we are committed to maintaining and increasing [investment in clinical research]," said Michele França, the company's medical director.

Another Swiss company investing in rare diseases, Novartis integrates the field into its neurology, renal, and hematology divisions. In 2025, it invested R$ 145 million in clinical research in the country. It also did not specify how much was allocated to rare diseases, but reported that 18 of the 107 ongoing studies in the country are related to this segment. "The country plays a relevant role in the global innovation strategy, with the infrastructure to host studies," said Sylvester Feddes, president of the laboratory in Brazil.

"There's a question of global positioning as innovative pharmaceutical companies" — Rubens Granja

One of the pioneers, the French company Sanofi, invested R$ 153 million in clinical research in the country in 2025. It also doesn't disclose how much was for rare diseases, but said that 11 of the approximately 50 studies underway in the country are in this field. "It's one of the priority areas, with a significant presence both in the global pipeline and in studies conducted locally," said Ana Resque, the company's medical director in the country.

The Japanese company Takeda, with more than 60% of its global portfolio focused on rare diseases, has defined Brazil as a priority country for research and development in 2025, and has already invested approximately US$ 250 million in a partnership with the state-owned Hemobrás for the production and transfer of technology for the treatment of hemophilia, a type of genetic disease that affects blood clotting. "It is estimated that it takes about ten years from initial research to the availability of a new drug," said the executive director in Brazil, Vivian Kiran Lee stated that the field demands resilience.

However, access to treatments like those offered by these laboratories remains a challenge in the country, despite their interest in expanding it. Today, incorporation into the SUS (Brazilian Public Health System) does not necessarily mean availability to the patient. A survey by the Moka panel, shared by Interfarma with Valor, shows that only 41% of the 232 technologies for rare diseases submitted for incorporation into the SUS since 2012 have been incorporated, and of those, only 51, or about 54% of the total, have actually been made available.

Among the companies mentioned, medications such as eculizumab (AstraZeneca), for rare blood diseases; nusinersen (Biogen) and risdiplam (Roche), for spinal muscular atrophy; emicizumab (Roche), for hemophilia; and imiglucerase and alpha-alglucosidase (Sanofi), for Gaucher and Pompe diseases, are already available in the SUS. Novartis also has the first gene therapy for spinal muscular atrophy in the public healthcare system, "Zolgensma," which costs an average of R$7 million and has been provided to children since 2025 under a risk-sharing agreement.

"The main challenge still lies in the stage of incorporation into the healthcare system and effective access." In other words, more innovation is arriving, but the bottleneck remains transforming this progress into real and equitable access,” said Antoine Daher, president of the Brazilian Federation of Associations for Rare Diseases (Febraras).

The Ministry of Health said it has expanded the availability of treatments for rare diseases and that the country now has 50 specialized services in operation. According to the ministry, this number is more than double that of 2022. “Around 20 new technologies were incorporated during this period,” it stated in a note, citing Novartis' gene therapy.

“We also invested more than R$ 1 billion in offering a new treatment for cystic fibrosis, Trikafta,” it added. “And we started offering complete exome sequencing, a high-precision test that identifies rare genetic alterations more quickly and safely,” it concluded.

06/08/2026  |  News Medical Life Sciences

https://www.news-medical.net/news/20260608/Online-tool-transforms-patient-experience-in-national-cancer-trials.aspx 

In a step forward for patient-centered cancer research, a new pilot project reveals that 84% of participants had a positive experience using a newly developed online tool created by the Alliance for Clinical Trials in Oncology called the Participant Engagement Portal (PEP). Alliance created PEP to foster direct connection between cancer researchers and the individuals who volunteer for clinical studies. The goal was to make it easier for patients and clinicians to share information, deliver trial updates, and self-report on social risk factors. The results of this national project are published in JNCI Cancer Spectrum.

