Orphan Drugs: US Payer Perspectives

So what will persuade US payers to better support orphan drugs?

US payer concern at the number of new orphan drugs coming to market, often with eye watering prices attached, can obscure a more positive message: payers will support orphan drug pricing if pharma can prove utility and wider health economic benefit. Failure, however, to prove value or transparently justify your pricing policy, and you can expect ever more severe formulary restrictions. If some payers had their way, you'd be facing government intervention to limit prices.

Pharma's pricing of orphan drugs is seen by many as unjustified and that the industry is pushing payers too hard. There is much you can learn from payers and that is why, in October 2018, we interviewed frontline US payers to reveal in Orphan Drugs: US Payer Perspectives how they see the key issues playing out and what they want pharma to do to gain their support and not their anger.

Payer experts explore key questions

How can pharma companies more effectively assess the factors that dictate the price of orphan drugs?
What innovative market access solutions should pharma companies aim for?
How can pharma companies avoid coverage denials and formulary exclusion for orphan drugs?
How do payers decide on the coverage and reimbursement of orphan therapies with multiple indications?
What factors and metrics should drug developers consider if they want to effectively demonstrate the value of their orphan drug to payers?
Why should pharma see the ongoing post launch generation of real world evidence as an investment and not a cost?
How do payers want to collaborate with pharma companies throughout the drug development lifecycle?

What the frontline experts say…

"The cost is extremely high and does not necessarily correlate to improved outcomes. There is a lack of efficacy or clinical benefit. This is of concern when you have a drug demonstrate statistical significance that is not considered clinically significant or a drug that demonstrates statistical significance in a measure that is not clinically valuable. There is also a lack of contracting opportunities and a lack of competition."
US Payer 7

"From a payer perspective, 'restrictions' is not the best word choice. Instead, we look at our measures as cost and medical necessity 'controls.' These controls include showing proof of medical necessity, documentation that other less costly treatment options were not effective, as well as other standard review and appeal processes to treat with drugs that are not on the member's formulary."
US Payer 8

""We need to develop a payer-agnostic registry to capture long-term outcomes data. This will help to define the long-term clinical value of these novel/expensive drugs. Pharma must become more transparent regarding drug development costs in order to ultimately establish a fair price and ROI for their products."
US Payer 1

"Early engagement will only be successful if there is a level of transparency that allows payers and pharma to address the pain points each party experiences with the launching of new orphan drugs. If there is transparency, payers will be able to contribute to the strategy of delivery and distribution. The more effective the distribution, the lower the cost the product can be."
US Payer 5

What to expect

A detailed report exploring the issues that are impacting US payers, based on:

An examination of the 6 key issues most relevant to US payers
20 targeted questions put to US payer professionals
Their responses that yielded 57 current insights supported by 140 directly quoted comments.

Expert contributors

Payer 1. Is a Medical Director at a large managed healthcare company, working on healthcare reform strategies.
Payer 2. Is Vice President at an American Fortune 500 pharmacy benefit management organisation with responsibility for orphan drug reimbursement.
Payer 3. Is Chief Medical Officer at a provider-sponsored health insurance company with responsibility for reviewing orphan drug policy and reimbursement for all clinical categories.
Payer 4. Is Executive Vice President at a prescription coverage firm with responsibility for overall pharmacy strategic initiatives, including clinical programs, operations, and trade relations with the pharmaceutical industry.
Payer 5. Is Senior Finance Director and Commercial PBM at a Fortune 500 managed care organisation.
Payer 6. Is an experienced Pharmacy Director with responsibilities for several large national and regional health insurance clients as well as with the PBM industry.
Payer 7. Is a Managed Care Executive with over 10 years of US payer experience managing formularies across commercial, Medicaid, and Medicare lines of business.
Payer 8. Is responsible for government managed care benefit programs operations, project management and process improvement

Why Choose FirstWord?

Are you fed up with management reports packed out with secondary research and uncorroborated content? If this is your experience, then turn to FirstWord because…

All our information is derived from primary research with experts whose knowledge and current experience is proven
We harness insights from their first-hand experience and don't pack our reports with secondary information and spurious observations
Our reports are based on the latest research and we don't reuse or recycle content
We have the expertise to create on-target content that dives deeply into the pressing commercial and market access issues of the day

Contact Us

Orphan Drugs: US Payer Perspectives

Browse by