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[e-drug] WHO Bullentin: Expensive medicines: ensuring objective appraisal and equitable access

E-DRUG: WHO Bulletin: Expensive medicines: ensuring objective appraisal and 
equitable access

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Expensive medicines: ensuring objective appraisal and equitable access
Suzanne R Hill, Lisa Bero, Geoff McColl & Elizabeth Roughhead
Bull World Health Organ
doi: http://dx.doi.org/10.2471/BLT.14.148924

In response to requests for the funding of new drugs, reimbursement 
agencies are re-evaluating some of the methods used in assessing these 
products. Many trials submitted for the regulatory review of new drugs 
do not provide adequate data for subsidy decisions. We argue that all 
involved in bringing medicines to market need to be explicit about the 
additional information required, decide how these data should be 
collected and assessed and the methods that should be used to set a fair 
price for a new drug.

In Australia, a formal appraisal of the cost-effectiveness and budget 
impact of a new medicine precedes any subsidy decision at national 
level. If a new product is subsidized, the government pays an agreed 
price to the manufacturer, sometimes with requirements for financial 
contracts to manage expenditure.

Increasingly, patients are asking for early access to new drugs, 
particularly for treating cancer. In consequence, strategies to 
subsidize drugs for use under conditions of coverage with evidence 
development or managed entry are being proposed. A new drug might be 
approved even if there is no evidence to show that it satisfies the 
standards typically applied in health technology assessments. However, 
this approval is often contingent upon additional requirements for 
subsequent randomized trials or the collection of data on the drug's 
effectiveness and safety in practice. There is no consensus on the best 
methods for identifying drugs appropriate for managed entry schemes, for 
collecting post-approval data or for the use of such data to modify 
decisions about coverage.

In Australia, as in many other countries, several questions need to be 
answered. Can stakeholders produce a workable framework for managed 
entry schemes? What can be done to reduce variation in the inputs used 
for cost-effectiveness models? How can drug or disease registries 
contribute useful information to inform reimbursement decisions? How 
should registry data be evaluated? What can be done to make registry 
data more representative of the population and what types of 
post-progression data should be included in trials of targeted cancer 

Regulatory agencies, insurers and clinicians also need to be able to 
determine if a new drug represents good value for money and what to do 
if an effective drug appears too highly priced for the benefit that it 
offers. The prices of several recently-introduced drugs -for example 
aflibercept, ivacaftor and sofosbuvir - have been questioned.

The way in which drug prices change over time has generally been a 
function of the market. Typically, a new drug is launched under patent 
and can command a good price until the patent expires and competition 
and/or generic products emerge. Exceptionally, public pressure and legal 
challenges decreased the price of several antiretroviral drugs in 
countries with high burdens of human immunodeficiency virus before 
patent expiry. Other strategies, such as compulsory licensing, have had 
limited success. Tiered pricing has also been proposed but defining each 
tier and an appropriate price for each has proved challenging. The 
recent approval of high-priced medicines for many conditions has 
prompted a new round of discussions and calls for radical changes to the 
current commercial model for drug development.

We consider that it is time for a global forum to discuss objectivity 
and equity in access to high-priced drugs. Such a forum should extend 
beyond the usual networks of payers and authorities on health technology 
assessment. It needs to define the methods needed to manage the early 
entry of promising products, i.e. how to evaluate the data that are 
available for early market entry, determine an appropriate initial 
price, optimize the collection of data from clinical practice, enable 
independent trials and manage the exit of products that, in practice, 
are found to be insufficiently effective. The forum should promote the 
development of a method for pricing new drugs. Such a method needs to 
reconcile the need for fair pricing, with the difficulties of obtaining 
accurate information on research, development and manufacturing costs. 
The forum should include representatives of patient and consumer groups, 
so that the right questions are asked, appropriate research priorities 
are set and outcomes are communicated.

In managing access to new drugs, simply continuing to react 
country-by-country and disease-by-disease is not sustainable. We need to 
be more forward-thinking and take some of the pressure off small 
purchasers and countries that are currently trying to make equitable 
decisions in isolation. We need to solve the fundamental problem of how 
to balance objectivity of appraisal and equity in access to new 
products; ensuring that medical advances are affordable, working with a 
viable pharmaceutical industry that responds to public health needs.

Available at: http://www.who.int/bulletin/volumes/93/1/14-148924
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