10 ways to optimise patient access through the product life cycle with Managed Access Programmes

A PMGroup Publication, March 2013, by Idis

Modern healthcare systems are about providing cost-effective and sustainable treatment options that benefit patients, society and the public purse.

Delivering quality care through innovative medicines, whenever and wherever there is unmet medical need, requires flexible market access strategies in an environment marked by unrelenting pressure to contain healthcare costs.

At the same time, patient awareness of therapeutic options throughout the drug life cycle has never been more acute. Cultural change, the internet and the proliferation of dialogue through social media all make for a better educated, empowered patient. This is happening at a breathtaking pace, particularly in the networked world of social media. The number of people using social networks rose by an estimated 174 per cent between March 2007 and October 2011, while internet usage increased by a cumulative 88 per cent in the same period. Health is a big part of that conversation, with some 20 per cent of adults likely to join a health forum. And it is tilting the balance in relations between patients and healthcare professionals.

In the UK, for example, NHS Choice gets an estimated 10 million hits a month, with 74 per cent of patients using it before seeing their GP and 40 per cent using it either as an alternative to, or to reduce their reliance on, GP consultations. These patients know what potential treatments are out there and will question why they do not have access to them, especially where the therapeutic options are limited, ineffective or non-existent.

Addressing that demand is not just to do with clinical development or outcomes data. It goes to the heart of the mission statement of every major pharmaceutical company. It is a concrete demonstration of industry putting patients first and shaping its value offering to their needs.

Strategic imperative

Demand for access to innovative medicines can start building well before drug approval and may continue right through to discontinuation of a product line.

So enabling and maintaining access for patients in need should be a strategic imperative from top management down. Companies should start thinking about Managed Access Programmes (MAPs) as early as possible in the transition from drug development to commercialisation.

They should also look to a specialist provider with intimate knowledge of the various regulations and compliance and quality standards that apply specifically to MAPs across different markets worldwide.

MAPs can take a number of forms: compassionate-use or Expanded Access Programs (EAPs) in the US; NamedPatient Programmes (NPPs) in the EU; or Autorisations Temporaires d’Utilisation (ATUs) in France.

Even within an ostensibly harmonised framework such as the EU’s NPP, each national market has nuances that must be addressed and managed expertly if a MAP is to roll out successfully on a global scale.

These programmes open up access to needed medicines outside conventional commercial marketing or clinical trials parameters. They also address demand in the hiatus between regulatory approval and commercial launch or maintain access once a product has been discontinued commercially.

Market research suggests that around 48 per cent of companies already consider using MAPs during phase III/IIIb of product development and about 10 per cent consider them as early as during phases I and II.

In approximately 38 per cent of cases, companies will use a MAP to broaden access to patients who do not meet the eligibility criteria for clinical trials. Roughly 64 per cent will maintain access for patients who have completed a clinical trial through a MAP; 77 per cent will use the programme to get medicines to non-trial patients while a registration application is under review; and 30 per cent will use it to address patient need after discontinuing product development.

New options

Applying a carefully thought out MAP strategy early in the product life cycle can reap multiple benefits. Most importantly, it can offer new treatment options to healthcare professionals and patients where all other alternatives have been exhausted.

MAPs potentially help to build awareness of new therapies, develop relationships with key opinion leaders, provide realworld insight into treatment and usage patterns and may provide information that is of use to wider teams within pharma such as HEOR. All of these strategies are geared to optimising the product experience and value on all sides.

1. MAPs ARE INTEGRAL FROM AN EARLY STAGE

Considered early enough in the development process, and assigned to a specialist provider with deep-rooted knowledge of regulatory systems and market variations, MAPs are more than just an ethical, efficient and cost-effective way of meeting patient demand for access outside the clinical trial or commercial settings.

They also serve as a strategic tool to inform decisions throughout the product life cycle. Too often, however, manufacturers are ill-prepared to respond efficiently and effectively to patient demand because they run access programmes internally on an ad hoc basis, without advanced planning for budget allocation or resource management.

A strategic approach that assesses the need for a MAP early in each product’s development means you can respond readily and ethically, within preestablished frameworks and in compliance with local regulations, whenever patients and their advocates are seeking access. It is an ethical imperative as much as anything: critically ill patients may not have time to wait for a programme to be set up.

