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The Process of Drug Development PowerPoint Presentation

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Description : Check out this medical PowerPoint presentation titled "The Process of Drug Development" by Liana S. Rosenthal, MD, PhD and Melissa Wolfe beiner, MD.

This medical PowerPoint presentation is about the drug development process, a long and complex process that involves multiple stages and various stakeholders. Here are the typical stages of drug development:

Discovery and Preclinical Testing: Scientists identify potential drug targets and develop compounds that could affect those targets. They then conduct preclinical testing in the laboratory and animal studies to assess the safety and efficacy of the drug candidate.
Investigational New Drug (IND) Application: If the preclinical testing shows promising results, the drug developer submits an IND application to the regulatory agency (such as the FDA in the US) for approval to conduct clinical trials in humans.
Clinical Trials: The clinical trial process has three phases. Phase 1 trials typically involve a small number of healthy volunteers and are designed to assess the safety and tolerability of the drug candidate. Phase 2 trials involve a larger group of patients and aim to evaluate the drugs efficacy and side effects. Phase 3 trials involve an even larger group of patients and compare the drug candidate to existing treatments or placebo to establish its safety and efficacy.
New Drug Application (NDA) and Approval: If the clinical trial results show that the drug is safe and effective, the drug developer submits an NDA to the regulatory agency for approval to market the drug. The regulatory agency reviews the NDA and decides whether to approve the drug for sale.
Post-Marketing Surveillance: Even after a drug is approved and marketed, the drug developer and regulatory agency continue to monitor its safety and efficacy. Adverse events and new information about the drugs effects are reported and evaluated, and the drug label may be updated accordingly.

The drug development process is a lengthy and expensive one, with many drug candidates failing to make it past the preclinical testing or clinical trial phases. It can take over a decade and cost billions of dollars to bring a new drug to market.

Tags : Drug Development, Drug Discovery and Preclinical Testing, Investigational New Drug, Clinical Trials, New Drug Application, pharmaceuticals, pharmaceuticals PowerPoint presentations

