The Need for Greater Access
Patient access to medicines across the lifecycle from Phase I testing through to discontinuation follows a typical pattern
(Figure 1-A). The number of patients gaining access grows as the trial process moves forward and continues to increase following
first (1-B) and subsequent launches (1-C).
Actual patient demand for medicines that address an unmet medical need (1-D) often exceeds the level of access that is typically
delivered. The gap between actual patient access and the total demand for access reflects the fact that not all patients who
may benefit from the drug are able to gain access via traditional routes.
Figure 2 illustrates the delay in access that patients in Europe typically experience even after a drug has been approved.
Each year, the European Federation of Pharmaceutical Industries and Associations publishes the Patients W.A.I.T. Indicator,
which stands for Patients Waiting to Access Innovative Medicines. The data represent all medicines available in 2010 that
were approved by the EMA during the years 2007 to 2009.
For those new medicines that doctors can prescribe under national healthcare provisions, the average time elapsing between
the date of European Medicines Agency (EMA) market authorization and the "accessibility" date (i.e. date of completion of
pricing/reimbursement procedures, represented by the green bars in Figure 2) in 11 European countries varied from 88 to 392
days, not considering Germany and the UK. The orange bars represent the percentage of medicines available commercially in
these countries with a valid EMA marketing authorization during the previous three years. This ranged between 39 percent for
Portugal to 86 percent for Greece. Even with a centralized marketing authorization, the delay to access can still be significant
depending on in which European country a patient lives. These delays are even more profound in other territories.
Managed Access Programs can be used to bridge these gaps, in a fully compliant manner. Such programs can be implemented on
a patient-by-patient basis, or for a group or cohort of patients, and are effective at helping companies address patient demand
for medicines around the world.
Patients in the U.S.
FDA's expanded access program (EAP) rules were amended in 2009 to ensure "broad and equitable access to investigational drugs
for treatment." Regulations include specific criteria for authorization, submission requirements, and safeguards to protect
patients and the clinical trial process. The regulations also define the different types of mechanisms that are available,
including individual access under a single-patient IND (investigational new drug; or Emergency IND); access for small to intermediate
groups of patients; and for larger groups via a Treatment IND or Treatment Protocol.
A single-patient IND is a request from a physician to the FDA that an individual patient be allowed access to an investigational
drug. Provisions are also in place to obtain medicines on an emergency-use basis, allowing patient treatment prior to the
completion of the requirements for a single-patient IND.
The FDA established specific criteria for when this type of IND may be appropriate. One example is when the drug is not being
developed because the disease is so rare, which impedes a company's ability to recruit patients for clinical trials. FDA may
also ask the company to consolidate a significant number of single-patient INDs for the same use under an intermediate-size
patient population IND. Treatment INDs and treatment protocols provide access to investigational drugs for large numbers of
patients who would not otherwise qualify for clinical trials.