Drug Development Consulting

Drug Development Services focus on simple, cost effective clinical development based on robust science. Emphasis is placed on risk-benefit assessment to quickly establish the need for further development. RxMD designs scientifically sound studies with careful selection of end points that are approvable and marketable.

RxMD provides strategic consulting (due diligence, target product profile, IND, clinical development plans, early differentiators, proof of relevance and concept) and oversight for execution of clinical development programs.

Filing of INDs

Sponsor developing novel cell therapy for induction of life-long immune tolerance in renal transplant recipients. RxMD rapidly assimilates into the clinical development team and contributes to key clinical sections of the IND, IB, target product profile, clinical development plan, study protocol and briefing book for special protocol assessment.

A novel technology to expand umbilical cord blood hematopoietic stem cells developed by Sponsor. RxMD authors clinical sections of IND for the use of the novel technology in inborn errors of metabolism.

Sponsor develops a metal protein attenuating compound for neurodegenerative conditions. RxMD provides strategic medical, scientific input into the IND and the phase 1 program.

First in Human Studies

In parallel with the filing of an IND by sponsors, RxMD (in partnership with clinical pharmacologists) develops several novel phase 1 study designs for small molecules and biologics that incorporate both sequential dose escalation and a parallel study design with a lean minimalistic approach.

Scientific Monitoring

RxMD requested to oversee leading CRO faltering in large Phase II trial. RxMD makes decisions on eligibility, protocol violators and deviators based on non-inferiority nature of trial (primary efficacy analysis population = evaluable population) for successful conduct of trial, data review, database closure, interpretation of results and finalization of study report.

Study Management

Sponsor delegates to RxMD oversight of large Phase II non-inferiority clinical trial being conducted by premier CRO. RxMD successfully executes trial design, case report forms, database validations, scientific review of data, blinded signal detection, development of statistical analysis plan, identification of programming errors in blinded statistical outputs, interpretation of unblinded outputs and authoring of final study report.

Statistical Analysis Plan (SAP) Development

RxMD, on review of biopharma SAP for Phase III superiority trial notes that analysis for primary efficacy (ITT) population does not account for visit windows, values outside windows, missing values, multiple values within windows, programming for major violations (exclusions from per protocol population), and inconsistencies between SAP and CRF design. RxMD recommends and implements changes on behalf of biopharma and FDA concurs with SAP revisions.

Novel Study Designs

Sponsor consents to a post approval commitment to establish efficacy in an immunocompromised population (precludes placebo control). RxMD establishes definition for immunocompromised population and designs trial using two active control arms to be compared with a placebo arm from the pivotal trial in healthy adults. Novel strategy meets with regulatory approval.

EU Scientific Advice

Sponsor developing drug for a rare neurodegenerative condition with a significant unmet medical need. RxMD personnel join Sponsor for a face to face scientific advice meeting with the UK MHRA and Swedish MPA.

Drug Development in Infants (less than one year olds)

In response to a public health crisis, RxMD quickly develops a protocol to evaluate an antiviral in infants. In a novel study design, RxMD makes allowances for both the gestational and postnatal age of infants, thereby allowing for appropriate exposure efficacy analysis. The study forms the basis for approval of the drug in infants in US and EU. Read the associated publication here.

Due Diligence/In-licensing

Potential investor requests clinical input into the approvability of a new candidate (estimated peak annual sale USD 700-900m) for sickle cell anemia in Phase III clinical development. RxMD, after rigorous scrutiny of Sponsors clinical data, estimates a reasonable probability of success for approval and marketability. Consequent to RxMDs due diligence and recommendation, investor partners with Sponsor (top-rung global pharma).

A mid-sized biopharma wishing to in-license cell therapy in clinical development for osteoarthritis, enlists RxMD as a key member of on-site due diligence team. RxMD estimates a minimal to moderate probability of success for approval and marketability. Consequent to RxMDs due diligence, biopharma builds-in alternative (risk-mitigated) scenarios into discussions with Sponsor.