Published on : Jun 23, 2023 | Views : 1547 | Downloads : 0

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Slide 1-

The Process of Drug Development Liana S. Rosenthal, MD, PhD Melissa Wolfe beiner, MD
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Agenda Clinical Trial Overview/the drug development process Clinical Trial readiness and the challenges inherent to rare disease How do clinical trials contribute to advancing research in a given disease space NAF (Patient Advocacy) Role in Accelerating Drug Development and Aligning Regulators, Clinicians, and Industry NAF and Biohaven DDC OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 2
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Basic Science Research/Clinical Research Basic Science Research Investigation of a subject to increase knowledge and understanding about it In the sciences, usually refers to research performed on cells, animals, etc Knowledge for the sake of knowledge, but can then be translated to other things Example: An atomic clock that is critical to satellite-based GPS Clinical Research Any research that involves humans Goal is to improve human health or increase understanding of human biology May be observational vs interventional Observational: we observe—clinical assessments, blood, spinal fluid, imaging, etc in order to understand more about human biology Interventional/clinical trial: we determine if our intervention changes an underlying condition OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 3
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Examples: Observational Studies Framingham Heart Study US Census COVID vaccine monitoring OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 4
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Examples: Clinical Trials Every prescription drug or vaccine you have ever taken, was once part of a clinical trial Antibiotics Birth control pills Blood pressure medications Riluzole, 4-aminopyridine, amantadine, baclofen, etc COVID 19 vaccines OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 5
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Clinical Trials and research terms to know Placebo: A substance or thing with no therapeutic effect Double blind: The researchers and the patients do not know if they are taking placebo or active medication/intervention Powered: the ability of a study to detect a difference, if a difference actually exists Endpoints: an event or outcome that can be measured objectively to determine whether the intervention being studied is beneficial OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 6
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Overview: Clinical Trial Readiness and Advancing SCA Clinical Trials OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 7
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FDA Draft Guidance on Important Elements for Drug Development OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 8 CRC-SCA Outcome Assessments Patient, Caregiver, Advocacy Group Involvement Drug Quality Considerations
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Natural History Studies/Observational Studies Not required by FDA but important to all drug development programs. Knowledge of natural history of disease helps: Define disease population and clinical course: Prognostic enrichment: allows selection of patients more likely to progress and develop disease endpoints Importance of understanding the epidemiology of SCA across the globe and across the USA (NAF genetic testing initiative) Define entry criteria and adequate trial duration Select sensitive and specific outcome assessments Identify new or validate existing biomarkers Clinical Research Consortium-Spinocerebellar ataxia (CRC-SCA)(e.g. natural history study) (CSF collection/Blood draws) IDEA study READISCA study (MRI) OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 9
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The Stages of Clinical Trials OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 10
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OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 11 The Long Process of Drug Development Drug Discovery ~10,000 Compounds Preclinical Testing ~250 Compounds Clinical Testing ~5 Drugs FDA Approval 1 Drug ~4-6 Years ~6-7 Years ~1-2 Years ~12-15 Years
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The Value of Clinical Trials in Advancing Research
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JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 13 The drug or intervention do not work Not enough patients enrolled in the study, so it is not adequately powered SCA 3 is different from SCA 1 so a SCA 3 drug may not work on SCA 1. A study with both groups needs to be adequately powered. The endpoint is not objective or ideal Why do clinical trials fail?
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JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 14 Post-hoc analysis of patients enrolled in long-term extension of Phase 2b/3 troriluzole SCA trial Primary efficacy endpoint: change from baseline in the Total SARA Score after 48 weeks Patients from BHV4157-201 trial versus eligibility criteria matched Ashizawa Natural History cohort: SCA Genotype SCA1, SCA2, SCA3, SCA6 Age at baseline 18 to 75 years of age Gender SARA Score at baseline ≥ 8 and ≤ 30, and Initial SARA gait item score ≥ 2 Troriluzole Treated SCA Patients Treated for 1 Year Compared to Matched Ashizawa Natural History Cohort Troriluzole Study (BHV4157-206) in Spinocerebellar Ataxia (SCA) Achieved Phase 2/3 start in 1Q19 Least Squares Mean2 Change in Total SARA Score (from baseline ± SE) Difference: -1.41 ± 0.411 (95% confidence interval of -2.22 to -0.60) suggesting therapeutic benefits of troriluzole (p=0.0007) SARA: Scale for the Assessment and Rating of Ataxia SCA Patients on Troriluzole vs. Natural History Cohort 1. Matched on eligibility criteria 2. ANCOVA model with fixed effects for cohort, sex, & SCA genotype with age and baseline SARA scores as covariates
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Learnings and Next Steps FDA suggests use of gait, balance and language Items of the SARA Importance of Variability in SARA Scoring Rationale for enriched target population (prognostic enrichment) Natural History Studies Study data and anecdotal reports Long-term study design may extinguish placebo effects Exposure-response relationship suggests use of higher troriluzole dose in subsequent study Overall study data (extension phase and subset of randomization phase) consistent with potential therapeutic effect ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES CONFIDENTIAL 15 OCTOBER 2018
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Preparing for BHV4157-206 and future clinical trials OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 16
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JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 17 Anti-sense oligonucleotides (ASO) DNA --> RNA --> Proteins ASO’s bind to the RNA and stop it from making the “bad” protein Coming soon, probably starting with SCA 3 Clinical trials underway
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JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 18 Gene therapy trial for SCA 3 Administered to the cerebrospinal fluid, 1 time Replaces SCA 3 mutated gene with non-mutated gene replacement Uses viral vector to administer gene (viral vector also used in AstraZenaca COVID vaccine) Clinical trials coming soon
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Looking to the Future: Advocacy and Drug Development OCTOBER 2018 ADVANCING INNOVATIVE THERAPIES FOR NEUROLOGICAL DISEASES 19
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Thank you and questions!