"We know that participating in a clinical trial can feel overwhelming. By designing PEP directly with patients and advocates, we built a digital space that feels supportive, respectful and incredibly easy to use. This portal isn't just about recruiting more people into studies; it's about treating our participants as true partners in research." Norah Crossnohere, PhD, MHS, lead author of the study and Assistant Professor in the Division of General Internal Medicine and member of the Cancer Control Program, The Ohio State University

PEP had excellent usability among participants, with 96% of users saying the platform was easy to access and 93% reporting its surveys were easy to complete. Most notably, 93% of participants agreed to be contacted for future research opportunities.

For this pilot project, PEP was tested as an optional component for participants in the Multi-Cancer Early Detection (MCED) Biobank Study (Alliance A212102), an ongoing national clinical trial looking into future blood tests that might detect certain cancers early. Out of 2,221 people in the biobank study, 899 participants (40%) chose to opt in to PEP. Beyond high satisfaction rates, the PEP pilot revealed several important findings:

• Gathering vital patient-centered data: The portal successfully gathered information on "social determinants of health," such as education, insurance status, housing stability, food insecurity, and financial concerns. Gathering these data helps researchers better understand how a patient's everyday environment affects their long-term cancer journey and treatment outcomes.
• Data sharing: Participants demonstrated a high willingness to securely share limited personal data. For example, all respondents (100%) provided their zip code and 94% were comfortable disclosing their ancestry or ethnic origin.
• Community vs. academic differences: PEP was most successful at local community clinics, outperforming larger academic medical centers in patient enrollment rate and participation.

Understanding how PEP works
PEP is a secure, user-friendly website built to support patients becoming more active partners in cancer research. Unlike traditional digital health tools focused on recruitment, PEP is specifically designed to support and enrich experiences of individuals already enrolled in a clinical trial.

The portal functions through several innovative features:

• Two-way communication: Traditionally, study information only flows one way. Instead, PEP creates a "bidirectional" bridge, allowing researchers to send patient-centered newsletters, study progress updates, and final study results directly to the participants in the method of their choosing.
• Password-free access: To eliminate frustration, PEP did not require usernames and passwords. Instead, patients receive a secure, unique web link, via text or email, that takes them straight to their portal and survey pages without requiring a login.
• Accessible and multilingual design: PEP was shaped by an advisory board of healthcare providers, researchers, patient advocates, and cancer patients and survivors. It uses plain language and colorful infographics, limiting medical jargon. PEP content and messaging is available in both English and Spanish, using a "transcreation" process to ensure the content is culturally meaningful and nuanced.

"We were eager to offer trial participants a format to allow connection to the study team," said Suzanne George, MD, senior author on the study project and Professor of Medicine at Harvard Medical School. "Nearly all participants who engaged with the tool opted in for recontact for future research which allows the ability to build a research community. PEP gives patients a direct way self-report key data elements, such as specific social and economic factors which may impact a person's cancer journey."

While the PEP pilot was a success, researchers noted that future versions must focus on reaching a more diverse group of patients, as PEP enrollees skewed more female and less ethnically diverse compared to the overall biobank study population. Moving forward, Alliance plans to build on these findings by creating educational videos for clinical staff and expanding PEP across more national clinical trials.

"The Alliance connects thousands of cancer specialists across North America, and PEP provides us with a scalable roadmap for modernizing how we interact with our study participants," said Nancy Campbell, MSN, BSN, co-author and Associate Director of Operations of the Alliance Data Innovation Lab, a part of Alliance for Clinical Trials in Oncology. "This tool is a tangible step toward making clinical trials more transparent, collaborative, and hopefully more successful for the communities we serve."

PEP was funded by the Alliance for Clinical Trials in Oncology Foundation. It was also supported in part by Alliance Foundation Trials, LLC, and Genentech, a member of the Roche Group. N.L.C. received support from NCI award U24CA252977 during the period of this study.

Source:
Alliance for Clinical Trials in Oncology

Journal reference:
Crossnohere, N. L., et al. (2026). Digitally engaging participants in cancer clinical trials: Design and pilot of the Alliance Participant Engagement Portal (PEP). JNCI Cancer Spectrum. DOI: 10.1093/jncics/pkag038. https://academic.oup.com/jncics/article/10/3/pkag038/8651741

06/08/2026  |  Ferdous Al-Faruque  |  Regulatory Affairs Professionals Society

https://www.raps.org/resource/industry-groups-seek-more-flexibility-for-dhts-in-clinical-trials.html 

Industry groups have asked the US Food and Drug Administration (FDA) to provide more clarity about its regulatory approach to digital health technologies (DHTs), as well as for greater flexibility in their use in clinical trials.

