Portfolio Modeling and Forecasting of Single-Use Rare Disease Treatments

Biopharmaceutical companies are increasingly exploring cutting-edge novel gene therapies (GTs) in an effort to cure rare diseases. This capstone develops and tests a practical forecasting framework for sharing capacity across Roche’s evolving GT portfolio and driving strategic global supply chain ne...

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Main Authors: Leising, Jordan Michael, Goldman, Olivia Claire
Format: Other
Language:en_US
Published: 2021
Subjects:
Online Access:https://hdl.handle.net/1721.1/130983
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author Leising, Jordan Michael
Goldman, Olivia Claire
author_facet Leising, Jordan Michael
Goldman, Olivia Claire
author_sort Leising, Jordan Michael
collection MIT
description Biopharmaceutical companies are increasingly exploring cutting-edge novel gene therapies (GTs) in an effort to cure rare diseases. This capstone develops and tests a practical forecasting framework for sharing capacity across Roche’s evolving GT portfolio and driving strategic global supply chain network design. Our problem is challenging, even by the highly regulated pharmaceutical industry standards, with: (1) substantial R&D and mergers and acquisitions investments, (2) some of the world’s smallest disease populations, (3) one-time patients, (4) lacking commercial infrastructure, and (5) scarce historical or long- term pipeline data. We created three forecast types based on the target disease state knowledge available to predict an asset’s prevalence and incidence patient adoption curves. The resulting asset forecasts are also aggregated into a comprehensive portfolio dashboard. Our user-friendly point model enables stakeholders to market size the prospective current pipeline and risk pool portfolio capacity by clinical phase. We then applied simulations to illustrate long-term product launch scenarios. These tools cater to various stakeholders helping address the key GT production planning and asset targeting problems. Roche has already began utilizing our capstone to methodically consider unknown future assets, with unknown orphan disease severity or populations, in their strategic make vs. buy GT network design decisions.
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spelling mit-1721.1/1309832021-06-16T19:49:24Z Portfolio Modeling and Forecasting of Single-Use Rare Disease Treatments Leising, Jordan Michael Goldman, Olivia Claire Demand Planning Healthcare Supply Chain Strategy Biopharmaceutical companies are increasingly exploring cutting-edge novel gene therapies (GTs) in an effort to cure rare diseases. This capstone develops and tests a practical forecasting framework for sharing capacity across Roche’s evolving GT portfolio and driving strategic global supply chain network design. Our problem is challenging, even by the highly regulated pharmaceutical industry standards, with: (1) substantial R&D and mergers and acquisitions investments, (2) some of the world’s smallest disease populations, (3) one-time patients, (4) lacking commercial infrastructure, and (5) scarce historical or long- term pipeline data. We created three forecast types based on the target disease state knowledge available to predict an asset’s prevalence and incidence patient adoption curves. The resulting asset forecasts are also aggregated into a comprehensive portfolio dashboard. Our user-friendly point model enables stakeholders to market size the prospective current pipeline and risk pool portfolio capacity by clinical phase. We then applied simulations to illustrate long-term product launch scenarios. These tools cater to various stakeholders helping address the key GT production planning and asset targeting problems. Roche has already began utilizing our capstone to methodically consider unknown future assets, with unknown orphan disease severity or populations, in their strategic make vs. buy GT network design decisions. 2021-06-16T19:49:23Z 2021-06-16T19:49:23Z 2021-06-16 Other https://hdl.handle.net/1721.1/130983 en_US CC0 1.0 Universal http://creativecommons.org/publicdomain/zero/1.0/ application/pdf
spellingShingle Demand Planning
Healthcare
Supply Chain Strategy
Leising, Jordan Michael
Goldman, Olivia Claire
Portfolio Modeling and Forecasting of Single-Use Rare Disease Treatments
title Portfolio Modeling and Forecasting of Single-Use Rare Disease Treatments
title_full Portfolio Modeling and Forecasting of Single-Use Rare Disease Treatments
title_fullStr Portfolio Modeling and Forecasting of Single-Use Rare Disease Treatments
title_full_unstemmed Portfolio Modeling and Forecasting of Single-Use Rare Disease Treatments
title_short Portfolio Modeling and Forecasting of Single-Use Rare Disease Treatments
title_sort portfolio modeling and forecasting of single use rare disease treatments
topic Demand Planning
Healthcare
Supply Chain Strategy
url https://hdl.handle.net/1721.1/130983
work_keys_str_mv AT leisingjordanmichael portfoliomodelingandforecastingofsingleuserarediseasetreatments
AT goldmanoliviaclaire portfoliomodelingandforecastingofsingleuserarediseasetreatments