Despite high expectations of economic returns, large investments in regenerative medicine technology have yet to materialize, partly due to a lack of proven business and investment models, regulatory hurdles, and a greater focus on cost-effectiveness for reimbursement decisions by payors. Adoption of new economic modeling methods will better link investment decisions to value-based criteria of health systems.
Get full access to this article
View all access options for this article.
References
1.
CaulfieldT. (2010). Stem cell research and economic promises. J Law Med Ethic, 38:303–313.
2.
MasonC, BrindleyDA, Culme-SeymourE, DavieNL. (2011). Cell therapy industry: billion dollar global business with unlimited potential. Regen Med, 6:265–272.
3.
MartinPA, CoveneyC, KraftA, BrownN, BathP. (2006). Commercial development of stem cell technology: lessons from the past, strategies for the future. Regen Med, 1:801–807.
4.
DaleyGQ. (2010). Stem cells: roadmap to the clinic. J Clin Invest, 120:8–10.
5.
RaoMS. (2011). Funding translational work in cell-based therapy. Cell Stem Cell, 9:7–10.
6.
SimoensS. (2011). Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis, 6:42–49.
7.
BubelaT, LiMD, HafezM, BieberM, AtkinsH. (2012). Is belief larger than fact: expectations, optimism and reality for translational stem cell research. BMC Med, 10:133.
DaleyGQ. (2012). The promise and perils of stem cell therapeutics. Cell Stem Cell, 10:740–749.
10.
LiMD, AtkinsH, BubelaT (2014). The global landscape of stem cell clinical trials. Regen Med [Epub ahead of print]; DOI:10.2217/RME.13.80.
11.
ReedW, NogaSJ, GeeAP, RooneyCM, WagnerJE, McCulloughJ, McKennaDH, WhitesideTL, DonnenbergADet al.. (2009). Production Assistance for Cellular Therapies (PACT): four-year experience from the United States National Heart, Lung, and Blood Institute (NHLBI) contract research program in cell and tissue therapies. Transfusion, 49:786–796.
12.
Canadian Agency for Drug, Technologies in Health. (2006). Guidelines for the Economic Evaluation of Health Technologies: Canada, 3rd edn. Canadian Agency for Drug and Technologies in Health, Ottawa, Canada.
13.
McCabeC, ClaxtonK, CulyerAJ. (2008). The NICE cost effectiveness threshold: what it is and what that means. Pharmacoeconomics, 26:733–744.
14.
ClaxtonK, BriggsA, BuxtonMJ, CulyerAJ, McCabeC, WalkerS, SculpherMJ. (2008). Value based pricing for NHS drugs: an opportunity not to be missed?. BMJ, 336:251–254.
15.
StafinskiT, McCabeC, MenonD. (2010). Funding the unfundable: mechanisms for managing uncertainty in decisions on the introduction of innovative technologies into health care systems. Pharmacoeconomics, 28:113–142.
16.
von TigerstromBJ. (2008). The challenges of regulating stem cell-based products. Trends Biotechnol, 26:653–658.
17.
BalconiM, BrusoniS, OrsenigoL. (2010). In defence of the linear model: an essay. Res Policy, 39:1–13.
18.
CorrP, WilliamsD (2009). The pathway from idea to regulatory approval: examples for drug development.In: Conflict of Interest in Medical Research, Practice and Education. LoB and FieldsMJ, eds. National Academies Press, Washington, DC, pp 375–383.
19.
JolyY, LivingstoneA, DoveES. (2012). Moving beyond commercialization: strategies to maximize the economic and social impact of genomics research. Genome Canada Policy Brief No. 5.
20.
ClaxtonK, NeumanPJ, ArakiSS, WeinsteinMC. (2001). The value of information: an application to a policy model of Alzheimer's disease. Int J Technol Assess Health Care, 17:38–55.
21.
GriffinS, ClaxtonK, HawkinsN, SculpherMJ. (2006). Probabilistic analysis and computationally expensive models: necessary and required?. Value Health, 9:244–252.
22.
PhillipsZ, ClaxtonK, PalmerS. (2008). The half-life of truth? What is the appropriate time horizons for research decisions?. Med Decis Making, 28:287–299.
23.
WillanAR. (2008). Optimal sample size determinations from an industry perspective based on the expected value of information. Clinical Trials, 5:587–594.
24.
GriffinS, ClaxtonK, WeltonN. (2010). Exploring the research decision space: the expected value of information for sequential research designs. Med Decis Making, 30:155–162.
25.
HallPJ, EdlinR, KharroubiSR, GregoryW, McCabeC. (2012). Expected net present value of sample information from burden to investment. Med Decis Making, 32:E11–E21.
26.
ClaxtonK. (1999). The irrelevance of inference: a decision-making approach to the stochastic evaluation of health care technologies. J Health Econ, 18:341–364.
27.
SculpherM, ClaxtonK. (2005). Establishing the cost-effectiveness of new pharmaceuticals under conditions of uncertainty—when is there sufficient evidence. Value Health, 8:433–446.
28.
WillanAR, GoereeR, BoutisK. (2012). Value of Information methods for planning and analyzing clinical studies optimize decision making and research planning. J Clin Epidemiol, 65:870–876.
29.
StafinskiT, MenonD, MarshallD, CaulfieldT. (2011). Societal values in the allocation of health care resources: is it all about the health gain?. Patient, 4:207–225.
30.
MenonD, StafinskiT. (2008). Engaging the public in priority-setting for health technology assessment: findings from a citizens' jury. Health Expect, 11:282–293.
31.
Canadian Institutes of Health Research. (2012). Citizen Engagement in Health Casebook. Canadian Institutes of Health Research, Ottawa, Canada.
32.
McKennaSP. (2011). Measuring patient-reported outcomes: moving beyond misplaced common sense to hard science. BMC Med, 9:8–98.
33.
WalkerS, SculpherM, ClaxtonK, PalmerS. (2012). Coverage with evidence development, only in research, risk sharing or patient access scheme? A framework for coverage decisions. Value Health, 15:570–579.
34.
BackhouseME. (1998). An investment appraisal approach to clinical trial design. Health Econ, 7:605–619.
35.
McCabeC, ClaxtonK, O'HaganA. (2008). Why licensing authorities need to consider the net value of new drugs—addressing the tension between licensing and reimbursement. Int J Technol Assess Health Care, 24:140–145.
36.
Organization for Economic Co-operation, Development Health Project. (2005). Health Technologies and Decision Making. Organization for Economic Co-operation and Development Publishing, Paris.
37.
PetraR. (2012). Prochymal—first stem cell drug approved. Bexhill-on-Sea (UK), Medical News Today, May22. www.medicalnewstoday.com/articles/245704.php
38.
Osiris Therapeutics, Inc. (2013). Prochymal.www.osiris.com/therapeutics.php
39.
National Institute for Health, Care Excellence. (2012). Citizens Council. www.nice.org.uk/aboutnice/howwework/citizenscouncil/citizens_council.jsp
40.
Ontario Ministry of Health, Long-term Care. (2012). Citizen's Council. www.health.gov.on.ca/en/public/programs/drugs/councils/
41.
Health Canada. (2009). Good Manufacturing Practices (GMP) guidelines—2009 edn., verison 2 (GUI-0001). www.hc-sc.gc.ca/dhp-mps/compli-conform/gmp-bpf/docs/gui-0001-eng.php