Clinical trials are expensive and often burdensome on patients. The productivity of pharmaceutical research and development is declining due to high failure rates in clinical research. The resources, time, and funding investments grow with the clinical development moving with successive stages, from pre-clinical to phase 3. Thus, the cost of a failed phase 3 trial is not just the cost associated with the trial itself but the cost of all prior trials as well as the cost of lost time pursuing a potentially viable alternative.  



Small and medium biotech approaching the clinical research stage of their product development faces many challenges, like all pharma companies. Still, in the case of small/ medium biotech, these challenges need special attention and trustworthy solutions because these companies failing clinical trials could impact the company and the technology’s survival. 



Challenges to the regulatory strategy include missing the correct regulatory path forward or the opportunities to ask the right questions during the early interaction with regulators. 



BBCR Consulting, thanks to the implementation of the Strategic Clinical Innovation Organization (SCIO) process, combined with the specificity and seniority of its experts, the capability to integrate any additional expertise needed, and the geographic location in one of the best life science hubs in the world, is set apart from competitors.



The SCIO process was developed by BBCR, who explicitly designed the SCIO process to help pharmaceutical innovators address the complexity and challenges of developing high technological therapies in the context of continually evolving regulatory requirements. The SCIO process provides confidence to researchers and innovators seeking a more straightforward path to market. The FDA has been calling for a more thoughtful, more innovative approach, and the SCIO is the integrative, multidisciplinary process that delivers this innovative approach. 



The SCIO allows for time and cost efficiencies and risk mitigation with a Clear strategy to meet goals, Streamlined protocols for Cost-Effective Trials, Flexible Road-Map, and Robust Clinical Data for a clear and fast path to market.



Real-world evidence (RWE) is essential to understand patients’ unmet medical needs and life experiences with a given pathology. In addition, RWE provides vital information for competing in patient recruitment and product marketing. Finally, RWE drives the development of the Patient Report Outcome questionnaires that can best collect valuable patients’ experiences. For some time now, the FDA and EMA regulators have shown interest in agreeing with the use of RWE for Orphan Drug development. RWE is an excellent source of data which use has been expanded. BBCR’s long-time experience in Orphan Drug development allowed them to review cases of RWE data with the FDA, thus providing knowledge and understanding in RWE selection and multiple adoptions.



BBCR was founded on the well-documented observation that Orphan Drug clinical research has been more likely to succeed than non-orphan clinical research, to market approval. Thus, the creation of the SCIO, which aims to innovate the clinical research process by impacting the early clinical research with a comprehensive and integrated approach. The SCIO process strategies the optimization of the clinical study looking at personalized drug development. The SCIO process will streamline the clinical plan and trial design, saving up to 30% in trial time and cost and reducing the risk of late failure.



Precision medicine may indicate that the drug’s mechanism of action is only relevant in a subpopulation of patients within a disease, that subpopulation may define an orphan subset. Developing drugs in small, molecularly defined, pharmacologically relevant subpopulations of patients with the same clinically recognized disease is increasingly viewed as a viable pathway for bringing drugs to market. 



BBCR promotes and offers the strategy as the starting point. They are agnostic about the best technology to adopt as one fits all. BBCR views the different technologies as tools to be selected based on the product and development specificities. They adopt a combination of bioinformatic sources to assess the product mechanism of action (MOA) and the diseases in their pathophysiology. BBCR is now evaluating different artificial intelligence platforms. Enriching the current bioinformatic data within the specifically designed artificial intelligence platform will further optimize the development plan, trial design, and risk management in clinical research. They target 2022 as the year for artificial intelligence integration into the SCIO process. 


Candida Fratazzi, M.D., Founder & CEO of BBCR, had the opportunity to develop a clinical plan for a secondto-market product for a rare disease. This development offered multiple challenges, among which availability of a minimal number of patients naïve to treatment and how to differentiate this new product in the marketplace. The FDA approved our proposed clinical plan at the first submission without changes, and later, the product was granted market approval. In addition, Dr. Carla Epps, medical officer at the FDA/CDER/OND, in a presentation in 2011, described the above-mentioned clinical plan as Well planned; Clinically meaningful endpoints; Each trial had a distinct purpose.



“Our belief at BBCR is that there are no goals without a strategy and that any therapeutic product needs a strategy to generate quality data. To best achieve our goal to develop a customized strategy for every product, we are committed to integrating technology tools into our SCIO innovative approach,” she says. “Currently, we have integrated bioinformatics tools for the development of our SCIO-generated strategies. We are now looking at adopting artificial intelligence tools into our proprietary SCIO process.”


The SCIO allows for time and cost efficiencies and risk
mitigation with a Clear strategy to meet goals, Streamlined
protocols for Cost-Effective Trials, Flexible Road-Map, and
Robust Clinical Data for a clear and fast path to market


In addition, integrating the SCIO into the clinical research process of drug development allows the use of the technologies supporting the CRO implementation of trials and will best explode their benefit with the customized and streamlined protocol. BBCR Consulting has worked with domestic and international sponsors for many years. Science has no geographic barriers, and we know that exposure to international regulatory, clinical, and biomarker knowledge benefits our customers and enriches our consultant knowledge. 



The orphan drug development experience has proven to be successful. BBCR learned that people affected by many rare diseases might have very different symptoms and disease severity, thus a slow or fast disease progression. In addition, it is well known that epigenetic impacts every patient to determine their response to therapies and much more. “Our biomarkers for personalized medicine senior advisor includes molecular biomarkers in the early development studies, which could provide evidence for stratification of patients, responders, and non-responders to a given treatment, thus accelerating the product’s clinical research and market approval,” adds Candida. “The shift toward patient-centered healthcare has opened the door for individualized approaches to diagnostics and treatment.” 



Personalized medicine aims to provide the right treatment at the right time for every patient instead of a onesize-fits-all approach. It requires a precise understanding of the patient’s condition, experience and needs to tailor treatment. Digital transformation is not about simply adopting the latest technology. The success of a digital transformation requires healthcare leaders to understand their patient’s values and expectations deeply and build expertise and empower staff to work in new ways. Determining which technology would bring the most significant value requires apparent targets and strategic goals, coupled with a comprehensive understanding of relevant operations and linked processes.