Named patient programs
Governments worldwide have created provisions for granting access to drugs prior to approval for patients who have exhausted all alternative treatment options and do not match clinical trial entry criteria. Often grouped under the labels of compassionate use, expanded access, or named patient supply, these programs are governed by rules which vary by country defining access criteria, data collection, promotion, and control of drug distribution.
Within the United States, pre-approval demand is generally met through treatment IND (investigational new drug) applications (INDs), or single-patient INDs. These mechanisms, which fall under the label of expanded access programs, provide access to drugs for groups of patients or individuals residing in the US.
Outside the US, named patient programs provide controlled, pre-approval access to drugs in response to requests by physicians on behalf of specific, or “named”, patients before those medicines are licensed in the patient’s home country.
Through these programs, patients are able to access drugs in late-stage clinical trials or approved in other countries for a genuine, unmet medical need, before those drugs have been licensed in the patient’s home country.
A 1989 European Union (EU) Council Directive laid out the framework for the supply of unregulated medicines in response to unsolicited requests for use by individual patients “on his personal responsibility”. The current legal basis for access to pre-launched medicines in the EU is Article 5 of Directive 2001/83/EC. This legislation offered the possibility of pre-launch use as an exception to the rule that medicines must be authorized before use or used within the context of an approved clinical trial.
With the expansion of the EU in 2004, Regulation 726/2004 evolved compassionate use programmes in order to better meet the expectations of patients.
While pre-launch use is permitted by EU legislation, these programs are governed by the individual member states; each of the 30 member states of the European Economic Area (EEA) has its own nationalized regulations regarding the import of pre-launch medicines. Additionally, even after a drug receives approval, authorities in each country can take several months to make reimbursement decisions, which can delay the ability of patients to access these drugs.
In a majority of EU member states, an authorization from a competent authority is required to initiate named patient supply; in other countries it may be retrospective. For example, the French regulatory agency (AFSSAPS) is primarily concerned with the science supporting a patient’s request for access to a pre-launch drug and has in place a proactive approval system. In contrast to the French system, in Ireland, the prime concern is transparency of supply and patient safety, so retrospective notification is required.
Despite these differences, the reporting of adverse events is expected in all EU member states (set forth in Article 83). The marketing authorization holder or manufacturer must report serious adverse reactions (SARs) occurring within the EU to the competent authority of the member state in which the incident occurred.
Beyond the EU
Many countries outside of the EU offer similar types of pre-launch programs. Canada’s well defined “Special Access Programme” (SAP) provides access to non-marketed drugs to practitioners treating patients with serious or life-threatening illnesses when conventional therapies have failed, are unsuitable, or are unavailable. In this procedure the practitioner is responsible for initiating a request on behalf of a patient and ensuring that the decision to prescribe the drug is supported either by credible evidence available in the medical literature or provided by the manufacturer. This process is time intensive as each case requires a review, followed by an authorization by Health Sante to enable an unregistered drug to be administered to a patient with an otherwise unmet medical need.
Preparing for pre-launch access
As part of a global market access strategy, named patient programs are typically put in place at any stage post phase II and can run in parallel with phase III trials until commercial launch.
Named patient programs can be utilized to provide patient access at a number of stages throughout a product’s life cycle:
- During Phase III, to patients with an unmet medical need who do not meet the clinical trials inclusion criteria
- Bridging the gap between the end of Phase III and receipt of marketing authorization
- Alongside the staggered global launch whilst approval and reimbursement is being sought across countries worldwide
Named patient programs also provide an alternative route to patient access as part of a global commercialization strategy where:
- A formal launch is not planned
- A sales infrastructure does not exist
- It is not commercially viable to seek marketing authorization
Timing for a program allowing pre-launch access should be carefully thought through; ensuring care is taken not to interfere with established clinical trials. If the program is in place too early, the company might not have an adequate supply of drug available to meet the named patient demand. If the program is started too late, there may not be sufficient use of the program. In cases where demand for the drug is extremely high, however, starting the program within just a few months of expected authorization is warranted.
Sufficient planning time must be allowed to bring key stakeholders into the process; these stakeholders include medical affairs, clinical development, regulatory, supply chain, pharmacovigilance, and the global commercial group and affiliates. In addition, adequate time must be allocated to develop information for physicians and pharmacists regarding dosing, administration and restrictions, and establish treatment criteria to insure proper selection of patients.
Benefits to the sponsoring company
In addition to providing significant benefit to patients with unmet medical needs, named patient programs can offer important benefits to the sponsoring company in the areas of assessment and planning, relationship building and operations.
Assessment and planning: By their very nature, these programs provide two important insights: If a company is receiving pre-launch demand for a drug, knowing where that demand is coming from can help define the unmet need. Also, knowing about patients’ demographics, diagnosis and previous treatment at the time their physicians make the decision to request the medication helps the company understand where the drug fits into the current treatment pathway.
Relationship building: Named patient programs foster relationship development with local key opinion leaders and other physicians who would be likely prescribers post-launch. Participating physicians may also serve as resources for regulators and other local healthcare providers who are considering the benefits the drug can provide to patients in that country. Post-launch, physicians who have experience with the medication may ultimately be the go-to physicians locally or regionally for others who are trying to gain that experience.
Operations: When launching a new drug, a company must align its deployment and operations objectives around its overall strategy, while tailoring tactics to the local situation. In the context of a named patient program, the sponsoring company can work out any problems in the supply chain, fine-tune its educational support for physicians, pharmacists and patients, and coordinate internal functions ahead of time – increasing the chances that the launch will run smoothly.
- Helene S (2010). "EU Compassionate Use Programmes (CUPs): Regulatory Framework and Points to Consider before CUP Implementation". Pharm Med 24 (4): 223–229. doi:10.1007/bf03256820.
- Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use
- Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency
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- Boundaries of expanded access. Pharmaceutical Executive, May 2008.
- Compassionate use guideline finalized. Applied Clinical Trials, September 2007.
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- Implementing a pre-launch named patient program - evidence of increased market share. Journal of Medical Marketing, September 2008.
- Meeting unmet needs. Pharmaceutical Executive Europe, October 2008.
- Fighting for a Last Chance at Life. New York Times, May 16, 2009.