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The Alliance for Patient Access: tackling barriers to healthcare for people with IgG4-RD and other rare diseases


In rare diseases there are often huge challenges for patients. The path to diagnosis is often long and difficult, and there is seldom a specific approved treatment available. Even if a specific treatment is available, patients may struggle to access it for several reasons, not least because of the barriers that insurers put up. The work of the Alliance for Patient Access to help to address these issues, across rare diseases and within IgG4-RD, is explained here

Written by Geoff Case, digital editor, RARE Revolution Magazine
Interview with Josie Cooper, executive director at Alliance for Patient Access
The Alliance for Patient Access

Dr David Charles, a neurologist and movement disorder specialist, started the Alliance for Patient Access (AfPA) in 2006. His goal: an organisation that would empower healthcare providers so they could advocate for policy improvements that would put patients at the heart of healthcare.

Today the non-profit organisation recruits, trains and mobilises providers to tackle the many barriers patients face in accessing the care that is right for them. Josie Cooper, AfPA’s executive director, explains how healthcare providers are well-placed to do this. “They have an incredible perspective: they see first-hand the barriers patients have in accessing the care they need.”

AfPA works across dozens of disease areas, but its attention to rare diseases has increased as more treatments specifically targeting these have been developed. Unfortunately, the sweetness of excitement around the arrival of treatments has often been followed by the bitterness of disappointment later as barriers to access appear.

The rare diseases working group

In 2020 AfPA launched its rare diseases working group to address some of the broad issues affecting people with rare diseases—not least the “pretty narrow framework” that is often used to assess the value of new therapies. Josie argues that policymakers and insurers need to take a holistic view to fully understand the value these therapies bring.

“We believe that determinations of value need to encompass what’s important to patients, to caregivers, to health care providers: they need a voice in that conversation.”

This poor understanding of a therapy’s true value contributes to access problems for patients after approval. Pleasingly, a number of states are establishing rare disease advisory councils to give patients, caregivers, providers and other stakeholders the opportunity to influence decisions impacting rare disease patients, including access. These are the sort of developments that AfPA wants to see.

Even if a specific treatment is available, patients may struggle to access it for several reasons, not least because of the barriers that insurers put up. The work of the Alliance for Patient Access to help to address these issues
The IgG4-RD working group

In parallel with its high-level advocacy to shape legislation and policies, AfPA targets the specific barriers that impede access for patients, and it has a number of initiatives that work in the interests of patients with specific rare diseases. They do this proactively even while new treatments are being developed: barriers to access can be most significant just around the time that new treatments are approved.

“We frequently see patients wait for years in the rare disease space to get a treatment. Then we see folks caught unaware by the access barriers that pop up.”

Disease awareness can be a significant barrier when a new therapy arrives, Josie explains. Just because a treatment has been approved does not mean that patients and doctors have immediate awareness of the disease; if they cannot diagnose it, patients cannot access the treatment for it. IgG4-RD is a relatively newly recognised disease, so that information gap could be a particular issue for these patients.

Another barrier to access, especially soon after a treatment is approved, is cost. Innovation is expensive, so as insurers seek to limit their costs, they often put into place measures that limit access through utilisation management tools or by increasing patients’ costs.

AfPA has carefully recruited a broad range of specialists from its network of volunteers and beyond who have expressed an interest in IgG4-related disease and want to nip these issues in the bud. Reflecting the varied journeys that patients typically take to get a diagnosis, the group welcomes not only key specialists in IgG4-related disease, but also others who may encounter IgG4-RD, including rheumatologists, gastroenterologists and other health care providers.

The group met for the first time in the spring, and AfPA is now writing a policy paper to capture the attendees’ insights into the access barriers that people with IgG4-related disease may face, and the opportunities for educational initiatives and advocacy to address these. Typically, as new treatments are developed, working groups engage with federal and state policymakers and with the insurance industry to help educate them on why timely access to treatment is so critical. The white paper will be reviewed by the providers and published later this year.

“These providers are really incredible folks who are donating their time in order to improve the healthcare system. We’re very grateful for that.”