In a March request for information, FDA asked stakeholders what opportunities there are for using DHTs when developing medical products and what challenges they face. The agency said the feedback would be used to develop guidance and to inform its decision-making. It noted that DHTs encompass a wide range of software and connected products that may include sensors in wearable, implantable, and ingestible devices.

FDA published a 2023 framework that includes demonstration projects, public meetings, guidance development, and the creation of a DHT steering committee that can promote the use of DHTs and help bring new drugs and devices to market. The same year, the agency published guidance on using DHT to collect clinical investigation data remotely. However, it noted that over the past three years, DHTs and sensors have advanced significantly, offering new opportunities for their use.

"Many of these sensors are present in smartwatches and mobile phones and may be customized using mobile applications (apps) for clinical investigations," said FDA. "Apps and other DHTs are being designed to perform interactive clinical tests of patient function.

"Besides mechanical tasks, screen-based technologies are being explored to test neuropsychiatric functions such as reaction time, cognition, vision, hearing and to evaluate conditions such as autism or post-traumatic stress disorder," the agency added. "In addition, DHTs are being designed specifically for pediatric use and may play a role in evaluating new drugs and biological products in children."

PhRMA
Among the stakeholders who responded to the FDA's RFI, the Pharmaceutical Research and Manufacturers of America (PhRMA), argued that, despite the agency's efforts, there has been limited progress toward allowing the use of DHTs to inform regulatory decision-making. It noted that DHT-derived endpoints have not been used as primary or secondary endpoints in clinical trials to the extent anticipated.

PhRMA noted that DHTs have the potential to advance clinical investigations by enabling innovative trial designs and data acquisition, and to support decentralized clinical trials that allow patients who otherwise would not be able to participate.

"Technologies, such as wearable, implantable, and ingestible sensors, software, and mobile applications, enrich our understanding of patient experiences and outcomes through continuous monitoring and more frequent, robust data collection," said FDA. "The COVID-19 pandemic highlighted the utility of DHTs by demonstrating how remote data acquisition can maintain trial continuity during public health emergencies.

"PhRMA encourages FDA to advance DHT adoption by building on learnings from demonstration projects, pilot programs, public meetings, and guidance development," the group added. "We also encourage increased engagement through the DHT Steering Committee and the Digital Health Center of Excellence (“DHCoE”), including enhanced opportunities for targeted engagements with sponsors (e.g., through pre-IND meetings)."

PhRMA asked FDA for flexibility to accommodate evolving DHTs. More specifically, the group asked the agency to provide a clear, flexible, and risk-based approach to allow use of fit-for-purpose DHTs; provide practical tools such as templates and decision trees to use DHTs; clarify the agency's expectations for managing DHT changes, such as software updates; and ensure regulatory harmonization on DHTs not only between product centers but also with other drug regulators.

CPC
The Combination Products Coalition (CPC) wrote to FDA and said there is a lack of clarity about differentiation across DHT use cases, timing and placement of DHT information in submissions, and cross-center expectations.

The group asked the agency to promote transparency by clarifying how DHTs will be evaluated and incorporated into regulatory decision-making. It also asked that DHCoE be empowered to serve as a formal cross-center coordination body for DHT matters and that the agency play a leadership role in promoting global harmonization on the topic. It also asked FDA to support least‑burdensome submission mechanisms, such as enabling a master file-type approach for DHTs.

"This would allow third-party providers to maintain centralized, risk-proportionate, documentation on DHT and AI tool performance, validation, and lifecycle management, which sponsors could then reference across multiple programs, especially in cases where a third-party is producing a clinical trial only use product and therefore does not seek FDA clearance/approval," said CPC. "Such an approach is particularly valuable where proprietary information limits the ability of third-party developers to fully disclose underlying methodologies directly to multiple sponsors, creating inefficiencies and potential barriers to adoption. This helps to reduce duplicative submissions, facilitate more efficient review, and appropriately delineate responsibilities between product sponsors and external technology developers.