A strategic approach will also allow you to allocate budget for access management proactively while keeping internal resources focused on regulatory approvals and commercial launches. In the longer term, it will enable you to leverage MAPs strategically throughout the product life cycle, to anticipate and meet patient demand on a global scale.

2. REACHING PATIENTS WHO CANNOT ACCESS ONGOING CLINICAL TRIALS

Good news travels fast. Strong clinical trial data can trigger demand for access to an investigational drug from patients who do not meet study protocol criteria or who live too far from a trial site.

For these patients, access to a drug outside the clinical trial setting offers a new and, in many cases, life-saving treatment option.

Implementing a MAP allows your company to address patient needs effectively without adversely impacting ongoing trials. Rather than undermining patient recruitment into registration trials, evaluating patients for your MAP may actually steer eligible patients towards them.

3. A MORE COST-EFFICIENT OPTION FOR MAINTAINING ACCESS AT THE END OF A CLINICAL TRIAL

Pharmaceutical and biotech companies recognise their responsibility to ensure that patients coming out of clinical trials have continued access to therapy until a drug is available commercially.

Open-Label Extension Studies (OLEs) have been the industry standard in these circumstances. However, extending access in this way can involve significant costs, while drawing on internal resources that a company may prefer to leverage elsewhere.

MAPs offer a more appropriate, efficient and cost-effective means of maintaining post-trial access to therapy when that is your primary objective.

4. BRIDGE THE ACCESS GAP BETWEEN APPROVAL AND COMMERCIAL AVAILABILITY

Once a drug is approved for marketing, there may be significant delays before it is actually available to patients. National pricing and reimbursement procedures in particular can slow market entry to such an extent that, for some patients it means the difference between life and death.

The European Federation of Pharmaceutical Industries and Associations (EFPIA) tracks delays through its Patients Waiting to Access Innovative Medicines (WAIT) Indicator, which is published annually.

Figure 1 (opposite) shows the delays in access typically experienced by patients in Europe even when drugs are approved through the European Medicines Agency’s centralised procedure. The data cover all medicines available in 2010 after securing marketing authorisation from the EMA between 2007 to 2009.

For new drugs that could be prescribed under national healthcare provisions, the average time between EMA approval and the ‘accessibility’ date (pricing/ reimbursement procedures completed) in 11 European countries, excluding Germany and the UK, varied between 88 and 392 days.

The proportion of medicines with a valid EMA marketing authorisation available commercially in these countries ranged from 39 per cent for Portugal to 86 per cent for Greece. These delays are even more marked in other territories.

MAPs can bridge these gaps, giving patients regulatory compliant access to medicines in situations where they may otherwise face a long and potentially life-threatening wait for commercial availability.

5. MITIGATE THE RISK OF OPEN MARKET ACCESS

Once a drug is available in one country, a company may lose control of it in international markets where registrations are delayed or where they may never occur.

If patients in these international markets are getting access to your product via distribution channels in the country where it was approved, you may not be able to ensure the drug is stored and handled properly; that instructions on dosing and administration are followed, and the indications treated are those on the labelling.

Shipping conditions may be sub-standard, while inadequate product information may result in compromised efficacy or inappropriate adverse events. Moreover, you may have no idea what price is being charged for your drug when access is uncontrolled.

At the initial approval stage, MAPs provide a formalised approach that mitigates the risks of patient access to a drug through non-validated channels in the open market

6. LEVERAGE MAPs TO DELIVER REAL-WORLD INSIGHTS

As well as offering an important access route for patients with unmet medical needs, MAPs present a unique opportunity to collect real-world insight outside the clinical trial framework.

In today’s fiercely competitive and valueconscious environment, drug makers can no longer rely exclusively on safety and efficacy data from tightly defined clinical trials to secure market access. Increasingly, regulators and payers are demanding evidence of how drugs perform in a realworld setting.

MAPs can provide supportive real-world data insights which in some cases can inform health technology assessments or dossiers and submissions, facilitating understanding of the product and progress towards commercialisation.

These data may highlight efficacy in patient subgroups not represented in a clinical trial, identify global usage patterns, or provide additional safety information outside the controlled environment of a clinical trial prior to any commercial launch.

These data may also provide strategic insights to support internal decision-making and planning. Companies can learn about demand levels by geography, key prescribing centres, treatment pathways and off-label use from one country to another.