Even if a specific treatment is available, patients may struggle to access it for several reasons, not least because of the barriers that insurers put up. The work of the Alliance for Patient Access to help to address these issues
Utilisation management tools

Insurers’ use (or misuse) of utilisation management tools is one of the key issues that AfPA wants to address. Conceived as tools to address unnecessary medical costs, they have become a way to limit costs by limiting access for patients, Josie says. The use of these tactics has gotten very out of hand.”

Prior authorisation is a management tool commonly used by insurers. It is a paperwork process that doctors must complete before the insurer approves the prescription of a medicine. “That could be as simple as filling out one form, but it could be filling out 10 forms,” Josie explains. “It could mean hours on the phone, with the insurer trying to negotiate what’s really needed, tracking down lab tests or other things that oftentimes might not be strictly necessary.”

These requirements not only mean that the patient’s care is delayed—for weeks sometimes—but also contribute to significant levels of burnout for doctors. As Josie says, doctors often have to take time out, away from their patients, to go through administrative hoops for a medication they believe is best for their patients. AfPA believes that the misuse of this tool creates a “huge challenge” for the health system.

Step therapy is another tool insurers use. Here, the insurer asks the patient to try other medications that cost the insurer less, prior to accessing the medication prescribed by a health care provider. Access to the treatment that the clinician believes is most beneficial will only be granted if the others are unsuccessful. This may result in delays to effective treatment, new symptoms for the patient and additional costs for the healthcare system.

“It’s short-sighted because it doesn’t factor in what’s best for the patient. But, also, it doesn’t always factor in what’s going to be most cost-effective for the healthcare system overall.”

Non-medical switching is when an insurer decides to switch a stable patient to a different medication, not for any medical reason but because of cost considerations. A patient may only learn of this when they go to the pharmacy. Such changes should be a matter for a doctor and a patient, rather than an insurer, Josie says. When insurers use this management tool, it undermines that vital relationship.

Co-pay accumulator programmes sometimes become another barrier to access. In these, a manufacturer of a treatment provides a coupon to help patients pay towards its cost—and pay down their annual deductible, or out-of-pocket cost. Increasingly insurers are limiting the value of those coupons through co-pay accumulators, which allow a patient to use the coupon, but do not apply the payment toward a patient’s deductible. That means that when the value of the co-pay coupon runs out, the patient faces a large bill that they may not be able to afford. That leaves patients with a complicated decision: do I take this medication and forgo my mortgage payment? Do I go off the medication? Do I ration my medication?”

What these utilisation management tools have in common are time-consuming and energy-sapping protocols that delay access to the care pathways that patients and doctors have agreed upon.

“We want to make sure that clinicians can use all the tools in their toolbox… AfPA’s goal is policy reforms that will protect all patients from these types of tools and put the patient–physician relationship back at the centre of health care decisions.”

Even if a specific treatment is available, patients may struggle to access it for several reasons, not least because of the barriers that insurers put up. The work of the Alliance for Patient Access to help to address these issues
Incremental change

AfPA’s work to systematically tackle and remove barriers to patient care is hugely important because it foregrounds the critical relationship between the patient and the provider. This should be the “backbone of the health care system”, Josie says. Other parties, such as insurance companies and pharmacy benefit managers, who are implementing utilisation management tools, should not be getting in the way.

To date, AfPA’s endeavours have helped to achieve incremental but meaningful changes. There have been reforms to utilisation management tools like step therapy and co-pay accumulators in a number of states, with progress made in dozens more. But, as Josie says, much remains to be done.

“It’s like playing Whack-a-Mole. We knock down a barrier, and they put a new one up. We’re seeing progress, but we’ve got a lot more to do.”

The documents contained in this website are presented for information purposes only. The material is in no way intended to replace professional medical care by a qualified specialist and should not be used as a basis for diagnosis or treatment. This digital spotlight has been made possible with financial support from Horizon Therapeutics, who recommended the potential contributors. With the exception of a factual accuracy check, Horizon Therapeutics had no editorial control over the content of this article. All opinions are those of the contributor. RARE Revolution Magazine and Horizon Therapeutics are not responsible for the content of any external sites linked to within this article. RARE Revolution Magazine retains all copyright.

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