Rare Disease

Rare Disease

Biologics can provide opportunity for identifying a treatment or cure in cases of rare disease. BBCR team experience in biologics for rare diseases included rare cancers and precision medicine can help develop a targeted strategy including studies with fewer patients that control for safety issues.

March 23rd, 2022 | Rare Disease

BBCR team experience in biologics for rare diseases included rare cancers and precision medicine can help develop a targeted strategy including studies with fewer patients that control for safety issues. Indications analysis and prioritization Strategic drug assessment Clinical study design and protocol Biomarker strategy Early Clinical Development FDA meeting and submission Pre-ND integrated development plan […]

BBCR partners with small and medium sized drug and device biotechnology firms. Our clients are clinical researchers and innovators looking for an efficient path to approval, and come to us from across the globe. Reach out today to learn more.

March 16th, 2022 | Rare Disease

The BBCR mission is to simplify clinical research, encourage cost-effective trials, and help innovators navigate through the regulatory process. Who We Serve: Small & Medium Sized Biotech Companies currently moving from pre-clinical studies toward clinical trials in need of interim Chief Medical Officer in need of regulartory strategy consultancy in need of preparing a pre-IND meeting […]

Boston Biotech Clinical Research (BBCR) works with biotech, pharmaceutical, and device companies to develop a clinical and regulatory roadmap consisting of simplified clinical programs, streamlined protocols, and cost-effective trials.

February 22nd, 2022 | Rare Disease

Our Boston-based, integrated, boutique consulting team specializes in rare disease and orphan indications, and is dedicated to supporting pharmaceutical innovators and nurturing each product’s strengths. The BBCR mission is to simplify clinical research, encourage cost-effective trials, and help innovators navigate through the regulatory process.

BBCR’s experience and understanding of how the FDA views orphan applications and structuring a development program to deliver the data and rationale to satisfy the FDA can substantially reduce the review period and increase approval.

February 16th, 2022 | Rare Disease

A strategic approach creates opportunity for time efficiencies The Orphan Drug Act is an important piece of legislation that uses financial incentives to encourage the development of drugs that treat rare diseases and precision medicine impacting disease sub-populations. BBCR’s team of experts can help your company create a roadmap specially customized to ensure successful product […]

BBCR uses innovative approaches to de-risk your product development. For our clients interested in Proof of Mechanism and Proof of Concept – PoM and PoC – BBCR has the expertise to ensure successful product development at any stage of development.

February 2nd, 2022 | Rare Disease

The BBCR team designs Proof of Concept (PoC) Trials and Proof of Mechanism (PoM) studies with the drug clinical plan and regulatory strategy in mind. Proof of Mechanism (PoM) Usually in Healthy Volunteers, Phase 1 study Essential for the selection of appropriate dose for PoC, disease model and biomarkers Investigate drug concentration at the target […]

Trial for Duchenne muscular dystrophy cell therapy.

January 27th, 2022 | Rare Disease

Duchenne Muscular Dystrophy is the most common fatal genetic disorder to affect children around the world. The first patient, a six year old boy, received, in November 2021, an infusion of DT-DEC01, a novel cell therapy containing Dystrophin Expressing Chimeric Cells (DEC),  in Phase I pilot clinical study for the treatment of Duchenne muscular dystrophy (DMD). […]

BBCR is highly experienced in developing innovative approaches to de-risk your product development during the early clinical development stage, including designing Proof of Concept (PoC) Trials and Proof of Mechanism (PoM) studies.

January 25th, 2022 | Rare Disease

BBCR specializes in the strategy and delivery of early-phase clinical development services to enable informed, timely decision making for our clients. Proof of Mechanism (PoM) Usually in Healthy Volunteers, Phase 1 study Essential for the selection of appropriate dose for PoC, disease model and biomarkers Investigate drug concentration at the target site of action Investigate […]

BBCR’s team expertise in rare diseases and precision medicine, combined with SCIO approach, helps streamline trials for swift regulatory approvals. For our clients, this ensures that the proper focus is given to cost containment and value-based developments that allow sponsors to move more treatments to market faster.

December 16th, 2021 | Rare Disease

BBCR provides expert market guidance and embraces innovative strategy consulting for effective clinical development plan and regulatory strategy. Developing drugs for rare diseases and precision medicine indications can be rewarding, but navigating the challenges is not for the faint of heart. Expert guidance is essential in an area where the patients are few, lack of […]

BBCR has developed an approach to meet the challenges of researchers and innovators seeking a clearer path to market. Learn more about the advantages of our Strategic Clinical Innovation Organization (SCIO) concept.

December 15th, 2021 | Rare Disease

We aim to transform the transition stage between pre-clinical and clinical endpoints with smart strategy that, at a minimum, redirects the costs to benefit patients. The FDA has been calling for a smarter, more innovative approach, and we believe SCIO is the integrative, multidisciplinary approach to deliver it. Why SCIO? Investing in Strategy Accelerate Patient […]

Our industry needs innovative strategies, and reduced-risk clinical trials. BBCR clinical development services and drug development consulting integrates real world evidence (RWE) into clinical development plans and regulatory strategies.

November 18th, 2021 | Rare Disease

Focus must go to cost containment and value-based developments that allow sponsors to move more treatments to market faster. BBCR’s team expertise in rare diseases and precision medicine combined with SCIO approach helps streamline trials for swift regulatory approvals. Real-World Data can be collected and approved by regulators for Evidence Generation Regarding Safety and Effectiveness. […]

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