7. SUPPORT PHYSICIAN EXPERIENCE AND TRAINING OBJECTIVES

MAPs can help to ensure that early adopter, non-investigator physicians are educated properly in the appropriate use of a drug, driving optimal patient outcomes. This is particularly crucial for complex treatment regimens in areas such as oncology.

These programmes are also an opportunity to identify and work with global thought leaders earlier in the product development process.

8. STRENGTHEN YOUR RELATIONSHIPS WITH PATIENT ADVOCACY GROUPS

Patients look to advocacy groups for information on particular diseases, research advances, clinical trials and MAPs. These groups have strong relationships with key opinion leaders and healthcare providers that help to ensure accurate and timely communications with patients, families and carers.

Advocacy groups can contribute to shaping expectations about MAPs and how they operate alongside clinical trials. They can act as the voice of the patient to the drug developer while pre-screening patients for inclusion in MAPs.

9. PROVIDE ACCESS WHEN COMMERCIAL LAUNCH IS NOT PLANNED OR WHEN A DISCONTINUATION IS IMMINENT

In some global markets, it may not be viable to launch a drug commercially. However, there may still be a small population of patients with unmet needs who could benefit from treatment.

MAPs provide an ethical and compliant access solution in these circumstances, without the formalised registration procedures required for market authorisation or a commercial infrastructure in the local market.

In countries where a company plans to discontinue its product, MAPs provide a means of continued access when patients may have no other treatment options.

10. CONSIDER PARTNERING WITH A MAP SPECIALIST

MAPs enable companies to address unmet patient and healthcare professional needs for new or existing drugs through legal, reliable and ethically driven mechanisms. This includes importing needed products all around the globe in a timely manner and in accordance with local regulations.

The complexity of this undertaking, and in particular its varied application from market to market, calls for expert input.

A MAP specialist can provide you with information essential to securing better managed access, and an understanding of how timely access affects your ongoing relationship with patients and physicians.

A MAP specialist can alleviate the added stress put on internal resources to respond to early access requests inhouse. Since addressing just one patient request for access to a medicine can take valuable time away from internal staff in multiple departments, partnering with a specialist can allow internal resources to remain focused elsewhere.

A MAP specialist can guide you through the development and implementation of a strategic programme that ensures patients with unmet medical needs around the world can secure access to your drug in the most efficient, ethical and compliant way possible. They are experts in the key components of a successful MAP: the in-depth knowledge of local and regional regulations, the flexibility to tailor solutions to your product portfolio, and the ability to reach patients anywhere in the world quickly.

MAPs offer an efficient solution to address the unmet treatment needs of patients throughout the product life cycle. They should be an integral part of every product strategy.

What your peers are saying about MAPs

A recent independent survey of 101 decision-makers from mainly large companies in the pharmaceutical and biotechnology industries generated these insights:

Uses of MAPs

More than 70 per cent of surveyed companies used MAPs to give non-trial patients access to medicines while a registration application was under review.

Over 50 per cent of the companies considered implementing a MAP post-launch when their drug was approved in at least one market.

More than 60 per cent of the companies surveyed used MAPs to continue providing drugs to patients who had completed clinical trials.

Choosing a MAP service partner

Of all the surveyed companies, 73 per cent had used an external MAP service partner in the past, while the remaining 27 per cent planned to do so within the next 12 months.

Over 80 per cent of respondents expressed a preference for using companies that specialise in MAP services.

These respondents felt a specialist provider’s more focused expertise meant he would be more knowledgeable about MAPs, understanding the regulatory needs and the risks involved.

Respondents also thought a specialist provider’s experience enabled him to operate more efficiently and cost effectively than if the MAP were run internally.

Among those attributes seen as most important in selecting a MAP service provider were: level of MAP expertise; ability to provide global coverage; knowledge of the global regulatory landscape; clinical expertise; cost for perceived value; operational expertise; and best-in-class logistics.

Learn more

Idis is a global leader in MAPs; with 25 years of experience working alongside the most innovative pharmaceutical companies to develop and implement these programmes, Idis can create an access strategy customised to your needs.

To learn more about how Managed Access Programmes can optimise your product development efforts visit www.idispharma.com or email solutions@idispharma.com.