Orphan Drugs: European Payer Perspectives

Maximum Purchase:
1 unit
Publication Date:
October 2018
Adding to cart… The item has been added

European payers reveal how pharma can build trust and gain their support for orphan drug products

European payers are concerned about orphan drugs citing unjustified high prices and a lack of evidence of utility and value. Pharma has a trust issue with payers, so how can the industry get back on track and gain European payer support for the expanding orphan drug sector?

Payers may have concerns but they also have ideas. We interviewed experienced frontline payers in France, Germany and the UK to reveal in Orphan Drugs: European Payer Perspectives what they see as the key issues and how industry can proactively engage with them.

Payer experts explore key questions

  • Should the evidence of clinical effectiveness and safety be less stringent for drugs that are intended to treat rare conditions?
  • What factors do payers think manufacturers should take into consideration when setting prices for orphan drugs?
  • Is there any justification for manufacturers charging high prices for branded formulations of drugs that are available generically for other indications?
  • Should there be a correlation between the annual cost of treatment for an orphan drug and the size of the treatable patient population?
  • How can early engagement with payers help to improve planning and manage costs?
  • How does national pricing and reimbursement policy and regulation impact on payer perspectives and what does this mean for pharma's engagement programme?
  • What can drug developers do to ensure that orphan drugs will be considered for coverage and reimbursement funding in the future?

What the frontline experts say…

"I think that's largely around having better information so that there is an understanding within the payer communities of what the potential cost-effectiveness of an orphan drug is compared to how they would normally commission medicines. What we're talking about here is cost equality and it may well be that we will use different thresholds for orphan drugs than we would routinely use within a NICE appraisal process. I think, in terms of [where] access could be improved, there may well be a slight responsibility of the pharma company that's developed the drug to provide the background information in terms of the level of evidence that supports its use, what the tolerability and side-effect profile are, where would it fit within a normal treatment pathway compared to the other medicines and interventions that might be available for a particular condition, and to provide some interpretations to Commissioners so that those choices can be made using the usual type of criteria of cost-effectiveness, affordability, clinical effectiveness, safety and tolerability."
UK Payer 1

"We have lost trust many decades ago… We've had a price increases of 35 percent since introducing the end of regulation in 2011. I think this is incredible. This is not only reserved for orphan drugs, this happens also for chronic diseases. In my opinion, price setting by the pharmaceutical industry is really questionable."
German Payer 3

"The challenge for the payer is to take into account the pressure of patient associations and the media, in case an orphan drug is not [available] because of its price. It is probably difficult for health authorities to justify a situation like that, and so there is probably a high patient pressure that would really push the authorities to take a decision in terms of market access. I think it's a good argument for both public authorities and companies to try to get an agreement as fast as possible. There is also a compassionate use process to get early access to new drugs before that market authorisation. I think there are ways to avoid some delay in market access through the compassionate use process [which] may be used for orphan drugs."
French Payer 1

What to expect

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

  • An examination of the 6 key issues most relevant to European payers
  • 28 targeted questions put to payer experts in France, Germany and the UK,
  • Their responses that provide 49 current insights supported by 114 directly quoted comments

Expert contributors

  • French Payer 1. A health economist and former member of the French Transparency Committee with responsibility for the clinical evaluation of new drugs. Currently works as a consultant in market access.
  • French Payer 2. A former member of the Pricing Committee in France who has been involved in both market approval and market access discussions for all types of conditions.
  • German Payer 1. A member of the German Medical Association who was formerly a member of the Drug Committee of the G-BA (Federal Joint Committee) and has held responsibility for drug negotiations at regional and federal level.
  • German Payer 2. Budget manager for a state in Germany and head of the department for drug reimbursement of a big German payer organisation.
  • German Payer 3. Deputy member of the arbitration board that sets the reimbursement price for normal and orphan drugs in cases where the pharmaceutical company and the National Association of Statutory Health Insurance Funds cannot agree on a reimbursement price.
  • UK Payer 1. Director of an NHS Trust who works closely with Commissioners in NHS England and within local Clinical Commissioning Groups (CCGs), and as part of a regional health technology assessment group.
  • UK Payer 2. Chief Pharmacist in a specialist hospital, responsible for the introduction of new drugs and ensuring that funding comes in from Commissioners.
  • UK Payer 3. Chief Pharmacist for a 900-bed District General Hospital; also a member of a drugs and therapies committee, an area prescribing committee, and the NICE group for both the acute hospital and the CCG.

Why Choose FirstWord?

Are you fed up with management reports that over promise and under deliver? 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