"This supports scalable adoption of DHTs and AI-enabled tools, while ensuring FDA maintains appropriate oversight of critical information supporting regulatory decision-making," the group added.

BIO
The Biotechnology Innovation Organization (BIO) said that its members agreed the most significant barriers to adopting DHTs include the lack of clarity about evidentiary expectations for DHT-derived endpoints, verification and validation requirements, and selecting the right regulatory pathways for DHT use in drug development. The group also noted that the lack of cross-center consistency, the opaqueness of FDA's internal processes regarding DHT reviews, and the lack of publicly available information have been major challenges for its members.

"BIO recommends timely Agency-stakeholder engagement mechanisms, both formal and informal, including prioritizing in-person meetings, transparent feedback timelines, and coordinated cross-Center alignment," said BIO. "BIO members also highlight the need for practical tools, such as risk‑tiered templates, case studies, decision trees, and examples to support consistent implementation.

"Specifically, BIO considers evidentiary templates to be essential, practical instruments that enable fit-for-purpose development and promote more consistent and predictable integration of DHTs into clinical research," the group added.

BIO also asked for more clarity on how sponsors should handle missing or incomplete data, manage software and firmware updates during clinical trials, establish composite or multimodal endpoints, and determine when evidence can be reused or bridged across contexts of use, populations, and device versions. The group also said it's important for FDA to address equity and accessibility considerations to ensure DHTs reliably perform across diverse populations and don't widen the digital divide.

06/08/2026  |  Gareth Macdonald  |  BioXconomy

https://www.bioxconomy.com/clinical-and-research/digital-twins-reshape-drug-trials-from-protocol-design-to-physician-training 

Computer models simulate patient responses, cutting costs and time while maintaining trial integrity through predictive analytics and training.

“Digital twins” are having a significant impact on drug trials, reshaping everything from protocol design and cohort balancing to study staff training, according to a new analysis.

Hamed Taherdoost, a professor in the Department of Arts, Communications and Social Sciences, at University Canada West, discussed the increasingly important role twins are playing in the trials space in a study published last week.

“In silico trials employ computer models to simulate clinical trials, enabling researchers to generate predictions of patient responses without relying on traditional, resource-intensive approaches,” he wrote, citing a recent breast cancer screening study as an example.

“The Virtual Imaging Clinical Trial for Regulatory Evaluation (VICTRE) showed that in silico methods can replicate clinical trial outcomes, providing a cost-effective alternative to conventional trials.”

Twins and “virtual patients” have also helped researchers predict how diseases like tuberculosis will respond to candidate treatment regimens during the protocol design stage, Taherdoost said.

“By generating heterogeneous virtual patient populations before conducting actual trials, researchers can compare various outcomes and optimize trial designs. This approach not only reduces costs but also shortens the time to market for therapies.”

Cohort curation
Cohort balancing is another area in which digital twins are making an impact. 

In drug trials, it is important that treatment groups are comparable and composed of people with similar characteristics. The challenge is that, as studies progress, participants can drop out, thereby changing the makeup of a cohort.

And this is where digital twins are helping, according to Taherdoost.

“DTs can model dropout risk using real-time feedback on patient engagement levels and health status. This allows for preemptive intervention to keep patients engaged and preserve cohort integrity. 

“These insights may be utilized to develop dropout prevention strategies by addressing patient needs before they lead to disengagement. Through dynamic cohort balancing, researchers can maintain comparable trial arms, thus enhancing the validity of the findings,” he continued.

Thinking caps on
Digital twins are already influencing the design and operation of studies, but their impact on the drug trials sector is even more profound, according to Taherdoost, who pointed to their growing use as a training tool.

“The Oncology Thinking Cap trial illustrated the potential of using scaffolded simulations to enhance clinical trial design education for medical students, enabling iterative improvement through simulated feedback.

“This approach demonstrates the potential of digital twins not only to improve trial processes but also to educate and empower stakeholders involved in conducting clinical research,” he wrote.